FDA/CDC

The FDA has accepted Pfizer’s application for a COVID-19 vaccine for children under age 5, which clears the way for approval and distribution in June.

Pfizer announced June 1 that it completed the application for a three-dose vaccine for kids between 6 months and 5 years old, and the FDA said it received the emergency use application.

Children in this age group – the last to be eligible for COVID-19 vaccines – could begin getting shots as early as June 21, according to White House COVID-19 response coordinator Ashish Jha, MD.

Meanwhile, COVID-19 cases are still high – an average of 100,000 cases a day – but death numbers are about 90% lower than they were when President Joe Biden first took office, Dr. Jha said. 

The FDA’s advisory group, the Vaccines and Related Biological Products Advisory Committee, is scheduled to meet June 14 and June 15 to discuss data submitted by both Pfizer and Moderna.  

If the FDA gives them the green light, the CDC will then weigh in.

“We know that many, many parents are eager to vaccinate their youngest kids, and it’s important to do this right,” Dr. Jha said at a White House press briefing on June 2. “We expect that vaccinations will begin in earnest as early as June 21 and really roll on throughout that week.”

States can place their orders as early as June 3, Dr. Jha said, and there will initially be 10 million doses available. If the FDA gives emergency use authorization for the vaccines, the government will begin shipping doses to thousands of sites across the country.

“The good news is we have plenty of supply of Pfizer and Moderna vaccines,” Dr. Jha said. “We’ve asked states to distribute to their highest priority sites, serving the highest risk and hardest to reach areas.”

Pfizer’s clinical trials found that three doses of the vaccine for children 6 months to under 5 years were safe and effective and proved to be 80% effective against Omicron.

The FDA announced its meeting information with a conversation about the Moderna vaccine for ages 6-17 scheduled for June 14 and a conversation about the Pfizer and Moderna vaccines for young children scheduled for June 15.

Moderna applied for FDA authorization of its two-dose vaccine for children under age 6 on April 28. The company said the vaccine was 51% effective against infections with symptoms for children ages 6 months to 2 years and 37% effective for ages 2-5.

Pfizer’s 3-microgram dose is one-tenth of its adult dose. Moderna’s 25-microgram dose is one-quarter of its adult dose.

A version of this article first appeared on Medscape.com.

The FDA has accepted Pfizer’s application for a COVID-19 vaccine for children under age 5, which clears the way for approval and distribution in June.

Pfizer announced June 1 that it completed the application for a three-dose vaccine for kids between 6 months and 5 years old, and the FDA said it received the emergency use application.

Children in this age group – the last to be eligible for COVID-19 vaccines – could begin getting shots as early as June 21, according to White House COVID-19 response coordinator Ashish Jha, MD.

Meanwhile, COVID-19 cases are still high – an average of 100,000 cases a day – but death numbers are about 90% lower than they were when President Joe Biden first took office, Dr. Jha said. 

The FDA’s advisory group, the Vaccines and Related Biological Products Advisory Committee, is scheduled to meet June 14 and June 15 to discuss data submitted by both Pfizer and Moderna.  

If the FDA gives them the green light, the CDC will then weigh in.

“We know that many, many parents are eager to vaccinate their youngest kids, and it’s important to do this right,” Dr. Jha said at a White House press briefing on June 2. “We expect that vaccinations will begin in earnest as early as June 21 and really roll on throughout that week.”

States can place their orders as early as June 3, Dr. Jha said, and there will initially be 10 million doses available. If the FDA gives emergency use authorization for the vaccines, the government will begin shipping doses to thousands of sites across the country.

“The good news is we have plenty of supply of Pfizer and Moderna vaccines,” Dr. Jha said. “We’ve asked states to distribute to their highest priority sites, serving the highest risk and hardest to reach areas.”

Pfizer’s clinical trials found that three doses of the vaccine for children 6 months to under 5 years were safe and effective and proved to be 80% effective against Omicron.

The FDA announced its meeting information with a conversation about the Moderna vaccine for ages 6-17 scheduled for June 14 and a conversation about the Pfizer and Moderna vaccines for young children scheduled for June 15.

Moderna applied for FDA authorization of its two-dose vaccine for children under age 6 on April 28. The company said the vaccine was 51% effective against infections with symptoms for children ages 6 months to 2 years and 37% effective for ages 2-5.

Pfizer’s 3-microgram dose is one-tenth of its adult dose. Moderna’s 25-microgram dose is one-quarter of its adult dose.

A version of this article first appeared on Medscape.com.

The FDA has accepted Pfizer’s application for a COVID-19 vaccine for children under age 5, which clears the way for approval and distribution in June.

Pfizer announced June 1 that it completed the application for a three-dose vaccine for kids between 6 months and 5 years old, and the FDA said it received the emergency use application.

Children in this age group – the last to be eligible for COVID-19 vaccines – could begin getting shots as early as June 21, according to White House COVID-19 response coordinator Ashish Jha, MD.

Meanwhile, COVID-19 cases are still high – an average of 100,000 cases a day – but death numbers are about 90% lower than they were when President Joe Biden first took office, Dr. Jha said. 

The FDA’s advisory group, the Vaccines and Related Biological Products Advisory Committee, is scheduled to meet June 14 and June 15 to discuss data submitted by both Pfizer and Moderna.  

If the FDA gives them the green light, the CDC will then weigh in.

“We know that many, many parents are eager to vaccinate their youngest kids, and it’s important to do this right,” Dr. Jha said at a White House press briefing on June 2. “We expect that vaccinations will begin in earnest as early as June 21 and really roll on throughout that week.”

States can place their orders as early as June 3, Dr. Jha said, and there will initially be 10 million doses available. If the FDA gives emergency use authorization for the vaccines, the government will begin shipping doses to thousands of sites across the country.

“The good news is we have plenty of supply of Pfizer and Moderna vaccines,” Dr. Jha said. “We’ve asked states to distribute to their highest priority sites, serving the highest risk and hardest to reach areas.”

Pfizer’s clinical trials found that three doses of the vaccine for children 6 months to under 5 years were safe and effective and proved to be 80% effective against Omicron.

The FDA announced its meeting information with a conversation about the Moderna vaccine for ages 6-17 scheduled for June 14 and a conversation about the Pfizer and Moderna vaccines for young children scheduled for June 15.

Moderna applied for FDA authorization of its two-dose vaccine for children under age 6 on April 28. The company said the vaccine was 51% effective against infections with symptoms for children ages 6 months to 2 years and 37% effective for ages 2-5.

Pfizer’s 3-microgram dose is one-tenth of its adult dose. Moderna’s 25-microgram dose is one-quarter of its adult dose.

A version of this article first appeared on Medscape.com.

The Food and Drug Administration has cleared Abbot’s Freestyle Libre 3 system for use by people aged 4 years and older with diabetes.

The new system was cleared for use for both iOS- and Android-compatible mobile apps, enabling real-time glucose readings in contrast to the “intermittently scanned” capability of prior Libre versions. The Libre 3 allows for optional alarms and notifications of urgent low or high glucose levels, as well as remote monitoring by health care professionals or the patient’s family members and/or friends.

The FreeStyle Libre 3 was granted a CE Mark in Europe in October 2020.

Smaller, thinner, and better integration

According to Abbott, the Libre 3 is the first continuous glucose monitoring (CGM) system to show a mean absolute relative difference (MARD) of less than 8% compared with a gold-standard glucose measure. The average Libre 3 MARD is 7.9%, compared with 9.3% for the Libre 2. The Libre 3 is also the “smallest and thinnest” CGM, roughly the size of two stacked U.S. pennies, worn on the upper arm.

And, the company said, the Libre 3 has a Bluetooth integration of up to 33 feet, a range 50% further than other CGMs.   

This version follows the FreeStyle Libre 2, approved in June 2020, and its compatible iPhone app, approved in August 2021.

The Libre 3 will be priced the same as the Libre 2, at about one-third the cost of other CGM systems. However, it is not currently eligible for Medicare reimbursement. Medicaid eligibility may vary by state.

“I applaud Abbott for making their CGM system the most affordable and addressing disparities in care so patients living with diabetes can avoid complications and optimize their quality of life,” Eugene E. Wright Jr., MD, of Duke University, Durham, N.C., said in an Abbott statement.

“I have seen real-world evidence that diabetes technologies like CGMs have helped my patients safely achieve improved glycemic control,” he said.

The FreeStyle Libre 3 sensor will be available at participating pharmacies later this year.

A version of this article first appeared on Medscape.com.

The Food and Drug Administration has cleared Abbot’s Freestyle Libre 3 system for use by people aged 4 years and older with diabetes.

The new system was cleared for use for both iOS- and Android-compatible mobile apps, enabling real-time glucose readings in contrast to the “intermittently scanned” capability of prior Libre versions. The Libre 3 allows for optional alarms and notifications of urgent low or high glucose levels, as well as remote monitoring by health care professionals or the patient’s family members and/or friends.

The FreeStyle Libre 3 was granted a CE Mark in Europe in October 2020.

Smaller, thinner, and better integration

According to Abbott, the Libre 3 is the first continuous glucose monitoring (CGM) system to show a mean absolute relative difference (MARD) of less than 8% compared with a gold-standard glucose measure. The average Libre 3 MARD is 7.9%, compared with 9.3% for the Libre 2. The Libre 3 is also the “smallest and thinnest” CGM, roughly the size of two stacked U.S. pennies, worn on the upper arm.

And, the company said, the Libre 3 has a Bluetooth integration of up to 33 feet, a range 50% further than other CGMs.   

This version follows the FreeStyle Libre 2, approved in June 2020, and its compatible iPhone app, approved in August 2021.

The Libre 3 will be priced the same as the Libre 2, at about one-third the cost of other CGM systems. However, it is not currently eligible for Medicare reimbursement. Medicaid eligibility may vary by state.

“I applaud Abbott for making their CGM system the most affordable and addressing disparities in care so patients living with diabetes can avoid complications and optimize their quality of life,” Eugene E. Wright Jr., MD, of Duke University, Durham, N.C., said in an Abbott statement.

“I have seen real-world evidence that diabetes technologies like CGMs have helped my patients safely achieve improved glycemic control,” he said.

The FreeStyle Libre 3 sensor will be available at participating pharmacies later this year.

