FDA/CDC

Two diabetes management devices that aid in the precision of insulin delivery have been recently cleared by the Food and Drug Administration.

On March 2, the FDA cleared the Android version of Bigfoot Biomedical’s Unity Mobile App for use with its system of smart pen caps that are compatible with different disposable insulin pens for administering both long-acting and rapid-acting insulin.

The system, which has been compatible with iOS devices since May 2021, is “the first and only FDA-cleared smart injection system that turns CGM [continuous glucose monitoring] data into dosing recommendations displayed right on the pen cap for people using multiple daily [insulin] injection therapy,” according to a company statement.

The Bigfoot app allows users to input and review provider treatment recommendations, displays current glucose ranges, and delivers real-time alerts.

Once it is commercially launched, the Android phone application will be available via the Google Play Store. “Given that 41% of U.S. smartphone users choose Android devices, this clearance enables expanded access to a large group of people with diabetes,” the company said.

On March 6, the FDA cleared the Abbott FreeStyle Libre 2 and FreeStyle Libre 3 devices as “integrated” CGM sensors. This means that they can now be used as components in automated insulin delivery systems, along with insulin pumps and connectivity software.

Abbott is working with insulin pump manufacturers Insulet and Tandem in the United States for integration with the FreeStyle Libre versions 2 and 3. Outside the United States, the Libre 3 is already authorized to work with mylife Loop from Ypsomed and CamDiab in Germany. Further launches are expected in the United Kingdom, Switzerland, and the Netherlands later this year.

The modified FreeStyle Libre 2 and FreeStyle Libre 3 sensors have been cleared for use by patients as young as age 2 years and for up to 15 days, in contrast to the previous versions, which were available for patients as young as 4 years for use up to 14 days. The FDA has cleared all Libre sensors – 2 and 3, current and future versions – for use by pregnant women with any type of diabetes.

The modified sensors will be available in the United States later this year and will eventually replace the Libre sensors in current use, the company said in a statement.

“The FreeStyle Libre portfolio is still the most affordable CGM on the market,” an Abbott representative said in an interview.

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

Two diabetes management devices that aid in the precision of insulin delivery have been recently cleared by the Food and Drug Administration.

On March 2, the FDA cleared the Android version of Bigfoot Biomedical’s Unity Mobile App for use with its system of smart pen caps that are compatible with different disposable insulin pens for administering both long-acting and rapid-acting insulin.

The system, which has been compatible with iOS devices since May 2021, is “the first and only FDA-cleared smart injection system that turns CGM [continuous glucose monitoring] data into dosing recommendations displayed right on the pen cap for people using multiple daily [insulin] injection therapy,” according to a company statement.

The Bigfoot app allows users to input and review provider treatment recommendations, displays current glucose ranges, and delivers real-time alerts.

Once it is commercially launched, the Android phone application will be available via the Google Play Store. “Given that 41% of U.S. smartphone users choose Android devices, this clearance enables expanded access to a large group of people with diabetes,” the company said.

On March 6, the FDA cleared the Abbott FreeStyle Libre 2 and FreeStyle Libre 3 devices as “integrated” CGM sensors. This means that they can now be used as components in automated insulin delivery systems, along with insulin pumps and connectivity software.

Abbott is working with insulin pump manufacturers Insulet and Tandem in the United States for integration with the FreeStyle Libre versions 2 and 3. Outside the United States, the Libre 3 is already authorized to work with mylife Loop from Ypsomed and CamDiab in Germany. Further launches are expected in the United Kingdom, Switzerland, and the Netherlands later this year.

The modified FreeStyle Libre 2 and FreeStyle Libre 3 sensors have been cleared for use by patients as young as age 2 years and for up to 15 days, in contrast to the previous versions, which were available for patients as young as 4 years for use up to 14 days. The FDA has cleared all Libre sensors – 2 and 3, current and future versions – for use by pregnant women with any type of diabetes.

The modified sensors will be available in the United States later this year and will eventually replace the Libre sensors in current use, the company said in a statement.

“The FreeStyle Libre portfolio is still the most affordable CGM on the market,” an Abbott representative said in an interview.

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

Two diabetes management devices that aid in the precision of insulin delivery have been recently cleared by the Food and Drug Administration.

On March 2, the FDA cleared the Android version of Bigfoot Biomedical’s Unity Mobile App for use with its system of smart pen caps that are compatible with different disposable insulin pens for administering both long-acting and rapid-acting insulin.

The system, which has been compatible with iOS devices since May 2021, is “the first and only FDA-cleared smart injection system that turns CGM [continuous glucose monitoring] data into dosing recommendations displayed right on the pen cap for people using multiple daily [insulin] injection therapy,” according to a company statement.

The Bigfoot app allows users to input and review provider treatment recommendations, displays current glucose ranges, and delivers real-time alerts.

Once it is commercially launched, the Android phone application will be available via the Google Play Store. “Given that 41% of U.S. smartphone users choose Android devices, this clearance enables expanded access to a large group of people with diabetes,” the company said.

On March 6, the FDA cleared the Abbott FreeStyle Libre 2 and FreeStyle Libre 3 devices as “integrated” CGM sensors. This means that they can now be used as components in automated insulin delivery systems, along with insulin pumps and connectivity software.

Abbott is working with insulin pump manufacturers Insulet and Tandem in the United States for integration with the FreeStyle Libre versions 2 and 3. Outside the United States, the Libre 3 is already authorized to work with mylife Loop from Ypsomed and CamDiab in Germany. Further launches are expected in the United Kingdom, Switzerland, and the Netherlands later this year.

The modified FreeStyle Libre 2 and FreeStyle Libre 3 sensors have been cleared for use by patients as young as age 2 years and for up to 15 days, in contrast to the previous versions, which were available for patients as young as 4 years for use up to 14 days. The FDA has cleared all Libre sensors – 2 and 3, current and future versions – for use by pregnant women with any type of diabetes.

The modified sensors will be available in the United States later this year and will eventually replace the Libre sensors in current use, the company said in a statement.