A version of this article first appeared on Medscape.com.

The Food and Drug Administration has cleared Abbot’s Freestyle Libre 3 system for use by people aged 4 years and older with diabetes.

The new system was cleared for use for both iOS- and Android-compatible mobile apps, enabling real-time glucose readings in contrast to the “intermittently scanned” capability of prior Libre versions. The Libre 3 allows for optional alarms and notifications of urgent low or high glucose levels, as well as remote monitoring by health care professionals or the patient’s family members and/or friends.

The FreeStyle Libre 3 was granted a CE Mark in Europe in October 2020.

Smaller, thinner, and better integration

According to Abbott, the Libre 3 is the first continuous glucose monitoring (CGM) system to show a mean absolute relative difference (MARD) of less than 8% compared with a gold-standard glucose measure. The average Libre 3 MARD is 7.9%, compared with 9.3% for the Libre 2. The Libre 3 is also the “smallest and thinnest” CGM, roughly the size of two stacked U.S. pennies, worn on the upper arm.

And, the company said, the Libre 3 has a Bluetooth integration of up to 33 feet, a range 50% further than other CGMs.   

This version follows the FreeStyle Libre 2, approved in June 2020, and its compatible iPhone app, approved in August 2021.

The Libre 3 will be priced the same as the Libre 2, at about one-third the cost of other CGM systems. However, it is not currently eligible for Medicare reimbursement. Medicaid eligibility may vary by state.

“I applaud Abbott for making their CGM system the most affordable and addressing disparities in care so patients living with diabetes can avoid complications and optimize their quality of life,” Eugene E. Wright Jr., MD, of Duke University, Durham, N.C., said in an Abbott statement.

“I have seen real-world evidence that diabetes technologies like CGMs have helped my patients safely achieve improved glycemic control,” he said.

The FreeStyle Libre 3 sensor will be available at participating pharmacies later this year.

A version of this article first appeared on Medscape.com.

The U.S. Food and Drug Administration has approved a label extension for oral risdiplam (Evrysdi, Genentech) to include presymptomatic infants younger than 2 months old with spinal muscular atrophy (SMA).

As previously reported, the FDA first approved oral risdiplam for SMA in children older than age 2 years in 2020.

The FDA expanded the indication for risdiplam to include babies younger than 2 months old because of interim safety and efficacy data from the ongoing RAINBOWFISH study. It includes 25 babies from birth to 6 weeks of age at first dose, all of whom have genetically diagnosed SMA but are not yet presenting with symptoms.

After 12 months of risdiplam treatment, the majority of presymptomatic infants with SMA reached key motor milestones, Genentech said in a news release.

Of the six babies with two or three copies of the SMN2 gene, all were able to sit after 1 year of active treatment, roughly two-thirds could stand, and half could walk independently.

All babies were alive at 12 months without permanent ventilation.

“The approval of Evrysdi for presymptomatic babies is particularly important, as early treatment of SMA, before symptoms start to arise, can help babies to achieve motor milestones,” Richard Finkel, MD, principal investigator of the trial, said in the release.

“With the inclusion of SMA in newborn screening programs, this approval provides the opportunity to start treating at home with Evrysdi soon after the diagnosis is confirmed,” added Dr. Finkel, who is director of the experimental neuroscience program, St. Jude Children’s Research Hospital, Memphis.
 

From newborns to older adults?

SMA is a rare and often fatal genetic disease that causes muscle weakness and progressive loss of movement.

SMA, which affects about 1 in 10,000 babies, is caused by a mutation in the survival motor neuron 1 (SMN1) gene. The gene encodes the SMN protein, which is critical for the maintenance and function of motor neurons.

Risdiplam is an orally administered, centrally and peripherally distributed small molecule that modulates survival motor neuron 2 (SMN2) premessenger RNA splicing to increase SMN protein levels.

As part of the label extension, the prescribing information for risdiplam has also been updated to include 2-year pooled data from parts 1 and 2 of the FIREFISH study, which demonstrated long-term efficacy and safety in symptomatic infants with Type 1 SMA, the company noted.

“Because of its efficacy in multiple settings, Evrysdi is now available for people with SMA, from presymptomatic newborns to older adults,” Levi Garraway, MD, PhD, chief medical officer and head of global product development at Genentech, said in the release. 

“We are proud of this achievement, which has the potential to make a real difference to those living with SMA and their caregivers,” Dr. Garraway added.

A version of this article first appeared on Medscape.com.

The U.S. Food and Drug Administration has approved a label extension for oral risdiplam (Evrysdi, Genentech) to include presymptomatic infants younger than 2 months old with spinal muscular atrophy (SMA).

As previously reported, the FDA first approved oral risdiplam for SMA in children older than age 2 years in 2020.

The FDA expanded the indication for risdiplam to include babies younger than 2 months old because of interim safety and efficacy data from the ongoing RAINBOWFISH study. It includes 25 babies from birth to 6 weeks of age at first dose, all of whom have genetically diagnosed SMA but are not yet presenting with symptoms.

After 12 months of risdiplam treatment, the majority of presymptomatic infants with SMA reached key motor milestones, Genentech said in a news release.

Of the six babies with two or three copies of the SMN2 gene, all were able to sit after 1 year of active treatment, roughly two-thirds could stand, and half could walk independently.

All babies were alive at 12 months without permanent ventilation.

“The approval of Evrysdi for presymptomatic babies is particularly important, as early treatment of SMA, before symptoms start to arise, can help babies to achieve motor milestones,” Richard Finkel, MD, principal investigator of the trial, said in the release.

“With the inclusion of SMA in newborn screening programs, this approval provides the opportunity to start treating at home with Evrysdi soon after the diagnosis is confirmed,” added Dr. Finkel, who is director of the experimental neuroscience program, St. Jude Children’s Research Hospital, Memphis.
 

From newborns to older adults?

SMA is a rare and often fatal genetic disease that causes muscle weakness and progressive loss of movement.

SMA, which affects about 1 in 10,000 babies, is caused by a mutation in the survival motor neuron 1 (SMN1) gene. The gene encodes the SMN protein, which is critical for the maintenance and function of motor neurons.

Risdiplam is an orally administered, centrally and peripherally distributed small molecule that modulates survival motor neuron 2 (SMN2) premessenger RNA splicing to increase SMN protein levels.

As part of the label extension, the prescribing information for risdiplam has also been updated to include 2-year pooled data from parts 1 and 2 of the FIREFISH study, which demonstrated long-term efficacy and safety in symptomatic infants with Type 1 SMA, the company noted.

“Because of its efficacy in multiple settings, Evrysdi is now available for people with SMA, from presymptomatic newborns to older adults,” Levi Garraway, MD, PhD, chief medical officer and head of global product development at Genentech, said in the release. 

“We are proud of this achievement, which has the potential to make a real difference to those living with SMA and their caregivers,” Dr. Garraway added.

A version of this article first appeared on Medscape.com.

The U.S. Food and Drug Administration has approved a label extension for oral risdiplam (Evrysdi, Genentech) to include presymptomatic infants younger than 2 months old with spinal muscular atrophy (SMA).

As previously reported, the FDA first approved oral risdiplam for SMA in children older than age 2 years in 2020.

The FDA expanded the indication for risdiplam to include babies younger than 2 months old because of interim safety and efficacy data from the ongoing RAINBOWFISH study. It includes 25 babies from birth to 6 weeks of age at first dose, all of whom have genetically diagnosed SMA but are not yet presenting with symptoms.

After 12 months of risdiplam treatment, the majority of presymptomatic infants with SMA reached key motor milestones, Genentech said in a news release.

Of the six babies with two or three copies of the SMN2 gene, all were able to sit after 1 year of active treatment, roughly two-thirds could stand, and half could walk independently.

All babies were alive at 12 months without permanent ventilation.

“The approval of Evrysdi for presymptomatic babies is particularly important, as early treatment of SMA, before symptoms start to arise, can help babies to achieve motor milestones,” Richard Finkel, MD, principal investigator of the trial, said in the release.

“With the inclusion of SMA in newborn screening programs, this approval provides the opportunity to start treating at home with Evrysdi soon after the diagnosis is confirmed,” added Dr. Finkel, who is director of the experimental neuroscience program, St. Jude Children’s Research Hospital, Memphis.
 

From newborns to older adults?

SMA is a rare and often fatal genetic disease that causes muscle weakness and progressive loss of movement.

SMA, which affects about 1 in 10,000 babies, is caused by a mutation in the survival motor neuron 1 (SMN1) gene. The gene encodes the SMN protein, which is critical for the maintenance and function of motor neurons.

Risdiplam is an orally administered, centrally and peripherally distributed small molecule that modulates survival motor neuron 2 (SMN2) premessenger RNA splicing to increase SMN protein levels.

As part of the label extension, the prescribing information for risdiplam has also been updated to include 2-year pooled data from parts 1 and 2 of the FIREFISH study, which demonstrated long-term efficacy and safety in symptomatic infants with Type 1 SMA, the company noted.

“Because of its efficacy in multiple settings, Evrysdi is now available for people with SMA, from presymptomatic newborns to older adults,” Levi Garraway, MD, PhD, chief medical officer and head of global product development at Genentech, said in the release. 

“We are proud of this achievement, which has the potential to make a real difference to those living with SMA and their caregivers,” Dr. Garraway added.

A version of this article first appeared on Medscape.com.

The U.S. Food and Drug Administration is easing rules to allow infant formula imports from the United Kingdom, which would bring about 2 million cans to the U.S. in coming weeks.

Kendal Nutricare will be able to offer certain infant formula products under the Kendamil brand to ease the nationwide formula shortage.

“Importantly, we anticipate additional infant formula products may be safely and quickly imported in the U.S. in the near-term, based on ongoing discussions with manufacturers and suppliers worldwide,” Robert Califf, MD, the FDA commissioner, said in a statement.

Kendal Nutricare has more than 40,000 cans in stock for immediate dispatch, the FDA said, and the U.S. Department of Health and Human Services is talking to the company about the best ways to get the products to the U.S. as quickly as possible.

Kendamil has set up a website for consumers to receive updates and find products once they arrive in the U.S.

After an evaluation, the FDA said it had no safety or nutrition concerns about the products. The evaluation reviewed the company’s microbiological testing, labeling, and information about facility production and inspection history.

On May 24, the FDA announced that Abbott Nutrition will release about 300,000 cans of its EleCare specialty amino acid-based formula to families that need urgent, life-sustaining supplies. The products had more tests for microbes before release.

Although some EleCare products were included in Abbott’s infant formula recall earlier this year, the cans that will be released were in different lots, have never been released, and have been maintained in storage, the FDA said.

“These EleCare product lots were not part of the recall but have been on hold due to concerns that they were produced under unsanitary conditions observed at Abbott Nutrition’s Sturgis, Michigan, facility,” the FDA wrote.

The FDA encourages parents and caregivers to talk with their health care providers to weigh the potential risk of bacterial infection with the critical need for the product, based on its special dietary formulation for infants with severe food allergies or gut disorders.

The FDA also said that Abbott confirmed the EleCare products will be the first formula produced at the Sturgis facility when it restarts production soon. Other specialty metabolic formulas will follow.

Abbott plans to restart production at the Sturgis facility on June 4, the company said in a statement, noting that the early batches of EleCare would be available to consumers around June 20.

The products being released now are EleCare (for infants under 1 year) and EleCare Jr. (for ages 1 and older). Those who want to request products should contact their health care providers or call Abbott directly at 800-881-0876.

A version of this article first appeared on WebMD.com.

The U.S. Food and Drug Administration is easing rules to allow infant formula imports from the United Kingdom, which would bring about 2 million cans to the U.S. in coming weeks.

Kendal Nutricare will be able to offer certain infant formula products under the Kendamil brand to ease the nationwide formula shortage.

“Importantly, we anticipate additional infant formula products may be safely and quickly imported in the U.S. in the near-term, based on ongoing discussions with manufacturers and suppliers worldwide,” Robert Califf, MD, the FDA commissioner, said in a statement.

Kendal Nutricare has more than 40,000 cans in stock for immediate dispatch, the FDA said, and the U.S. Department of Health and Human Services is talking to the company about the best ways to get the products to the U.S. as quickly as possible.

Kendamil has set up a website for consumers to receive updates and find products once they arrive in the U.S.

After an evaluation, the FDA said it had no safety or nutrition concerns about the products. The evaluation reviewed the company’s microbiological testing, labeling, and information about facility production and inspection history.

On May 24, the FDA announced that Abbott Nutrition will release about 300,000 cans of its EleCare specialty amino acid-based formula to families that need urgent, life-sustaining supplies. The products had more tests for microbes before release.

Although some EleCare products were included in Abbott’s infant formula recall earlier this year, the cans that will be released were in different lots, have never been released, and have been maintained in storage, the FDA said.

“These EleCare product lots were not part of the recall but have been on hold due to concerns that they were produced under unsanitary conditions observed at Abbott Nutrition’s Sturgis, Michigan, facility,” the FDA wrote.

The FDA encourages parents and caregivers to talk with their health care providers to weigh the potential risk of bacterial infection with the critical need for the product, based on its special dietary formulation for infants with severe food allergies or gut disorders.

The FDA also said that Abbott confirmed the EleCare products will be the first formula produced at the Sturgis facility when it restarts production soon. Other specialty metabolic formulas will follow.

Abbott plans to restart production at the Sturgis facility on June 4, the company said in a statement, noting that the early batches of EleCare would be available to consumers around June 20.

The products being released now are EleCare (for infants under 1 year) and EleCare Jr. (for ages 1 and older). Those who want to request products should contact their health care providers or call Abbott directly at 800-881-0876.

A version of this article first appeared on WebMD.com.

The U.S. Food and Drug Administration is easing rules to allow infant formula imports from the United Kingdom, which would bring about 2 million cans to the U.S. in coming weeks.

Kendal Nutricare will be able to offer certain infant formula products under the Kendamil brand to ease the nationwide formula shortage.

“Importantly, we anticipate additional infant formula products may be safely and quickly imported in the U.S. in the near-term, based on ongoing discussions with manufacturers and suppliers worldwide,” Robert Califf, MD, the FDA commissioner, said in a statement.

Kendal Nutricare has more than 40,000 cans in stock for immediate dispatch, the FDA said, and the U.S. Department of Health and Human Services is talking to the company about the best ways to get the products to the U.S. as quickly as possible.

Kendamil has set up a website for consumers to receive updates and find products once they arrive in the U.S.

After an evaluation, the FDA said it had no safety or nutrition concerns about the products. The evaluation reviewed the company’s microbiological testing, labeling, and information about facility production and inspection history.

On May 24, the FDA announced that Abbott Nutrition will release about 300,000 cans of its EleCare specialty amino acid-based formula to families that need urgent, life-sustaining supplies. The products had more tests for microbes before release.

Although some EleCare products were included in Abbott’s infant formula recall earlier this year, the cans that will be released were in different lots, have never been released, and have been maintained in storage, the FDA said.

“These EleCare product lots were not part of the recall but have been on hold due to concerns that they were produced under unsanitary conditions observed at Abbott Nutrition’s Sturgis, Michigan, facility,” the FDA wrote.

The FDA encourages parents and caregivers to talk with their health care providers to weigh the potential risk of bacterial infection with the critical need for the product, based on its special dietary formulation for infants with severe food allergies or gut disorders.

The FDA also said that Abbott confirmed the EleCare products will be the first formula produced at the Sturgis facility when it restarts production soon. Other specialty metabolic formulas will follow.

Abbott plans to restart production at the Sturgis facility on June 4, the company said in a statement, noting that the early batches of EleCare would be available to consumers around June 20.

The products being released now are EleCare (for infants under 1 year) and EleCare Jr. (for ages 1 and older). Those who want to request products should contact their health care providers or call Abbott directly at 800-881-0876.

A version of this article first appeared on WebMD.com.

The anticipated U.S. Food and Drug Administration approval of VP-102 for the treatment of molluscum contagiosum has been delayed because of deficiencies identified at a contract manufacturing organization, according to Verrica Pharmaceuticals, which is developing the product.

VP-102 is a proprietary drug-device combination of cantharidin 0.7% administered through a single-use precision applicator, which has been evaluated in phase 3 studies of patients with molluscum aged 2 years and older. It features a visualization agent so the person applying the drug can see which lesions have been treated. It also contains a bittering agent to mitigate oral ingestion by children.

According to a press release from Verrica, the only deficiency listed in the FDA’s complete response letter stemmed from a general reinspection of Sterling Pharmaceuticals Services, which manufactures Verrica’s bulk solution drug product. Although none of the issues identified by the FDA during the reinspection were specific to the manufacturing of VP-102, FDA policy prevents approval of a new drug application when a contract manufacturing organization has an unresolved classification status or is placed on “official action indicated” status.

According to the press release, Verrica will “continue to work collaboratively” with the FDA to bring VP-102 to the market as soon as possible. The company has completed phase 2 studies of VP-102 for the treatment of common warts and for the treatment of external genital warts, the release said.

A version of this article first appeared on Medscape.com.

The anticipated U.S. Food and Drug Administration approval of VP-102 for the treatment of molluscum contagiosum has been delayed because of deficiencies identified at a contract manufacturing organization, according to Verrica Pharmaceuticals, which is developing the product.

VP-102 is a proprietary drug-device combination of cantharidin 0.7% administered through a single-use precision applicator, which has been evaluated in phase 3 studies of patients with molluscum aged 2 years and older. It features a visualization agent so the person applying the drug can see which lesions have been treated. It also contains a bittering agent to mitigate oral ingestion by children.

According to a press release from Verrica, the only deficiency listed in the FDA’s complete response letter stemmed from a general reinspection of Sterling Pharmaceuticals Services, which manufactures Verrica’s bulk solution drug product. Although none of the issues identified by the FDA during the reinspection were specific to the manufacturing of VP-102, FDA policy prevents approval of a new drug application when a contract manufacturing organization has an unresolved classification status or is placed on “official action indicated” status.

According to the press release, Verrica will “continue to work collaboratively” with the FDA to bring VP-102 to the market as soon as possible. The company has completed phase 2 studies of VP-102 for the treatment of common warts and for the treatment of external genital warts, the release said.

A version of this article first appeared on Medscape.com.

The anticipated U.S. Food and Drug Administration approval of VP-102 for the treatment of molluscum contagiosum has been delayed because of deficiencies identified at a contract manufacturing organization, according to Verrica Pharmaceuticals, which is developing the product.

VP-102 is a proprietary drug-device combination of cantharidin 0.7% administered through a single-use precision applicator, which has been evaluated in phase 3 studies of patients with molluscum aged 2 years and older. It features a visualization agent so the person applying the drug can see which lesions have been treated. It also contains a bittering agent to mitigate oral ingestion by children.

According to a press release from Verrica, the only deficiency listed in the FDA’s complete response letter stemmed from a general reinspection of Sterling Pharmaceuticals Services, which manufactures Verrica’s bulk solution drug product. Although none of the issues identified by the FDA during the reinspection were specific to the manufacturing of VP-102, FDA policy prevents approval of a new drug application when a contract manufacturing organization has an unresolved classification status or is placed on “official action indicated” status.

According to the press release, Verrica will “continue to work collaboratively” with the FDA to bring VP-102 to the market as soon as possible. The company has completed phase 2 studies of VP-102 for the treatment of common warts and for the treatment of external genital warts, the release said.

A version of this article first appeared on Medscape.com.

More than 3 million live births occurred in the United States in 2021, the largest increase in the nation’s birth rate since 2014, according to the U.S. Centers for Disease Control and Prevention.

Provisional data showed a 1% uptick in births, to 3.66 million, after 6 years of dropping by approximately 2% per year. The gains were concentrated among birthing people ages 25 and older. Teenage births, on the other hand, are at their lowest level since the 1990s, according to the CDC. The agency reported a record 6% decrease in births for teenagers aged 15 to 19 years between 2020 and 2021. Women ages 20 to 25 years also had a record decrease in births of 4% during that period.

Brady E. Hamilton, PhD, of the CDC’s National Center for Health Statistics, and the lead author of the new report, said the rise in births points to childbearing that was postponed during the pandemic. Data from 2021 showed a 4% drop in the nation’s birth rate between 2019 and 2020.