“The FreeStyle Libre portfolio is still the most affordable CGM on the market,” an Abbott representative said in an interview.

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

The Centers for Disease Control and Prevention has issued a Health Alert Network (HAN) health advisory notifying clinicians and public health officials of a confirmed measles case in an individual who for 2 days (February 17-18) attended a large religious gathering that was attended by an estimated 20,000 people at Asbury University in Wilmore, Ky.

Given that large numbers of people might have been exposed to the attendee (who was not vaccinated) and that the individual had a history of recent international travel, the CDC has encouraged clinicians to be vigilant for patients presenting with symptoms that meet the measles case definition. A steady increase in measles cases from 49 in 2021 to 121 in 2022 in children who were not fully vaccinated – coupled with outbreaks in Ohio and Minnesota – underscores the potential gravity of the CDC advisory as well as the need to mitigate the risk of ongoing or secondary transmission.

Currently, little is known about the individual who contracted measles other than the fact that he is a resident of Jessamine County, Ky., according to a news release issued by the Kentucky Department of Public Health. It is the third confirmed case in Kentucky over the past 3 months. State and national health officials are concerned that the individual might have transmitted measles to attendees visiting from other states.

David Sugerman, MD, MPH, a medical officer in CDC’s division of viral diseases and lead for the measles, rubella, and cytomegalovirus team, noted that the timing of the alert coincides with the period in which persons who had had contact with the initial case patient might be expected to develop symptoms.

For clinicians, “It’s really about considering measles in any un- or undervaccinated patient that arrives at a clinic and recently traveled internationally,” Dr. Sugerman told this news organization. He explained that “when doctors are seeing patients, they’re not going to necessarily share that information off the bat when they present with fever or rash, or if their child has fever and rash, or that they traveled internationally. So, eliciting that history from the patient or their parents is really critical.”

The CDC recommends that measles be considered in anyone presenting with a febrile illness and symptoms that are clinically compatible with measles (that is, rash, cough, coryza, or conjunctivitis), as well as in patients who have recently traveled abroad, especially to countries with ongoing outbreaks, including India, Somalia, and Yemen.

“In general, if they’ve traveled internationally and they are undervaccinated, measles should be part of the differential diagnosis,” Sugerman said. He also emphasized the need to follow airborne isolation precautions in addition to general infection control measures.

Immediate triage is critical, especially since overcrowded waiting rooms might be filled with patients who are not yet eligible for vaccination or are not up to date or fully vaccinated.

“Measles is under airborne isolation criteria and precautions, and therefore, [patients] need to be placed as soon as possible into a negative pressure or airborne infection isolation room – and that should be a single room,” he explained. He noted, “In some settings, there may not be a negative pressure room, e.g., an outpatient pediatrics or family medicine office.”

Dr. Sugerman said that in these circumstances, patients should be placed in a room with masked health care providers who have received two doses of measles, mumps, and rubella (MMR) vaccine and that they should wear an N95 mask when entering the room and interviewing the patient.

Clinicians should follow CDC’s testing recommendations and collect a nasopharyngeal or throat swab or a urine specimen for PCR testing and a blood specimen for serology. In addition, they should immediately report cases to local and state public health authorities.

For all patients, it’s critical to be up to date on MMR vaccines, especially persons who are going to be traveling internationally. “We recommend that when they’ve got infants traveling with them who are 6-11 months of age, that they get a first dose (which we consider a zero dose), because they need a routine dose at 12-15 months, and then 4-6 years,” said Dr. Sugerman. He said that it’s safe for adults who are unsure of their status to receive an MMR dose as well.

Dr. Sugerman stressed that despite major strides, “we just don’t have enough coverage in all individuals in this country. Because people are traveling as often as they are, it can be imported. Until measles is eliminated globally, there’s going to be an ongoing risk of importation and potential spread amongst others in their household or community, especially amongst individuals who are not fully vaccinated and, in particular, amongst those who are unvaccinated,” he said.

Dr. Sugerman reports no relevant financial relationships.

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

The Centers for Disease Control and Prevention has issued a Health Alert Network (HAN) health advisory notifying clinicians and public health officials of a confirmed measles case in an individual who for 2 days (February 17-18) attended a large religious gathering that was attended by an estimated 20,000 people at Asbury University in Wilmore, Ky.

Given that large numbers of people might have been exposed to the attendee (who was not vaccinated) and that the individual had a history of recent international travel, the CDC has encouraged clinicians to be vigilant for patients presenting with symptoms that meet the measles case definition. A steady increase in measles cases from 49 in 2021 to 121 in 2022 in children who were not fully vaccinated – coupled with outbreaks in Ohio and Minnesota – underscores the potential gravity of the CDC advisory as well as the need to mitigate the risk of ongoing or secondary transmission.

Currently, little is known about the individual who contracted measles other than the fact that he is a resident of Jessamine County, Ky., according to a news release issued by the Kentucky Department of Public Health. It is the third confirmed case in Kentucky over the past 3 months. State and national health officials are concerned that the individual might have transmitted measles to attendees visiting from other states.

David Sugerman, MD, MPH, a medical officer in CDC’s division of viral diseases and lead for the measles, rubella, and cytomegalovirus team, noted that the timing of the alert coincides with the period in which persons who had had contact with the initial case patient might be expected to develop symptoms.

For clinicians, “It’s really about considering measles in any un- or undervaccinated patient that arrives at a clinic and recently traveled internationally,” Dr. Sugerman told this news organization. He explained that “when doctors are seeing patients, they’re not going to necessarily share that information off the bat when they present with fever or rash, or if their child has fever and rash, or that they traveled internationally. So, eliciting that history from the patient or their parents is really critical.”

The CDC recommends that measles be considered in anyone presenting with a febrile illness and symptoms that are clinically compatible with measles (that is, rash, cough, coryza, or conjunctivitis), as well as in patients who have recently traveled abroad, especially to countries with ongoing outbreaks, including India, Somalia, and Yemen.