“The option to forgo birth is not always viable for older women, but you saw a lot of that during the pandemic,” Dr. Hamilton said. “Events happened related to job security and the economy that caused people to wait to have a child.”

Dr. Hamilton said more data are needed to determine the full impact of increased overall birth rates on individuals. The final report, which will be released in July, will delve deeper into the influence increased birth rates had on demographics and preterm births, which Dr. Hamilton and his team found have increased by 4%.

“For those beginning to have children, we see these trends, but it will be interesting to see what happens to younger women in the future,” Dr. Hamilton said. “Once we have the final data for 2021, we will be able to see a more detailed pattern emerge and draw conclusions from that.”

Dr. Hamilton has disclosed no relevant financial relationships.

A version of this article first appeared on Medscape.com.

More than 3 million live births occurred in the United States in 2021, the largest increase in the nation’s birth rate since 2014, according to the U.S. Centers for Disease Control and Prevention.

Provisional data showed a 1% uptick in births, to 3.66 million, after 6 years of dropping by approximately 2% per year. The gains were concentrated among birthing people ages 25 and older. Teenage births, on the other hand, are at their lowest level since the 1990s, according to the CDC. The agency reported a record 6% decrease in births for teenagers aged 15 to 19 years between 2020 and 2021. Women ages 20 to 25 years also had a record decrease in births of 4% during that period.

Brady E. Hamilton, PhD, of the CDC’s National Center for Health Statistics, and the lead author of the new report, said the rise in births points to childbearing that was postponed during the pandemic. Data from 2021 showed a 4% drop in the nation’s birth rate between 2019 and 2020.

“The option to forgo birth is not always viable for older women, but you saw a lot of that during the pandemic,” Dr. Hamilton said. “Events happened related to job security and the economy that caused people to wait to have a child.”

Dr. Hamilton said more data are needed to determine the full impact of increased overall birth rates on individuals. The final report, which will be released in July, will delve deeper into the influence increased birth rates had on demographics and preterm births, which Dr. Hamilton and his team found have increased by 4%.

“For those beginning to have children, we see these trends, but it will be interesting to see what happens to younger women in the future,” Dr. Hamilton said. “Once we have the final data for 2021, we will be able to see a more detailed pattern emerge and draw conclusions from that.”

Dr. Hamilton has disclosed no relevant financial relationships.

A version of this article first appeared on Medscape.com.

More than 3 million live births occurred in the United States in 2021, the largest increase in the nation’s birth rate since 2014, according to the U.S. Centers for Disease Control and Prevention.

Provisional data showed a 1% uptick in births, to 3.66 million, after 6 years of dropping by approximately 2% per year. The gains were concentrated among birthing people ages 25 and older. Teenage births, on the other hand, are at their lowest level since the 1990s, according to the CDC. The agency reported a record 6% decrease in births for teenagers aged 15 to 19 years between 2020 and 2021. Women ages 20 to 25 years also had a record decrease in births of 4% during that period.

Brady E. Hamilton, PhD, of the CDC’s National Center for Health Statistics, and the lead author of the new report, said the rise in births points to childbearing that was postponed during the pandemic. Data from 2021 showed a 4% drop in the nation’s birth rate between 2019 and 2020.

“The option to forgo birth is not always viable for older women, but you saw a lot of that during the pandemic,” Dr. Hamilton said. “Events happened related to job security and the economy that caused people to wait to have a child.”

Dr. Hamilton said more data are needed to determine the full impact of increased overall birth rates on individuals. The final report, which will be released in July, will delve deeper into the influence increased birth rates had on demographics and preterm births, which Dr. Hamilton and his team found have increased by 4%.

“For those beginning to have children, we see these trends, but it will be interesting to see what happens to younger women in the future,” Dr. Hamilton said. “Once we have the final data for 2021, we will be able to see a more detailed pattern emerge and draw conclusions from that.”

Dr. Hamilton has disclosed no relevant financial relationships.

A version of this article first appeared on Medscape.com.

Regulators and the nation’s largest physician organization took separate steps in recent days to prepare for expected authorization of use of COVID-19 vaccines in children younger than age 6.

The Food and Drug Administration on May 23 announced its Vaccines and Related Biological Products Advisory Committee will meet June 15 to discuss expanding the use of COVID vaccines from Pfizer and Moderna.

The panel will examine a request from Pfizer and its partner BioNTech for an emergency use authorization (EUA) of its vaccine to cover children ages 6 months through 4 years. The EUA expansion for the Moderna shot would cover children ages 6 months through 5 years, the FDA said.

Many parents and physicians have been urging regulators to clear COVID shots for young children, among whom rates of infection are high.

The American Medical Association in February announced an update of its Current Procedural Terminology (CPT) to prepare for an eventual FDA clearance of the Pfizer-BioNTech shot for children aged 6 months to younger than 5 years. On May 19, the association announced a new CPT update to prepare for FDA clearance for use of the Moderna COVID-19 vaccine for children 6 months through 5 years.

“Extending COVID-19 vaccination protection to approximately 18 million young children will significantly reduce their risk of COVID-19 infection, hospitalization, and death, and give their parents incredible peace of mind,” Gerald Harmon, MD, AMA’s president, said in a statement. “We strongly urge all parents to get their infants and toddlers vaccinated as soon as they are eligible for a COVID-19 vaccine.”

Both the Moderna and the Pfizer-BioNTech COVID vaccines would be given to these young children in low doses.

On May 23, Pfizer announced results from a phase 2/3 trial evaluating a series of three shots of its vaccine in children ages 6 months to younger than 5 years.

Vaccine efficacy, which was a secondary endpoint in this study, was 80.3% in this age group, Pfizer said. The analysis was based on 10 symptomatic cases of COVID-19. The trial’s protocol specifies a formal analysis will be performed when at least 21 cases have accrued from 7 days after the third dose. The company said it would share final data on the effectiveness of the vaccine once the results are available.

Moderna on April 28 issued a statement with details about testing of its vaccine in young children. Vaccine efficacy was estimated at about 51% for children aged 6 months to younger than 2 years and 37% for the children aged 2 years to younger than 6. Paul Burton, MD, Moderna’s chief medical officer, spoke about this rate during a May 1 appearance on CBS’ Face the Nation.

“What it means for parents, for caregivers, is that if they give the Moderna vaccine to these little kids, they would basically cut in half the risk of that child getting symptomatic COVID,” Dr. Burton said in the interview. “Now, the number, 50%, I know is often lower than we are used to seeing with our vaccine, but it’s because this study was conducted during a time of Omicron.”

The FDA’s vaccine advisory committee also will meet on June 14 discuss potential use under an EUA of Moderna’s COVID vaccine for children and teenagers aged 6-17 years. The Pfizer-BioNTech vaccine already is authorized under an EUA for people aged 5 years and older.

The FDA has to date granted both conditional clearances, or EUAs, and regular approvals for COVID vaccines.

EUAs are meant to be temporary, allowing for rapid introduction of medicines in response to public health crises such as the pandemic. The FDA also uses EUAs to provide initial clearances of additional indications for products, as would be the case with the authorizations Moderna and Pfizer-BioNTech are seeking for their COVID vaccines.

Companies that want to continue to sell EUA-cleared products or promote EUA-cleared indications beyond the time of the public health crisis must seek regular approvals.

The FDA cleared the Pfizer-BioNTech and Moderna COVID vaccines under EUAs in December 2020. The agency then granted a regular approval for the Pfizer-BioNTech vaccine for people ages 16 and older in August 2021 based on more robust data. Regular approval for the Moderna vaccine for people ages 18 and older followed in January 2022.
 

Varied reactions among parents

Attitudes in the United States about pediatric COVID vaccines are far from uniform.

The initial uptake has disappointed physicians and researchers, who have been urging wider use of the COVID vaccination among children and teens for whom the FDA already has granted a clearance. Many parents are hesitating to bring their children for the COVID vaccines, according to the Centers for Disease Control and Prevention. Only 35.4% of children ages 5-11 had received at least one dose of a COVID vaccine, CDC staff said during a meeting.

Yet many other parents are demanding this medicine for their young children, urging the FDA to move quickly to clear COVID shots.

A private Facebook group called “Protect Their Future: A Call to Action for COVID Vaccines in Kids <5” boasts about 6,200 members. Many parents and physicians have used Twitter in recent months to press for a speedy review of COVID vaccines for the youngest children, often using the hashtag #immunizeunder5s. A group called Protect Their Future, which uses @ImmunizeUnder5s as its Twitter handle, had 5,288 followers as of the afternoon of May 23.

A special panel of the House of Representatives, the Select Subcommittee on the Coronavirus Crisis, on May 23 joined those tweeting about the need to soon authorize COVID vaccines for very young children.

“Parents have been waiting many months for vaccines for their young children,” the subcommittee tweeted. “They deserve to hear from @US_FDA why this lengthy process has been in children’s best interests.”

A version of this article first appeared on Medscape.com.

Regulators and the nation’s largest physician organization took separate steps in recent days to prepare for expected authorization of use of COVID-19 vaccines in children younger than age 6.

The Food and Drug Administration on May 23 announced its Vaccines and Related Biological Products Advisory Committee will meet June 15 to discuss expanding the use of COVID vaccines from Pfizer and Moderna.

The panel will examine a request from Pfizer and its partner BioNTech for an emergency use authorization (EUA) of its vaccine to cover children ages 6 months through 4 years. The EUA expansion for the Moderna shot would cover children ages 6 months through 5 years, the FDA said.

Many parents and physicians have been urging regulators to clear COVID shots for young children, among whom rates of infection are high.

The American Medical Association in February announced an update of its Current Procedural Terminology (CPT) to prepare for an eventual FDA clearance of the Pfizer-BioNTech shot for children aged 6 months to younger than 5 years. On May 19, the association announced a new CPT update to prepare for FDA clearance for use of the Moderna COVID-19 vaccine for children 6 months through 5 years.

“Extending COVID-19 vaccination protection to approximately 18 million young children will significantly reduce their risk of COVID-19 infection, hospitalization, and death, and give their parents incredible peace of mind,” Gerald Harmon, MD, AMA’s president, said in a statement. “We strongly urge all parents to get their infants and toddlers vaccinated as soon as they are eligible for a COVID-19 vaccine.”