“In general, if they’ve traveled internationally and they are undervaccinated, measles should be part of the differential diagnosis,” Sugerman said. He also emphasized the need to follow airborne isolation precautions in addition to general infection control measures.

Immediate triage is critical, especially since overcrowded waiting rooms might be filled with patients who are not yet eligible for vaccination or are not up to date or fully vaccinated.

“Measles is under airborne isolation criteria and precautions, and therefore, [patients] need to be placed as soon as possible into a negative pressure or airborne infection isolation room – and that should be a single room,” he explained. He noted, “In some settings, there may not be a negative pressure room, e.g., an outpatient pediatrics or family medicine office.”

Dr. Sugerman said that in these circumstances, patients should be placed in a room with masked health care providers who have received two doses of measles, mumps, and rubella (MMR) vaccine and that they should wear an N95 mask when entering the room and interviewing the patient.

Clinicians should follow CDC’s testing recommendations and collect a nasopharyngeal or throat swab or a urine specimen for PCR testing and a blood specimen for serology. In addition, they should immediately report cases to local and state public health authorities.

For all patients, it’s critical to be up to date on MMR vaccines, especially persons who are going to be traveling internationally. “We recommend that when they’ve got infants traveling with them who are 6-11 months of age, that they get a first dose (which we consider a zero dose), because they need a routine dose at 12-15 months, and then 4-6 years,” said Dr. Sugerman. He said that it’s safe for adults who are unsure of their status to receive an MMR dose as well.

Dr. Sugerman stressed that despite major strides, “we just don’t have enough coverage in all individuals in this country. Because people are traveling as often as they are, it can be imported. Until measles is eliminated globally, there’s going to be an ongoing risk of importation and potential spread amongst others in their household or community, especially amongst individuals who are not fully vaccinated and, in particular, amongst those who are unvaccinated,” he said.

Dr. Sugerman reports no relevant financial relationships.

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

The Centers for Disease Control and Prevention has issued a Health Alert Network (HAN) health advisory notifying clinicians and public health officials of a confirmed measles case in an individual who for 2 days (February 17-18) attended a large religious gathering that was attended by an estimated 20,000 people at Asbury University in Wilmore, Ky.

Given that large numbers of people might have been exposed to the attendee (who was not vaccinated) and that the individual had a history of recent international travel, the CDC has encouraged clinicians to be vigilant for patients presenting with symptoms that meet the measles case definition. A steady increase in measles cases from 49 in 2021 to 121 in 2022 in children who were not fully vaccinated – coupled with outbreaks in Ohio and Minnesota – underscores the potential gravity of the CDC advisory as well as the need to mitigate the risk of ongoing or secondary transmission.

Currently, little is known about the individual who contracted measles other than the fact that he is a resident of Jessamine County, Ky., according to a news release issued by the Kentucky Department of Public Health. It is the third confirmed case in Kentucky over the past 3 months. State and national health officials are concerned that the individual might have transmitted measles to attendees visiting from other states.

David Sugerman, MD, MPH, a medical officer in CDC’s division of viral diseases and lead for the measles, rubella, and cytomegalovirus team, noted that the timing of the alert coincides with the period in which persons who had had contact with the initial case patient might be expected to develop symptoms.

For clinicians, “It’s really about considering measles in any un- or undervaccinated patient that arrives at a clinic and recently traveled internationally,” Dr. Sugerman told this news organization. He explained that “when doctors are seeing patients, they’re not going to necessarily share that information off the bat when they present with fever or rash, or if their child has fever and rash, or that they traveled internationally. So, eliciting that history from the patient or their parents is really critical.”

The CDC recommends that measles be considered in anyone presenting with a febrile illness and symptoms that are clinically compatible with measles (that is, rash, cough, coryza, or conjunctivitis), as well as in patients who have recently traveled abroad, especially to countries with ongoing outbreaks, including India, Somalia, and Yemen.

“In general, if they’ve traveled internationally and they are undervaccinated, measles should be part of the differential diagnosis,” Sugerman said. He also emphasized the need to follow airborne isolation precautions in addition to general infection control measures.

Immediate triage is critical, especially since overcrowded waiting rooms might be filled with patients who are not yet eligible for vaccination or are not up to date or fully vaccinated.

“Measles is under airborne isolation criteria and precautions, and therefore, [patients] need to be placed as soon as possible into a negative pressure or airborne infection isolation room – and that should be a single room,” he explained. He noted, “In some settings, there may not be a negative pressure room, e.g., an outpatient pediatrics or family medicine office.”

Dr. Sugerman said that in these circumstances, patients should be placed in a room with masked health care providers who have received two doses of measles, mumps, and rubella (MMR) vaccine and that they should wear an N95 mask when entering the room and interviewing the patient.

Clinicians should follow CDC’s testing recommendations and collect a nasopharyngeal or throat swab or a urine specimen for PCR testing and a blood specimen for serology. In addition, they should immediately report cases to local and state public health authorities.

For all patients, it’s critical to be up to date on MMR vaccines, especially persons who are going to be traveling internationally. “We recommend that when they’ve got infants traveling with them who are 6-11 months of age, that they get a first dose (which we consider a zero dose), because they need a routine dose at 12-15 months, and then 4-6 years,” said Dr. Sugerman. He said that it’s safe for adults who are unsure of their status to receive an MMR dose as well.

Dr. Sugerman stressed that despite major strides, “we just don’t have enough coverage in all individuals in this country. Because people are traveling as often as they are, it can be imported. Until measles is eliminated globally, there’s going to be an ongoing risk of importation and potential spread amongst others in their household or community, especially amongst individuals who are not fully vaccinated and, in particular, amongst those who are unvaccinated,” he said.

Dr. Sugerman reports no relevant financial relationships.

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

The Food and Drug Administration has expanded the indication for adjuvant abemaciclib (Verzenio) in combination with endocrine therapy for patients with hormone receptor–positive, HER2-negative, node-positive early breast cancer who are at high risk for recurrence.

Abemaciclib was previously approved for this group of high-risk patients with the requirement that they have a Ki-67 score of at least 20%. The new expansion removes the Ki-67 testing requirement, meaning more patients are now eligible to receive this drug. High-risk patients eligible for the CDK4/6 inhibitor can now be identified solely on the basis of nodal status, tumor size, and tumor grade.

The FDA’s decision to expand the approval was based on 4-year data from the phase 3 monarchE trial of adjuvant abemaciclib, which showed benefit in invasive disease-free survival beyond the 2-year treatment course.

At 4 years, 85.5% of patients remained recurrence free with abemaciclib plus endocrine therapy, compared with 78.6% who received endocrine therapy alone, an absolute difference in invasive disease-free survival of 6.9%.

“The initial Verzenio FDA approval in early breast cancer was practice changing and now, through this indication expansion, we have the potential to reduce the risk of breast cancer recurrence for many more patients, relying solely on commonly utilized clinicopathologic features to identify them,” Erika P. Hamilton, MD, an investigator on the monarchE clinical trial, said in a press release.

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

The Food and Drug Administration has expanded the indication for adjuvant abemaciclib (Verzenio) in combination with endocrine therapy for patients with hormone receptor–positive, HER2-negative, node-positive early breast cancer who are at high risk for recurrence.

Abemaciclib was previously approved for this group of high-risk patients with the requirement that they have a Ki-67 score of at least 20%. The new expansion removes the Ki-67 testing requirement, meaning more patients are now eligible to receive this drug. High-risk patients eligible for the CDK4/6 inhibitor can now be identified solely on the basis of nodal status, tumor size, and tumor grade.

The FDA’s decision to expand the approval was based on 4-year data from the phase 3 monarchE trial of adjuvant abemaciclib, which showed benefit in invasive disease-free survival beyond the 2-year treatment course.

At 4 years, 85.5% of patients remained recurrence free with abemaciclib plus endocrine therapy, compared with 78.6% who received endocrine therapy alone, an absolute difference in invasive disease-free survival of 6.9%.

“The initial Verzenio FDA approval in early breast cancer was practice changing and now, through this indication expansion, we have the potential to reduce the risk of breast cancer recurrence for many more patients, relying solely on commonly utilized clinicopathologic features to identify them,” Erika P. Hamilton, MD, an investigator on the monarchE clinical trial, said in a press release.

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

The Food and Drug Administration has expanded the indication for adjuvant abemaciclib (Verzenio) in combination with endocrine therapy for patients with hormone receptor–positive, HER2-negative, node-positive early breast cancer who are at high risk for recurrence.

Abemaciclib was previously approved for this group of high-risk patients with the requirement that they have a Ki-67 score of at least 20%. The new expansion removes the Ki-67 testing requirement, meaning more patients are now eligible to receive this drug. High-risk patients eligible for the CDK4/6 inhibitor can now be identified solely on the basis of nodal status, tumor size, and tumor grade.

The FDA’s decision to expand the approval was based on 4-year data from the phase 3 monarchE trial of adjuvant abemaciclib, which showed benefit in invasive disease-free survival beyond the 2-year treatment course.

At 4 years, 85.5% of patients remained recurrence free with abemaciclib plus endocrine therapy, compared with 78.6% who received endocrine therapy alone, an absolute difference in invasive disease-free survival of 6.9%.

“The initial Verzenio FDA approval in early breast cancer was practice changing and now, through this indication expansion, we have the potential to reduce the risk of breast cancer recurrence for many more patients, relying solely on commonly utilized clinicopathologic features to identify them,” Erika P. Hamilton, MD, an investigator on the monarchE clinical trial, said in a press release.

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

The Food and Drug Administration has declined to approve omecamtiv mecarbil (Cytokinetics) for treatment of adults with chronic heart failure with reduced ejection fraction (HFrEF), citing a lack of evidence on efficacy.

Waldemarus/iStock/Getty Images Plus

Omecamtiv mecarbil is a first-in-class, selective cardiac myosin activator designed to improve cardiac performance.

Last December, a panel of FDA advisers recommended against approval of omecamtiv mecarbil for chronic HFrEF, as reported by this news organization.

The FDA Cardiovascular and Renal Drugs Advisory Committee voted 8 to 3 (with no abstentions) that the benefits of omecamtiv mecarbil do not outweigh the risks for HFrEF. The drug had a PDUFA date of February 28.

The committee’s decision was based on results from the phase 3 GALACTIC-HF trial, which enrolled 8,256 patients with HFrEF who were at risk of hospitalization and death, despite standard-of-care therapy.

As previously reported by this news organization, omecamtiv mecarbil produced a positive result for the study’s primary endpoint, with a 2.1% absolute reduction in the combined rate of cardiovascular (CV) death, first HF hospitalization, or first urgent visit for HF, compared with placebo during a median follow-up of about 22 months.

This represented an 8% relative risk reduction and broke down as a 0.6% absolute drop in CV death, compared with placebo, a 0.7% cut in HF hospitalization, and a 0.8% drop in urgent outpatient HF visits.

In a complete response letter, the FDA said GALACTIC-HF is “not sufficiently persuasive to establish substantial evidence of effectiveness for reducing the risk of heart failure events and cardiovascular death” in adults with HFrEF, Cytokinetics shared in a news release.

Further, the FDA said results from an additional clinical trial of omecamtiv mecarbil are required to establish substantial evidence of effectiveness for the treatment of HFrEF, with benefits that outweigh the risks, Cytokinetics said.

The company said it will request a meeting with the FDA to gain a better understanding of what may be required to support potential approval of omecamtiv mecarbil. However, the company also said it has “no plans” to conduct an additional clinical trial of omecamtiv mecarbil.

Instead, the company said its focus remains on the development of aficamten, the next-in-class cardiac myosin inhibitor, currently the subject of SEQUOIA-HCM, a phase 3 clinical trial in patients with obstructive hypertrophic cardiomyopathy.

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

The Food and Drug Administration has declined to approve omecamtiv mecarbil (Cytokinetics) for treatment of adults with chronic heart failure with reduced ejection fraction (HFrEF), citing a lack of evidence on efficacy.