Both the Moderna and the Pfizer-BioNTech COVID vaccines would be given to these young children in low doses.

On May 23, Pfizer announced results from a phase 2/3 trial evaluating a series of three shots of its vaccine in children ages 6 months to younger than 5 years.

Vaccine efficacy, which was a secondary endpoint in this study, was 80.3% in this age group, Pfizer said. The analysis was based on 10 symptomatic cases of COVID-19. The trial’s protocol specifies a formal analysis will be performed when at least 21 cases have accrued from 7 days after the third dose. The company said it would share final data on the effectiveness of the vaccine once the results are available.

Moderna on April 28 issued a statement with details about testing of its vaccine in young children. Vaccine efficacy was estimated at about 51% for children aged 6 months to younger than 2 years and 37% for the children aged 2 years to younger than 6. Paul Burton, MD, Moderna’s chief medical officer, spoke about this rate during a May 1 appearance on CBS’ Face the Nation.

“What it means for parents, for caregivers, is that if they give the Moderna vaccine to these little kids, they would basically cut in half the risk of that child getting symptomatic COVID,” Dr. Burton said in the interview. “Now, the number, 50%, I know is often lower than we are used to seeing with our vaccine, but it’s because this study was conducted during a time of Omicron.”

The FDA’s vaccine advisory committee also will meet on June 14 discuss potential use under an EUA of Moderna’s COVID vaccine for children and teenagers aged 6-17 years. The Pfizer-BioNTech vaccine already is authorized under an EUA for people aged 5 years and older.

The FDA has to date granted both conditional clearances, or EUAs, and regular approvals for COVID vaccines.

EUAs are meant to be temporary, allowing for rapid introduction of medicines in response to public health crises such as the pandemic. The FDA also uses EUAs to provide initial clearances of additional indications for products, as would be the case with the authorizations Moderna and Pfizer-BioNTech are seeking for their COVID vaccines.

Companies that want to continue to sell EUA-cleared products or promote EUA-cleared indications beyond the time of the public health crisis must seek regular approvals.

The FDA cleared the Pfizer-BioNTech and Moderna COVID vaccines under EUAs in December 2020. The agency then granted a regular approval for the Pfizer-BioNTech vaccine for people ages 16 and older in August 2021 based on more robust data. Regular approval for the Moderna vaccine for people ages 18 and older followed in January 2022.
 

Varied reactions among parents

Attitudes in the United States about pediatric COVID vaccines are far from uniform.

The initial uptake has disappointed physicians and researchers, who have been urging wider use of the COVID vaccination among children and teens for whom the FDA already has granted a clearance. Many parents are hesitating to bring their children for the COVID vaccines, according to the Centers for Disease Control and Prevention. Only 35.4% of children ages 5-11 had received at least one dose of a COVID vaccine, CDC staff said during a meeting.

Yet many other parents are demanding this medicine for their young children, urging the FDA to move quickly to clear COVID shots.

A private Facebook group called “Protect Their Future: A Call to Action for COVID Vaccines in Kids <5” boasts about 6,200 members. Many parents and physicians have used Twitter in recent months to press for a speedy review of COVID vaccines for the youngest children, often using the hashtag #immunizeunder5s. A group called Protect Their Future, which uses @ImmunizeUnder5s as its Twitter handle, had 5,288 followers as of the afternoon of May 23.

A special panel of the House of Representatives, the Select Subcommittee on the Coronavirus Crisis, on May 23 joined those tweeting about the need to soon authorize COVID vaccines for very young children.

“Parents have been waiting many months for vaccines for their young children,” the subcommittee tweeted. “They deserve to hear from @US_FDA why this lengthy process has been in children’s best interests.”

A version of this article first appeared on Medscape.com.

Regulators and the nation’s largest physician organization took separate steps in recent days to prepare for expected authorization of use of COVID-19 vaccines in children younger than age 6.

The Food and Drug Administration on May 23 announced its Vaccines and Related Biological Products Advisory Committee will meet June 15 to discuss expanding the use of COVID vaccines from Pfizer and Moderna.

The panel will examine a request from Pfizer and its partner BioNTech for an emergency use authorization (EUA) of its vaccine to cover children ages 6 months through 4 years. The EUA expansion for the Moderna shot would cover children ages 6 months through 5 years, the FDA said.

Many parents and physicians have been urging regulators to clear COVID shots for young children, among whom rates of infection are high.

The American Medical Association in February announced an update of its Current Procedural Terminology (CPT) to prepare for an eventual FDA clearance of the Pfizer-BioNTech shot for children aged 6 months to younger than 5 years. On May 19, the association announced a new CPT update to prepare for FDA clearance for use of the Moderna COVID-19 vaccine for children 6 months through 5 years.

“Extending COVID-19 vaccination protection to approximately 18 million young children will significantly reduce their risk of COVID-19 infection, hospitalization, and death, and give their parents incredible peace of mind,” Gerald Harmon, MD, AMA’s president, said in a statement. “We strongly urge all parents to get their infants and toddlers vaccinated as soon as they are eligible for a COVID-19 vaccine.”

Both the Moderna and the Pfizer-BioNTech COVID vaccines would be given to these young children in low doses.

On May 23, Pfizer announced results from a phase 2/3 trial evaluating a series of three shots of its vaccine in children ages 6 months to younger than 5 years.

Vaccine efficacy, which was a secondary endpoint in this study, was 80.3% in this age group, Pfizer said. The analysis was based on 10 symptomatic cases of COVID-19. The trial’s protocol specifies a formal analysis will be performed when at least 21 cases have accrued from 7 days after the third dose. The company said it would share final data on the effectiveness of the vaccine once the results are available.

Moderna on April 28 issued a statement with details about testing of its vaccine in young children. Vaccine efficacy was estimated at about 51% for children aged 6 months to younger than 2 years and 37% for the children aged 2 years to younger than 6. Paul Burton, MD, Moderna’s chief medical officer, spoke about this rate during a May 1 appearance on CBS’ Face the Nation.

“What it means for parents, for caregivers, is that if they give the Moderna vaccine to these little kids, they would basically cut in half the risk of that child getting symptomatic COVID,” Dr. Burton said in the interview. “Now, the number, 50%, I know is often lower than we are used to seeing with our vaccine, but it’s because this study was conducted during a time of Omicron.”

The FDA’s vaccine advisory committee also will meet on June 14 discuss potential use under an EUA of Moderna’s COVID vaccine for children and teenagers aged 6-17 years. The Pfizer-BioNTech vaccine already is authorized under an EUA for people aged 5 years and older.

The FDA has to date granted both conditional clearances, or EUAs, and regular approvals for COVID vaccines.

EUAs are meant to be temporary, allowing for rapid introduction of medicines in response to public health crises such as the pandemic. The FDA also uses EUAs to provide initial clearances of additional indications for products, as would be the case with the authorizations Moderna and Pfizer-BioNTech are seeking for their COVID vaccines.

Companies that want to continue to sell EUA-cleared products or promote EUA-cleared indications beyond the time of the public health crisis must seek regular approvals.

The FDA cleared the Pfizer-BioNTech and Moderna COVID vaccines under EUAs in December 2020. The agency then granted a regular approval for the Pfizer-BioNTech vaccine for people ages 16 and older in August 2021 based on more robust data. Regular approval for the Moderna vaccine for people ages 18 and older followed in January 2022.
 

Varied reactions among parents

Attitudes in the United States about pediatric COVID vaccines are far from uniform.

The initial uptake has disappointed physicians and researchers, who have been urging wider use of the COVID vaccination among children and teens for whom the FDA already has granted a clearance. Many parents are hesitating to bring their children for the COVID vaccines, according to the Centers for Disease Control and Prevention. Only 35.4% of children ages 5-11 had received at least one dose of a COVID vaccine, CDC staff said during a meeting.

Yet many other parents are demanding this medicine for their young children, urging the FDA to move quickly to clear COVID shots.

A private Facebook group called “Protect Their Future: A Call to Action for COVID Vaccines in Kids <5” boasts about 6,200 members. Many parents and physicians have used Twitter in recent months to press for a speedy review of COVID vaccines for the youngest children, often using the hashtag #immunizeunder5s. A group called Protect Their Future, which uses @ImmunizeUnder5s as its Twitter handle, had 5,288 followers as of the afternoon of May 23.

A special panel of the House of Representatives, the Select Subcommittee on the Coronavirus Crisis, on May 23 joined those tweeting about the need to soon authorize COVID vaccines for very young children.

“Parents have been waiting many months for vaccines for their young children,” the subcommittee tweeted. “They deserve to hear from @US_FDA why this lengthy process has been in children’s best interests.”

A version of this article first appeared on Medscape.com.

The Food and Drug Administration has approved tapinarof cream, 1%, a steroid-free topical cream applied once a day, for the treatment of mild, moderate, or severe plaque psoriasis in adults, the manufacturer announced.

Tapinarof is an aryl hydrocarbon receptor agonist and is the first FDA-approved steroid-free topical medication in this class, according to a press release from the manufacturer, Dermavant.

Approval was based on results of three studies in a phase 3 clinical trial program (PSOARING 1, PSOARING 2), and an open-label extension study, (PSOARING 3), the company release said. In PSOARING 1 and 2, approximately 1,000 adults aged 18-75 years (median age, 51 years) with plaque psoriasis were randomized to once-daily topical tapinarof or placebo for up to 12 weeks; 85% were White and 57% were men. The study findings were published in the New England Journal of Medicine in December 2021.

The primary endpoint for both trials was the proportion of patients who achieved Physician Global Assessment (PGA) scores score of “clear” (0) or “almost clear” (1) and improvement of at least two grades from baseline.

After 12 weeks, 36% of the patients in PSOARING 1 and 40% in PSOARING 2 who received tapinarof met the primary outcome, compared with 6% of patients on placebo (P < .001 for both studies). Of these, a total of 73 patients from both studies who achieved PGA scores of 0 were entered in PSOARING 3, a 40-week open-label extension study, in which they stopped tapinarof treatment and retained PGA scores of 0 or 1 for approximately 4 months off treatment. An additional 312 patients who were enrolled in the PSOARING 3 extension study achieved PGA scores of 0 at least once during the study period, with “remittive” effects lasting a mean of 130 days off of treatment.