Waldemarus/iStock/Getty Images Plus

Omecamtiv mecarbil is a first-in-class, selective cardiac myosin activator designed to improve cardiac performance.

Last December, a panel of FDA advisers recommended against approval of omecamtiv mecarbil for chronic HFrEF, as reported by this news organization.

The FDA Cardiovascular and Renal Drugs Advisory Committee voted 8 to 3 (with no abstentions) that the benefits of omecamtiv mecarbil do not outweigh the risks for HFrEF. The drug had a PDUFA date of February 28.

The committee’s decision was based on results from the phase 3 GALACTIC-HF trial, which enrolled 8,256 patients with HFrEF who were at risk of hospitalization and death, despite standard-of-care therapy.

As previously reported by this news organization, omecamtiv mecarbil produced a positive result for the study’s primary endpoint, with a 2.1% absolute reduction in the combined rate of cardiovascular (CV) death, first HF hospitalization, or first urgent visit for HF, compared with placebo during a median follow-up of about 22 months.

This represented an 8% relative risk reduction and broke down as a 0.6% absolute drop in CV death, compared with placebo, a 0.7% cut in HF hospitalization, and a 0.8% drop in urgent outpatient HF visits.

In a complete response letter, the FDA said GALACTIC-HF is “not sufficiently persuasive to establish substantial evidence of effectiveness for reducing the risk of heart failure events and cardiovascular death” in adults with HFrEF, Cytokinetics shared in a news release.

Further, the FDA said results from an additional clinical trial of omecamtiv mecarbil are required to establish substantial evidence of effectiveness for the treatment of HFrEF, with benefits that outweigh the risks, Cytokinetics said.

The company said it will request a meeting with the FDA to gain a better understanding of what may be required to support potential approval of omecamtiv mecarbil. However, the company also said it has “no plans” to conduct an additional clinical trial of omecamtiv mecarbil.

Instead, the company said its focus remains on the development of aficamten, the next-in-class cardiac myosin inhibitor, currently the subject of SEQUOIA-HCM, a phase 3 clinical trial in patients with obstructive hypertrophic cardiomyopathy.

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

The Food and Drug Administration has declined to approve omecamtiv mecarbil (Cytokinetics) for treatment of adults with chronic heart failure with reduced ejection fraction (HFrEF), citing a lack of evidence on efficacy.

Waldemarus/iStock/Getty Images Plus

Omecamtiv mecarbil is a first-in-class, selective cardiac myosin activator designed to improve cardiac performance.

Last December, a panel of FDA advisers recommended against approval of omecamtiv mecarbil for chronic HFrEF, as reported by this news organization.

The FDA Cardiovascular and Renal Drugs Advisory Committee voted 8 to 3 (with no abstentions) that the benefits of omecamtiv mecarbil do not outweigh the risks for HFrEF. The drug had a PDUFA date of February 28.

The committee’s decision was based on results from the phase 3 GALACTIC-HF trial, which enrolled 8,256 patients with HFrEF who were at risk of hospitalization and death, despite standard-of-care therapy.

As previously reported by this news organization, omecamtiv mecarbil produced a positive result for the study’s primary endpoint, with a 2.1% absolute reduction in the combined rate of cardiovascular (CV) death, first HF hospitalization, or first urgent visit for HF, compared with placebo during a median follow-up of about 22 months.

This represented an 8% relative risk reduction and broke down as a 0.6% absolute drop in CV death, compared with placebo, a 0.7% cut in HF hospitalization, and a 0.8% drop in urgent outpatient HF visits.

In a complete response letter, the FDA said GALACTIC-HF is “not sufficiently persuasive to establish substantial evidence of effectiveness for reducing the risk of heart failure events and cardiovascular death” in adults with HFrEF, Cytokinetics shared in a news release.

Further, the FDA said results from an additional clinical trial of omecamtiv mecarbil are required to establish substantial evidence of effectiveness for the treatment of HFrEF, with benefits that outweigh the risks, Cytokinetics said.

The company said it will request a meeting with the FDA to gain a better understanding of what may be required to support potential approval of omecamtiv mecarbil. However, the company also said it has “no plans” to conduct an additional clinical trial of omecamtiv mecarbil.

Instead, the company said its focus remains on the development of aficamten, the next-in-class cardiac myosin inhibitor, currently the subject of SEQUOIA-HCM, a phase 3 clinical trial in patients with obstructive hypertrophic cardiomyopathy.

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

There is a potential risk of early structural valve deterioration with the Abbott Trifecta valve and Trifecta valve with glide technology (Trifecta GT), the U.S. Food and Drug Administration says in a letter to health care professionals posted on its website.  

Evidence in the literature suggests a higher cumulative incidence of early structural valve deterioration (SVD) and a lower freedom from reintervention due to SVD with the Trifecta valves, compared with other commercially available bovine pericardial valves, the FDA says.

The Trifecta and Trifecta GT valves are heart valve replacement devices intended to treat diseased, damaged, or malfunctioning native or prosthetic aortic heart valves, the letter notes. The first-generation Trifecta valve was approved in 2011 but is no longer marketed in the United States. The Trifecta GT valve was approved in 2016.

Medical device reports (MDRs) received by the FDA describe early SVD with Trifecta valves, with a peak time to SVD of 3 to 4 years post-implant. “Reported outcomes include surgical valve explant/replacement, transcatheter valve-in-valve intervention, and in some cases death,” the FDA notes.

In a letter to customers, Abbott says a “complaint analysis has shown that most cases of early SVD which occur within 5 years post-implant are characterized as a non-calcific leaflet tear, while most cases of late SVD which occur beyond 5 years post-implant are characterized as a fibrous-calcific SVD.” 