In addition, patients who received tapinarof in the PSOARING 1 and 2 studies showed significant improvement from baseline, compared with patients on placebo, across a range of secondary endpoints including a 75% or greater improvement in Psoriasis Area and Severity Index score (PASI 75).

In PSOARING 1, and 2, respectively, 36.1% and 47.6% of those on tapinarof achieved a PASI 75 response at week 12, compared with 10.2% and 6.9% of those on the vehicle (P < .001 for both).

Across all three studies, the majority adverse events were mild to moderate, and limited to the application site.

The most common adverse events reported by patients in the tapinarof groups were folliculitis, nasopharyngitis, and contact dermatitis. Headaches were more common among those treated with tapinarof than those on vehicle in the studies (3.8% vs. 2.4% in PSOARING 1, and 3.8% vs. 0.6% in PSOARING 2), leading to only three treatment discontinuations.

At the end of the PSOARING 3 study (at either week 40 or early termination), 599 participants responded to satisfaction questionnaires. Of these, 83.6% said they were satisfied with the results of tapinarof treatment, and 81.7% said it was more effective than previous topical treatments they had used, according to the company’s release.

Tapinarof cream can be used on all areas of the body, including the face, skin folds, neck, genitalia, anal crux, inflammatory areas, and axillae, according to the company release.

Full prescribing information is available here.

The Food and Drug Administration has approved tapinarof cream, 1%, a steroid-free topical cream applied once a day, for the treatment of mild, moderate, or severe plaque psoriasis in adults, the manufacturer announced.

Tapinarof is an aryl hydrocarbon receptor agonist and is the first FDA-approved steroid-free topical medication in this class, according to a press release from the manufacturer, Dermavant.

Approval was based on results of three studies in a phase 3 clinical trial program (PSOARING 1, PSOARING 2), and an open-label extension study, (PSOARING 3), the company release said. In PSOARING 1 and 2, approximately 1,000 adults aged 18-75 years (median age, 51 years) with plaque psoriasis were randomized to once-daily topical tapinarof or placebo for up to 12 weeks; 85% were White and 57% were men. The study findings were published in the New England Journal of Medicine in December 2021.

The primary endpoint for both trials was the proportion of patients who achieved Physician Global Assessment (PGA) scores score of “clear” (0) or “almost clear” (1) and improvement of at least two grades from baseline.

After 12 weeks, 36% of the patients in PSOARING 1 and 40% in PSOARING 2 who received tapinarof met the primary outcome, compared with 6% of patients on placebo (P < .001 for both studies). Of these, a total of 73 patients from both studies who achieved PGA scores of 0 were entered in PSOARING 3, a 40-week open-label extension study, in which they stopped tapinarof treatment and retained PGA scores of 0 or 1 for approximately 4 months off treatment. An additional 312 patients who were enrolled in the PSOARING 3 extension study achieved PGA scores of 0 at least once during the study period, with “remittive” effects lasting a mean of 130 days off of treatment.

In addition, patients who received tapinarof in the PSOARING 1 and 2 studies showed significant improvement from baseline, compared with patients on placebo, across a range of secondary endpoints including a 75% or greater improvement in Psoriasis Area and Severity Index score (PASI 75).

In PSOARING 1, and 2, respectively, 36.1% and 47.6% of those on tapinarof achieved a PASI 75 response at week 12, compared with 10.2% and 6.9% of those on the vehicle (P < .001 for both).

Across all three studies, the majority adverse events were mild to moderate, and limited to the application site.

The most common adverse events reported by patients in the tapinarof groups were folliculitis, nasopharyngitis, and contact dermatitis. Headaches were more common among those treated with tapinarof than those on vehicle in the studies (3.8% vs. 2.4% in PSOARING 1, and 3.8% vs. 0.6% in PSOARING 2), leading to only three treatment discontinuations.

At the end of the PSOARING 3 study (at either week 40 or early termination), 599 participants responded to satisfaction questionnaires. Of these, 83.6% said they were satisfied with the results of tapinarof treatment, and 81.7% said it was more effective than previous topical treatments they had used, according to the company’s release.

Tapinarof cream can be used on all areas of the body, including the face, skin folds, neck, genitalia, anal crux, inflammatory areas, and axillae, according to the company release.

Full prescribing information is available here.

The Food and Drug Administration has approved tapinarof cream, 1%, a steroid-free topical cream applied once a day, for the treatment of mild, moderate, or severe plaque psoriasis in adults, the manufacturer announced.

Tapinarof is an aryl hydrocarbon receptor agonist and is the first FDA-approved steroid-free topical medication in this class, according to a press release from the manufacturer, Dermavant.

Approval was based on results of three studies in a phase 3 clinical trial program (PSOARING 1, PSOARING 2), and an open-label extension study, (PSOARING 3), the company release said. In PSOARING 1 and 2, approximately 1,000 adults aged 18-75 years (median age, 51 years) with plaque psoriasis were randomized to once-daily topical tapinarof or placebo for up to 12 weeks; 85% were White and 57% were men. The study findings were published in the New England Journal of Medicine in December 2021.

The primary endpoint for both trials was the proportion of patients who achieved Physician Global Assessment (PGA) scores score of “clear” (0) or “almost clear” (1) and improvement of at least two grades from baseline.

After 12 weeks, 36% of the patients in PSOARING 1 and 40% in PSOARING 2 who received tapinarof met the primary outcome, compared with 6% of patients on placebo (P < .001 for both studies). Of these, a total of 73 patients from both studies who achieved PGA scores of 0 were entered in PSOARING 3, a 40-week open-label extension study, in which they stopped tapinarof treatment and retained PGA scores of 0 or 1 for approximately 4 months off treatment. An additional 312 patients who were enrolled in the PSOARING 3 extension study achieved PGA scores of 0 at least once during the study period, with “remittive” effects lasting a mean of 130 days off of treatment.

In addition, patients who received tapinarof in the PSOARING 1 and 2 studies showed significant improvement from baseline, compared with patients on placebo, across a range of secondary endpoints including a 75% or greater improvement in Psoriasis Area and Severity Index score (PASI 75).

In PSOARING 1, and 2, respectively, 36.1% and 47.6% of those on tapinarof achieved a PASI 75 response at week 12, compared with 10.2% and 6.9% of those on the vehicle (P < .001 for both).

Across all three studies, the majority adverse events were mild to moderate, and limited to the application site.

The most common adverse events reported by patients in the tapinarof groups were folliculitis, nasopharyngitis, and contact dermatitis. Headaches were more common among those treated with tapinarof than those on vehicle in the studies (3.8% vs. 2.4% in PSOARING 1, and 3.8% vs. 0.6% in PSOARING 2), leading to only three treatment discontinuations.

At the end of the PSOARING 3 study (at either week 40 or early termination), 599 participants responded to satisfaction questionnaires. Of these, 83.6% said they were satisfied with the results of tapinarof treatment, and 81.7% said it was more effective than previous topical treatments they had used, according to the company’s release.

Tapinarof cream can be used on all areas of the body, including the face, skin folds, neck, genitalia, anal crux, inflammatory areas, and axillae, according to the company release.

Full prescribing information is available here.

The U.S. Food and Drug Administration has approved dupilumab (Dupixent, Regeneron) to treat eosinophilic esophagitis (EoE) in adults and children aged 12 years and older weighing at least 40 kg.

EoE is a chronic inflammatory disorder driven by type 2 inflammation that damages the esophagus and causes difficulty swallowing and eating.

Dupilumab is a monoclonal antibody that acts to inhibit part of the inflammatory pathway. It’s the first drug to be approved by the FDA for EoE.

In a phase 3 trial, dupilumab 300 mg weekly significantly improved signs and symptoms of eosinophilic esophagitis, compared with placebo, underscoring the role of type 2 inflammation in this disease, Regeneron says in a news release.

According to the company, there are roughly 160,000 patients in the United States living with EoE who are currently using treatments not specifically approved for the disease. Of those patients, about 48,000 continue to experience symptoms despite multiple treatments.

“As researchers and clinicians have gained knowledge about eosinophilic esophagitis in recent years, more cases of the disorder have been recognized and diagnosed in the U.S.,” Jessica Lee, MD, director of the Division of Gastroenterology in the FDA’s Center for Drug Evaluation and Research, said in an FDA news release.

The approval of dupilumab will “fulfill an important unmet need for the increasing number of patients with eosinophilic esophagitis,” Dr. Lee said.

The efficacy and safety of dupilumab in EoE was demonstrated in a randomized, double-blind, parallel-group, multicenter, placebo-controlled trial that included two 24-week treatment periods (parts A and B) that were conducted independently in separate groups of patients.

In both part A and B, patients received dupilumab 300 mg or placebo every week.

In part A of the trial, 60% of the 42 patients who received dupilumab achieved the predetermined level of reduction of eosinophils in the esophagus, compared with 5% of the 39 patients who received placebo, the FDA said.

Patients who received dupilumab also experienced an average improvement of 22 points in the Dysphagia Symptom Questionnaire (DSQ) score, compared with 10 points for patients who received placebo.

In part B, 59% of the 80 patients who received dupilumab achieved the predetermined level of reduction of eosinophils in the esophagus, compared with 6% of the 79 patients who received placebo.

Patients who received dupilumab also experienced an average improvement of 24 points in their DSQ score, compared with 14 points for patients who received placebo.

“Assessments incorporating the perspectives from patients with EoE supported that the DSQ score improvement in patients who received Dupixent in the clinical trial was representative of clinically meaningful improvement in dysphagia,” the FDA noted.

“Treatment for patients with eosinophilic esophagitis can be challenging, particularly with no previously approved medications,” Evan Dellon, MD, principal investigator for the phase 3 trial, said in the company news release.

“Now, patients and their doctors have a treatment option available as part of their management plan that has the potential to control symptoms, improve inflammation, and heal the changes in the esophagus caused by this progressive and burdensome disease,” added Dr. Dellon, who is professor of medicine in the division of gastroenterology and hepatology at the University of North Carolina at Chapel Hill.

The FDA granted dupilumab priority review and breakthrough therapy designations for EoE.

Dupilumab is already approved in the United States for treatment of moderate to severe atopic dermatitis in adults and children aged 6 years and older whose disease is not adequately controlled by topical prescription therapies or for whom those therapies are not advisable.

The drug is also approved as an add-on maintenance treatment for adults and children aged 6 years and older with certain types of moderate to severe asthma and as an add-on maintenance treatment for adults with inadequately controlled chronic rhinosinusitis with nasal polyposis.

A version of this article first appeared on Medscape.com .

The U.S. Food and Drug Administration has approved dupilumab (Dupixent, Regeneron) to treat eosinophilic esophagitis (EoE) in adults and children aged 12 years and older weighing at least 40 kg.

EoE is a chronic inflammatory disorder driven by type 2 inflammation that damages the esophagus and causes difficulty swallowing and eating.

Dupilumab is a monoclonal antibody that acts to inhibit part of the inflammatory pathway. It’s the first drug to be approved by the FDA for EoE.

In a phase 3 trial, dupilumab 300 mg weekly significantly improved signs and symptoms of eosinophilic esophagitis, compared with placebo, underscoring the role of type 2 inflammation in this disease, Regeneron says in a news release.

According to the company, there are roughly 160,000 patients in the United States living with EoE who are currently using treatments not specifically approved for the disease. Of those patients, about 48,000 continue to experience symptoms despite multiple treatments.

“As researchers and clinicians have gained knowledge about eosinophilic esophagitis in recent years, more cases of the disorder have been recognized and diagnosed in the U.S.,” Jessica Lee, MD, director of the Division of Gastroenterology in the FDA’s Center for Drug Evaluation and Research, said in an FDA news release.

The approval of dupilumab will “fulfill an important unmet need for the increasing number of patients with eosinophilic esophagitis,” Dr. Lee said.

The efficacy and safety of dupilumab in EoE was demonstrated in a randomized, double-blind, parallel-group, multicenter, placebo-controlled trial that included two 24-week treatment periods (parts A and B) that were conducted independently in separate groups of patients.

In both part A and B, patients received dupilumab 300 mg or placebo every week.

In part A of the trial, 60% of the 42 patients who received dupilumab achieved the predetermined level of reduction of eosinophils in the esophagus, compared with 5% of the 39 patients who received placebo, the FDA said.

Patients who received dupilumab also experienced an average improvement of 22 points in the Dysphagia Symptom Questionnaire (DSQ) score, compared with 10 points for patients who received placebo.

In part B, 59% of the 80 patients who received dupilumab achieved the predetermined level of reduction of eosinophils in the esophagus, compared with 6% of the 79 patients who received placebo.

Patients who received dupilumab also experienced an average improvement of 24 points in their DSQ score, compared with 14 points for patients who received placebo.

“Assessments incorporating the perspectives from patients with EoE supported that the DSQ score improvement in patients who received Dupixent in the clinical trial was representative of clinically meaningful improvement in dysphagia,” the FDA noted.

“Treatment for patients with eosinophilic esophagitis can be challenging, particularly with no previously approved medications,” Evan Dellon, MD, principal investigator for the phase 3 trial, said in the company news release.

“Now, patients and their doctors have a treatment option available as part of their management plan that has the potential to control symptoms, improve inflammation, and heal the changes in the esophagus caused by this progressive and burdensome disease,” added Dr. Dellon, who is professor of medicine in the division of gastroenterology and hepatology at the University of North Carolina at Chapel Hill.

The FDA granted dupilumab priority review and breakthrough therapy designations for EoE.

Dupilumab is already approved in the United States for treatment of moderate to severe atopic dermatitis in adults and children aged 6 years and older whose disease is not adequately controlled by topical prescription therapies or for whom those therapies are not advisable.

The drug is also approved as an add-on maintenance treatment for adults and children aged 6 years and older with certain types of moderate to severe asthma and as an add-on maintenance treatment for adults with inadequately controlled chronic rhinosinusitis with nasal polyposis.

A version of this article first appeared on Medscape.com .

The U.S. Food and Drug Administration has approved dupilumab (Dupixent, Regeneron) to treat eosinophilic esophagitis (EoE) in adults and children aged 12 years and older weighing at least 40 kg.

EoE is a chronic inflammatory disorder driven by type 2 inflammation that damages the esophagus and causes difficulty swallowing and eating.

Dupilumab is a monoclonal antibody that acts to inhibit part of the inflammatory pathway. It’s the first drug to be approved by the FDA for EoE.

In a phase 3 trial, dupilumab 300 mg weekly significantly improved signs and symptoms of eosinophilic esophagitis, compared with placebo, underscoring the role of type 2 inflammation in this disease, Regeneron says in a news release.

According to the company, there are roughly 160,000 patients in the United States living with EoE who are currently using treatments not specifically approved for the disease. Of those patients, about 48,000 continue to experience symptoms despite multiple treatments.

“As researchers and clinicians have gained knowledge about eosinophilic esophagitis in recent years, more cases of the disorder have been recognized and diagnosed in the U.S.,” Jessica Lee, MD, director of the Division of Gastroenterology in the FDA’s Center for Drug Evaluation and Research, said in an FDA news release.

The approval of dupilumab will “fulfill an important unmet need for the increasing number of patients with eosinophilic esophagitis,” Dr. Lee said.

The efficacy and safety of dupilumab in EoE was demonstrated in a randomized, double-blind, parallel-group, multicenter, placebo-controlled trial that included two 24-week treatment periods (parts A and B) that were conducted independently in separate groups of patients.

In both part A and B, patients received dupilumab 300 mg or placebo every week.

In part A of the trial, 60% of the 42 patients who received dupilumab achieved the predetermined level of reduction of eosinophils in the esophagus, compared with 5% of the 39 patients who received placebo, the FDA said.

Patients who received dupilumab also experienced an average improvement of 22 points in the Dysphagia Symptom Questionnaire (DSQ) score, compared with 10 points for patients who received placebo.

In part B, 59% of the 80 patients who received dupilumab achieved the predetermined level of reduction of eosinophils in the esophagus, compared with 6% of the 79 patients who received placebo.

Patients who received dupilumab also experienced an average improvement of 24 points in their DSQ score, compared with 14 points for patients who received placebo.

“Assessments incorporating the perspectives from patients with EoE supported that the DSQ score improvement in patients who received Dupixent in the clinical trial was representative of clinically meaningful improvement in dysphagia,” the FDA noted.

“Treatment for patients with eosinophilic esophagitis can be challenging, particularly with no previously approved medications,” Evan Dellon, MD, principal investigator for the phase 3 trial, said in the company news release.

“Now, patients and their doctors have a treatment option available as part of their management plan that has the potential to control symptoms, improve inflammation, and heal the changes in the esophagus caused by this progressive and burdensome disease,” added Dr. Dellon, who is professor of medicine in the division of gastroenterology and hepatology at the University of North Carolina at Chapel Hill.

The FDA granted dupilumab priority review and breakthrough therapy designations for EoE.

Dupilumab is already approved in the United States for treatment of moderate to severe atopic dermatitis in adults and children aged 6 years and older whose disease is not adequately controlled by topical prescription therapies or for whom those therapies are not advisable.

The drug is also approved as an add-on maintenance treatment for adults and children aged 6 years and older with certain types of moderate to severe asthma and as an add-on maintenance treatment for adults with inadequately controlled chronic rhinosinusitis with nasal polyposis.

A version of this article first appeared on Medscape.com .

Each year, there are about 59,000 deaths from rabies globally. Most of these occur outside the United States and are the result of dog bites. Since infection with rabies is almost always fatal, there has been considerable attention given to vaccinating people at high risk before likely exposure and responding immediately to those bitten by a rabid animal.

The Centers for Disease Control and Prevention recently revised its preexposure prophylaxis (PrEP) recommendations for rabies. Under the previous 2008 guidelines, PrEP injections were given on days 0, 7, and 21 and cost more than $1,100. In trying to simplify recommendations and make immunization less expensive, the agency designated five risk levels with different advice based on the level of risk.

The first two groups are those with very high risk of occupational exposures – either working with rabies virus in the laboratory or working with or having contact with bats or performing animal necropsies. They are now advised to get two doses of rabies vaccine on days 0 and 7. The lab workers should have titers checked every 6 months to ensure that they remain adequately protected. And a booster should be given if the titer drops to < 0.5 IU/mL. The second group, with bat exposures, should have titers checked every 2 years.

Risk category 3 is those with long-term (> 3 years) exposure to mammals other than bats that might be rabid. This group would include veterinarians, wildlife biologists, animal control officers, and spelunkers (cavers). Category 3 also includes travelers who may encounter rabid dogs, which is not a risk in the United States. They would get the same initial two doses. The new recommendations for a third dose are based either on a titer drawn 1-3 years later being < 0.5 IU/mL or choosing to give a booster between 3 weeks and 3 years after the second dose.

The same groups are covered in risk group 4, but these are expected to have less than 3 years of potential exposure after PrEP. They would receive two doses on days 0 and 7.

Finally, group 5, at the lowest risk, includes most of the U.S. population. They do not require any PrEP.

Agam Rao, MD, CAPT, U.S. Public Health Service, CDC, told this news organization that the CDC’s Advisory Committee on Immunization Practices (ACIP) has been working on updating the 2008 rabies PrEP recommendations for several years. The committee wanted the new guideline to be “as easily followable as possible but also based on the evidence itself.”

There were two significant problems the committee tried to address. “One was that travelers who book their travel on kind of short notice don’t have enough time to get that third dose, which at the earliest can be given on day 21,” Dr. Rao said.

The second problem is that “a three-dose series [is] just really expensive. And what we found from data that had been published since the last ACIP recommendations is that fewer people than we recommend get vaccinated were getting vaccinated. So hopefully, the two-dose series helps with that.”

The ACIP used an adapted Grading of Recommendations Assessment, Development, and Evaluation (GRADE) approach to determine the certainty of the evidence for immunogenicity. The ACIP also used an evidence to recommendations (EtR) framework. “This incorporates a lot of other factors like the acceptability, usability, equity, all of these other variables that are important to the evidence being translated into recommendations,” Dr. Rao said. A table details their analysis.

Rabies expert Thiravat Hemachudha, MD, professor of neurology at WHO Collaborating Centre for Research and Training on Viral Zoonoses, Chulalongkorn University Hospital, Bangkok, told this news organization via email that “the ACIP relies mostly on serology, whereas the rest of the world cannot afford the test or testing may not be available.”