The FDA recommends that health care providers take the following actions:

• Be aware of the potential risk of early SVD with Trifecta valves, and current patient management considerations, as communicated by Abbott.
• Discuss the risks and benefits of all available aortic valve treatment options with patients and caregivers as part of shared clinical decision-making prior to surgery.
• Read and carefully follow the Instructions for Use when implanting a Trifecta GT valve.
• Monitor patients who have undergone implantation with Trifecta valves for signs and symptoms of potential SVD.
• Instruct patients to seek medical attention with new onset of symptoms such as shortness of breath or fatigue.
• Ensure lifelong follow-up visits, conducted at least yearly, including transthoracic echocardiogram assessment of the valve beginning 1-year post-implant.

The FDA is working with Abbott to further evaluate the issue and develop additional patient management strategies, if needed. The FDA says it will continue to monitor the literature and reports of adverse events related to the issue.

Clinicians are encouraged to report any adverse events or quality problems with the Trifecta valves to their local Abbott representative or the customer service department at 1-800-544-1664.

Health care professionals can also report adverse reactions or quality problems they experience using these devices to the FDA’s MedWatch program.

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

There is a potential risk of early structural valve deterioration with the Abbott Trifecta valve and Trifecta valve with glide technology (Trifecta GT), the U.S. Food and Drug Administration says in a letter to health care professionals posted on its website.  

Evidence in the literature suggests a higher cumulative incidence of early structural valve deterioration (SVD) and a lower freedom from reintervention due to SVD with the Trifecta valves, compared with other commercially available bovine pericardial valves, the FDA says.

The Trifecta and Trifecta GT valves are heart valve replacement devices intended to treat diseased, damaged, or malfunctioning native or prosthetic aortic heart valves, the letter notes. The first-generation Trifecta valve was approved in 2011 but is no longer marketed in the United States. The Trifecta GT valve was approved in 2016.

Medical device reports (MDRs) received by the FDA describe early SVD with Trifecta valves, with a peak time to SVD of 3 to 4 years post-implant. “Reported outcomes include surgical valve explant/replacement, transcatheter valve-in-valve intervention, and in some cases death,” the FDA notes.

In a letter to customers, Abbott says a “complaint analysis has shown that most cases of early SVD which occur within 5 years post-implant are characterized as a non-calcific leaflet tear, while most cases of late SVD which occur beyond 5 years post-implant are characterized as a fibrous-calcific SVD.” 

The FDA recommends that health care providers take the following actions:

• Be aware of the potential risk of early SVD with Trifecta valves, and current patient management considerations, as communicated by Abbott.
• Discuss the risks and benefits of all available aortic valve treatment options with patients and caregivers as part of shared clinical decision-making prior to surgery.
• Read and carefully follow the Instructions for Use when implanting a Trifecta GT valve.
• Monitor patients who have undergone implantation with Trifecta valves for signs and symptoms of potential SVD.
• Instruct patients to seek medical attention with new onset of symptoms such as shortness of breath or fatigue.
• Ensure lifelong follow-up visits, conducted at least yearly, including transthoracic echocardiogram assessment of the valve beginning 1-year post-implant.

The FDA is working with Abbott to further evaluate the issue and develop additional patient management strategies, if needed. The FDA says it will continue to monitor the literature and reports of adverse events related to the issue.

Clinicians are encouraged to report any adverse events or quality problems with the Trifecta valves to their local Abbott representative or the customer service department at 1-800-544-1664.

Health care professionals can also report adverse reactions or quality problems they experience using these devices to the FDA’s MedWatch program.

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

There is a potential risk of early structural valve deterioration with the Abbott Trifecta valve and Trifecta valve with glide technology (Trifecta GT), the U.S. Food and Drug Administration says in a letter to health care professionals posted on its website.  

Evidence in the literature suggests a higher cumulative incidence of early structural valve deterioration (SVD) and a lower freedom from reintervention due to SVD with the Trifecta valves, compared with other commercially available bovine pericardial valves, the FDA says.

The Trifecta and Trifecta GT valves are heart valve replacement devices intended to treat diseased, damaged, or malfunctioning native or prosthetic aortic heart valves, the letter notes. The first-generation Trifecta valve was approved in 2011 but is no longer marketed in the United States. The Trifecta GT valve was approved in 2016.

Medical device reports (MDRs) received by the FDA describe early SVD with Trifecta valves, with a peak time to SVD of 3 to 4 years post-implant. “Reported outcomes include surgical valve explant/replacement, transcatheter valve-in-valve intervention, and in some cases death,” the FDA notes.

In a letter to customers, Abbott says a “complaint analysis has shown that most cases of early SVD which occur within 5 years post-implant are characterized as a non-calcific leaflet tear, while most cases of late SVD which occur beyond 5 years post-implant are characterized as a fibrous-calcific SVD.” 

The FDA recommends that health care providers take the following actions:

• Be aware of the potential risk of early SVD with Trifecta valves, and current patient management considerations, as communicated by Abbott.
• Discuss the risks and benefits of all available aortic valve treatment options with patients and caregivers as part of shared clinical decision-making prior to surgery.
• Read and carefully follow the Instructions for Use when implanting a Trifecta GT valve.
• Monitor patients who have undergone implantation with Trifecta valves for signs and symptoms of potential SVD.
• Instruct patients to seek medical attention with new onset of symptoms such as shortness of breath or fatigue.
• Ensure lifelong follow-up visits, conducted at least yearly, including transthoracic echocardiogram assessment of the valve beginning 1-year post-implant.

The FDA is working with Abbott to further evaluate the issue and develop additional patient management strategies, if needed. The FDA says it will continue to monitor the literature and reports of adverse events related to the issue.

Clinicians are encouraged to report any adverse events or quality problems with the Trifecta valves to their local Abbott representative or the customer service department at 1-800-544-1664.

Health care professionals can also report adverse reactions or quality problems they experience using these devices to the FDA’s MedWatch program.

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

The European Commission has granted conditional marketing authorization for etranacogene dezaparvovec (Hemgenix), the first and only one-time gene therapy for the treatment of severe and moderately severe hemophilia B (congenital factor IX deficiency) in adults without a history of factor IX inhibitors.

The approval means that the product will now be available in all the countries of the European Union as well as the European Economic Area.