He added: “The issue of ‘long-term immunogenicity’ after receiving [PrEP is] an anamnestic response. All standard tissue culture rabies vaccines with appropriate dosage and route of delivery, either IM or ID, are considered safe and effective. There are many studies in Asian countries confirming that with only one primary series of PrEP, ID or IM with reduced doses, can produce immunity for as long as 20 years. Therefore, serology check is not necessary in general populations in rabies endemic countries where most of the rabies deaths occur. Investigation of all death cases was performed in Thailand and did not reveal any failure. Cases with PrEP in the past who died did not receive a booster after exposure.”

Dr. Rao offered one additional suggestion to clinicians faced with an urgent need to get a rabies titer: “They really should reach out to the lab (with all the information) before they send the specimen for the titer check ... so that the testing can be facilitated. All of these laboratories have the capacity to do stat and ASAP testing ... Clinicians do not know that they can call laboratories directly and expedite this sort of testing.” 

Dr. Rao emphasized that PrEP does not eliminate the need for postexposure prophylaxis (PEP). Still, it eliminates the need for rabies immunoglobulin and decreases the number of vaccine doses required for PEP. “I hope more people will take advantage of the titer checks and potentially save the patient some money,” she concluded.

Dr. Rao and Dr. Hemachudha have disclosed no relevant financial relationships.

A version of this article first appeared on Medscape.com.

Each year, there are about 59,000 deaths from rabies globally. Most of these occur outside the United States and are the result of dog bites. Since infection with rabies is almost always fatal, there has been considerable attention given to vaccinating people at high risk before likely exposure and responding immediately to those bitten by a rabid animal.

The Centers for Disease Control and Prevention recently revised its preexposure prophylaxis (PrEP) recommendations for rabies. Under the previous 2008 guidelines, PrEP injections were given on days 0, 7, and 21 and cost more than $1,100. In trying to simplify recommendations and make immunization less expensive, the agency designated five risk levels with different advice based on the level of risk.

The first two groups are those with very high risk of occupational exposures – either working with rabies virus in the laboratory or working with or having contact with bats or performing animal necropsies. They are now advised to get two doses of rabies vaccine on days 0 and 7. The lab workers should have titers checked every 6 months to ensure that they remain adequately protected. And a booster should be given if the titer drops to < 0.5 IU/mL. The second group, with bat exposures, should have titers checked every 2 years.

Risk category 3 is those with long-term (> 3 years) exposure to mammals other than bats that might be rabid. This group would include veterinarians, wildlife biologists, animal control officers, and spelunkers (cavers). Category 3 also includes travelers who may encounter rabid dogs, which is not a risk in the United States. They would get the same initial two doses. The new recommendations for a third dose are based either on a titer drawn 1-3 years later being < 0.5 IU/mL or choosing to give a booster between 3 weeks and 3 years after the second dose.

The same groups are covered in risk group 4, but these are expected to have less than 3 years of potential exposure after PrEP. They would receive two doses on days 0 and 7.

Finally, group 5, at the lowest risk, includes most of the U.S. population. They do not require any PrEP.

Agam Rao, MD, CAPT, U.S. Public Health Service, CDC, told this news organization that the CDC’s Advisory Committee on Immunization Practices (ACIP) has been working on updating the 2008 rabies PrEP recommendations for several years. The committee wanted the new guideline to be “as easily followable as possible but also based on the evidence itself.”

There were two significant problems the committee tried to address. “One was that travelers who book their travel on kind of short notice don’t have enough time to get that third dose, which at the earliest can be given on day 21,” Dr. Rao said.

The second problem is that “a three-dose series [is] just really expensive. And what we found from data that had been published since the last ACIP recommendations is that fewer people than we recommend get vaccinated were getting vaccinated. So hopefully, the two-dose series helps with that.”

The ACIP used an adapted Grading of Recommendations Assessment, Development, and Evaluation (GRADE) approach to determine the certainty of the evidence for immunogenicity. The ACIP also used an evidence to recommendations (EtR) framework. “This incorporates a lot of other factors like the acceptability, usability, equity, all of these other variables that are important to the evidence being translated into recommendations,” Dr. Rao said. A table details their analysis.

Rabies expert Thiravat Hemachudha, MD, professor of neurology at WHO Collaborating Centre for Research and Training on Viral Zoonoses, Chulalongkorn University Hospital, Bangkok, told this news organization via email that “the ACIP relies mostly on serology, whereas the rest of the world cannot afford the test or testing may not be available.”

He added: “The issue of ‘long-term immunogenicity’ after receiving [PrEP is] an anamnestic response. All standard tissue culture rabies vaccines with appropriate dosage and route of delivery, either IM or ID, are considered safe and effective. There are many studies in Asian countries confirming that with only one primary series of PrEP, ID or IM with reduced doses, can produce immunity for as long as 20 years. Therefore, serology check is not necessary in general populations in rabies endemic countries where most of the rabies deaths occur. Investigation of all death cases was performed in Thailand and did not reveal any failure. Cases with PrEP in the past who died did not receive a booster after exposure.”

Dr. Rao offered one additional suggestion to clinicians faced with an urgent need to get a rabies titer: “They really should reach out to the lab (with all the information) before they send the specimen for the titer check ... so that the testing can be facilitated. All of these laboratories have the capacity to do stat and ASAP testing ... Clinicians do not know that they can call laboratories directly and expedite this sort of testing.” 

Dr. Rao emphasized that PrEP does not eliminate the need for postexposure prophylaxis (PEP). Still, it eliminates the need for rabies immunoglobulin and decreases the number of vaccine doses required for PEP. “I hope more people will take advantage of the titer checks and potentially save the patient some money,” she concluded.

Dr. Rao and Dr. Hemachudha have disclosed no relevant financial relationships.

A version of this article first appeared on Medscape.com.

Each year, there are about 59,000 deaths from rabies globally. Most of these occur outside the United States and are the result of dog bites. Since infection with rabies is almost always fatal, there has been considerable attention given to vaccinating people at high risk before likely exposure and responding immediately to those bitten by a rabid animal.

The Centers for Disease Control and Prevention recently revised its preexposure prophylaxis (PrEP) recommendations for rabies. Under the previous 2008 guidelines, PrEP injections were given on days 0, 7, and 21 and cost more than $1,100. In trying to simplify recommendations and make immunization less expensive, the agency designated five risk levels with different advice based on the level of risk.

The first two groups are those with very high risk of occupational exposures – either working with rabies virus in the laboratory or working with or having contact with bats or performing animal necropsies. They are now advised to get two doses of rabies vaccine on days 0 and 7. The lab workers should have titers checked every 6 months to ensure that they remain adequately protected. And a booster should be given if the titer drops to < 0.5 IU/mL. The second group, with bat exposures, should have titers checked every 2 years.

Risk category 3 is those with long-term (> 3 years) exposure to mammals other than bats that might be rabid. This group would include veterinarians, wildlife biologists, animal control officers, and spelunkers (cavers). Category 3 also includes travelers who may encounter rabid dogs, which is not a risk in the United States. They would get the same initial two doses. The new recommendations for a third dose are based either on a titer drawn 1-3 years later being < 0.5 IU/mL or choosing to give a booster between 3 weeks and 3 years after the second dose.

The same groups are covered in risk group 4, but these are expected to have less than 3 years of potential exposure after PrEP. They would receive two doses on days 0 and 7.

Finally, group 5, at the lowest risk, includes most of the U.S. population. They do not require any PrEP.

Agam Rao, MD, CAPT, U.S. Public Health Service, CDC, told this news organization that the CDC’s Advisory Committee on Immunization Practices (ACIP) has been working on updating the 2008 rabies PrEP recommendations for several years. The committee wanted the new guideline to be “as easily followable as possible but also based on the evidence itself.”

There were two significant problems the committee tried to address. “One was that travelers who book their travel on kind of short notice don’t have enough time to get that third dose, which at the earliest can be given on day 21,” Dr. Rao said.

The second problem is that “a three-dose series [is] just really expensive. And what we found from data that had been published since the last ACIP recommendations is that fewer people than we recommend get vaccinated were getting vaccinated. So hopefully, the two-dose series helps with that.”

The ACIP used an adapted Grading of Recommendations Assessment, Development, and Evaluation (GRADE) approach to determine the certainty of the evidence for immunogenicity. The ACIP also used an evidence to recommendations (EtR) framework. “This incorporates a lot of other factors like the acceptability, usability, equity, all of these other variables that are important to the evidence being translated into recommendations,” Dr. Rao said. A table details their analysis.

Rabies expert Thiravat Hemachudha, MD, professor of neurology at WHO Collaborating Centre for Research and Training on Viral Zoonoses, Chulalongkorn University Hospital, Bangkok, told this news organization via email that “the ACIP relies mostly on serology, whereas the rest of the world cannot afford the test or testing may not be available.”

He added: “The issue of ‘long-term immunogenicity’ after receiving [PrEP is] an anamnestic response. All standard tissue culture rabies vaccines with appropriate dosage and route of delivery, either IM or ID, are considered safe and effective. There are many studies in Asian countries confirming that with only one primary series of PrEP, ID or IM with reduced doses, can produce immunity for as long as 20 years. Therefore, serology check is not necessary in general populations in rabies endemic countries where most of the rabies deaths occur. Investigation of all death cases was performed in Thailand and did not reveal any failure. Cases with PrEP in the past who died did not receive a booster after exposure.”

Dr. Rao offered one additional suggestion to clinicians faced with an urgent need to get a rabies titer: “They really should reach out to the lab (with all the information) before they send the specimen for the titer check ... so that the testing can be facilitated. All of these laboratories have the capacity to do stat and ASAP testing ... Clinicians do not know that they can call laboratories directly and expedite this sort of testing.” 

Dr. Rao emphasized that PrEP does not eliminate the need for postexposure prophylaxis (PEP). Still, it eliminates the need for rabies immunoglobulin and decreases the number of vaccine doses required for PEP. “I hope more people will take advantage of the titer checks and potentially save the patient some money,” she concluded.

Dr. Rao and Dr. Hemachudha have disclosed no relevant financial relationships.

A version of this article first appeared on Medscape.com.