The gene therapy was approved in the United States in November 2022. It was launched with a price tag of $3.5 million, making it the most expensive treatment to date.

The treatment comprises a one-time infusion of a functional gene that acts as a blueprint for coagulation factor IX, a protein important for blood clotting, stated the manufacturer, CSL.

People living with hemophilia B currently require lifelong treatment of intravenous infusions of factor IX to maintain sufficient levels, which can have a significant impact on their quality of life and well-being, the company explained in its press release.

The approval was based on findings from the pivotal HOPE-B trial, a single-arm, open-label study of 54 men who relied on factor IX replacement therapy; first results from this trial were reported at the 2020 annual meeting of the American Society of Hematology.

The results showed that patients with hemophilia B treated with the gene therapy demonstrated stable and durable increases in mean factor IX activity (with a mean factor IX activity of 36.9%), which led to an adjusted annualized bleeding rate reduction of 64%.

After receiving the gene therapy, 96% of patients discontinued routine factor IX prophylaxis and mean factor IX consumption was reduced by 97% at 18 months post treatment compared with the lead-in period, the company noted.

“Data from the HOPE-B study demonstrate the potential of Hemgenix to remove the need for routine prophylaxis by providing durable factor IX activity, as well as improved bleeding outcomes and quality of life for people with hemophilia B,” said one of the trialists, Wolfgang Miesbach, MD, PHD, head of coagulation disorders at the Comprehensive Care Center, University Hospital of Frankfurt, Germany.

This European approval “marks an important step forward in the treatment of hemophilia B, which could be transformative for people who are debilitated by bleeds into their muscles, joints, and internal organs, alleviating the burden of lifelong intravenous infusions of factor IX products,” Dr. Miesbach said in the company press release.

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

The European Commission has granted conditional marketing authorization for etranacogene dezaparvovec (Hemgenix), the first and only one-time gene therapy for the treatment of severe and moderately severe hemophilia B (congenital factor IX deficiency) in adults without a history of factor IX inhibitors.

The approval means that the product will now be available in all the countries of the European Union as well as the European Economic Area.

The gene therapy was approved in the United States in November 2022. It was launched with a price tag of $3.5 million, making it the most expensive treatment to date.

The treatment comprises a one-time infusion of a functional gene that acts as a blueprint for coagulation factor IX, a protein important for blood clotting, stated the manufacturer, CSL.

People living with hemophilia B currently require lifelong treatment of intravenous infusions of factor IX to maintain sufficient levels, which can have a significant impact on their quality of life and well-being, the company explained in its press release.

The approval was based on findings from the pivotal HOPE-B trial, a single-arm, open-label study of 54 men who relied on factor IX replacement therapy; first results from this trial were reported at the 2020 annual meeting of the American Society of Hematology.

The results showed that patients with hemophilia B treated with the gene therapy demonstrated stable and durable increases in mean factor IX activity (with a mean factor IX activity of 36.9%), which led to an adjusted annualized bleeding rate reduction of 64%.

After receiving the gene therapy, 96% of patients discontinued routine factor IX prophylaxis and mean factor IX consumption was reduced by 97% at 18 months post treatment compared with the lead-in period, the company noted.

“Data from the HOPE-B study demonstrate the potential of Hemgenix to remove the need for routine prophylaxis by providing durable factor IX activity, as well as improved bleeding outcomes and quality of life for people with hemophilia B,” said one of the trialists, Wolfgang Miesbach, MD, PHD, head of coagulation disorders at the Comprehensive Care Center, University Hospital of Frankfurt, Germany.

This European approval “marks an important step forward in the treatment of hemophilia B, which could be transformative for people who are debilitated by bleeds into their muscles, joints, and internal organs, alleviating the burden of lifelong intravenous infusions of factor IX products,” Dr. Miesbach said in the company press release.

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

The European Commission has granted conditional marketing authorization for etranacogene dezaparvovec (Hemgenix), the first and only one-time gene therapy for the treatment of severe and moderately severe hemophilia B (congenital factor IX deficiency) in adults without a history of factor IX inhibitors.

The approval means that the product will now be available in all the countries of the European Union as well as the European Economic Area.

The gene therapy was approved in the United States in November 2022. It was launched with a price tag of $3.5 million, making it the most expensive treatment to date.

The treatment comprises a one-time infusion of a functional gene that acts as a blueprint for coagulation factor IX, a protein important for blood clotting, stated the manufacturer, CSL.

People living with hemophilia B currently require lifelong treatment of intravenous infusions of factor IX to maintain sufficient levels, which can have a significant impact on their quality of life and well-being, the company explained in its press release.

The approval was based on findings from the pivotal HOPE-B trial, a single-arm, open-label study of 54 men who relied on factor IX replacement therapy; first results from this trial were reported at the 2020 annual meeting of the American Society of Hematology.

The results showed that patients with hemophilia B treated with the gene therapy demonstrated stable and durable increases in mean factor IX activity (with a mean factor IX activity of 36.9%), which led to an adjusted annualized bleeding rate reduction of 64%.

After receiving the gene therapy, 96% of patients discontinued routine factor IX prophylaxis and mean factor IX consumption was reduced by 97% at 18 months post treatment compared with the lead-in period, the company noted.

“Data from the HOPE-B study demonstrate the potential of Hemgenix to remove the need for routine prophylaxis by providing durable factor IX activity, as well as improved bleeding outcomes and quality of life for people with hemophilia B,” said one of the trialists, Wolfgang Miesbach, MD, PHD, head of coagulation disorders at the Comprehensive Care Center, University Hospital of Frankfurt, Germany.

This European approval “marks an important step forward in the treatment of hemophilia B, which could be transformative for people who are debilitated by bleeds into their muscles, joints, and internal organs, alleviating the burden of lifelong intravenous infusions of factor IX products,” Dr. Miesbach said in the company press release.

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

The Food and Drug Administration has approved efanesoctocog alfa (Altuviiio), a first-in-class, high-sustained factor VIII replacement therapy for adults and children with hemophilia A.

The product is used once a week and is indicated for routine prophylaxis and on-demand treatment to control bleeding episodes, as well as to control bleeding during surgery (perioperative management).

The manufacturer, Sanofi, says that this is “the first and only hemophilia A treatment that delivers normal to near-normal factor activity levels (over 40%) for most of the week with once-weekly dosing, and significantly reduces bleeds compared to prior factor VIII prophylaxis.”

With this product, “we have the opportunity to provide near-normal factor activity levels for an extended period of time (the majority of a week) with a single dose, which is a first for hemophilia A,” said Angela Weyand, MD, of Michigan Medicine, who was involved in the pivotal phase 3 XTEND-1 trial that led to approval.

Results from the XTEND-1 trial were recently published online in The New England Journal of Medicine. The results show that one injection of efanesoctocog alfa, a factor VIII therapy, resolved almost all bleeding episodes (97%) in the overall patient population and weekly prophylaxis provided mean factor VIII activity in the normal or near-normal range (> 40 IU/dL) for most of the week, as previously reported by this news organization.

The researchers noted that the factor VIII therapies that have been available up to now need to be administered frequently.

In an accompanying editorial, Cindy Leissinger, MD, called efanesoctocog alfa a “victory” for patients with hemophilia A.

“In a crowded field of transformative therapies for hemophilia, efanesoctocog alfa stands out as a winner – a major therapeutic advance that achieves highly protective factor VIII levels with a once-weekly infusion,” wrote Dr. Leissinger, director of the Louisiana Center for Bleeding and Clotting Disorders at Tulane University in New Orleans.

Sanofi said that Altuviiio is expected to be commercially available in April 2023. The company said that it will price the product at parity to the annual cost of treating a prophylaxis patient with its Eloctate product (antihemophilic factor [recombinant], Fc fusion protein). Sanofi will provide comprehensive patient support services and resources online and at 1-855-MyALTUVIIIO (855-692-5888).

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

The Food and Drug Administration has approved efanesoctocog alfa (Altuviiio), a first-in-class, high-sustained factor VIII replacement therapy for adults and children with hemophilia A.

The product is used once a week and is indicated for routine prophylaxis and on-demand treatment to control bleeding episodes, as well as to control bleeding during surgery (perioperative management).

The manufacturer, Sanofi, says that this is “the first and only hemophilia A treatment that delivers normal to near-normal factor activity levels (over 40%) for most of the week with once-weekly dosing, and significantly reduces bleeds compared to prior factor VIII prophylaxis.”

With this product, “we have the opportunity to provide near-normal factor activity levels for an extended period of time (the majority of a week) with a single dose, which is a first for hemophilia A,” said Angela Weyand, MD, of Michigan Medicine, who was involved in the pivotal phase 3 XTEND-1 trial that led to approval.

Results from the XTEND-1 trial were recently published online in The New England Journal of Medicine. The results show that one injection of efanesoctocog alfa, a factor VIII therapy, resolved almost all bleeding episodes (97%) in the overall patient population and weekly prophylaxis provided mean factor VIII activity in the normal or near-normal range (> 40 IU/dL) for most of the week, as previously reported by this news organization.

The researchers noted that the factor VIII therapies that have been available up to now need to be administered frequently.

In an accompanying editorial, Cindy Leissinger, MD, called efanesoctocog alfa a “victory” for patients with hemophilia A.

“In a crowded field of transformative therapies for hemophilia, efanesoctocog alfa stands out as a winner – a major therapeutic advance that achieves highly protective factor VIII levels with a once-weekly infusion,” wrote Dr. Leissinger, director of the Louisiana Center for Bleeding and Clotting Disorders at Tulane University in New Orleans.

Sanofi said that Altuviiio is expected to be commercially available in April 2023. The company said that it will price the product at parity to the annual cost of treating a prophylaxis patient with its Eloctate product (antihemophilic factor [recombinant], Fc fusion protein). Sanofi will provide comprehensive patient support services and resources online and at 1-855-MyALTUVIIIO (855-692-5888).

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

The Food and Drug Administration has approved efanesoctocog alfa (Altuviiio), a first-in-class, high-sustained factor VIII replacement therapy for adults and children with hemophilia A.

The product is used once a week and is indicated for routine prophylaxis and on-demand treatment to control bleeding episodes, as well as to control bleeding during surgery (perioperative management).

The manufacturer, Sanofi, says that this is “the first and only hemophilia A treatment that delivers normal to near-normal factor activity levels (over 40%) for most of the week with once-weekly dosing, and significantly reduces bleeds compared to prior factor VIII prophylaxis.”

With this product, “we have the opportunity to provide near-normal factor activity levels for an extended period of time (the majority of a week) with a single dose, which is a first for hemophilia A,” said Angela Weyand, MD, of Michigan Medicine, who was involved in the pivotal phase 3 XTEND-1 trial that led to approval.

Results from the XTEND-1 trial were recently published online in The New England Journal of Medicine. The results show that one injection of efanesoctocog alfa, a factor VIII therapy, resolved almost all bleeding episodes (97%) in the overall patient population and weekly prophylaxis provided mean factor VIII activity in the normal or near-normal range (> 40 IU/dL) for most of the week, as previously reported by this news organization.

The researchers noted that the factor VIII therapies that have been available up to now need to be administered frequently.

In an accompanying editorial, Cindy Leissinger, MD, called efanesoctocog alfa a “victory” for patients with hemophilia A.

“In a crowded field of transformative therapies for hemophilia, efanesoctocog alfa stands out as a winner – a major therapeutic advance that achieves highly protective factor VIII levels with a once-weekly infusion,” wrote Dr. Leissinger, director of the Louisiana Center for Bleeding and Clotting Disorders at Tulane University in New Orleans.

Sanofi said that Altuviiio is expected to be commercially available in April 2023. The company said that it will price the product at parity to the annual cost of treating a prophylaxis patient with its Eloctate product (antihemophilic factor [recombinant], Fc fusion protein). Sanofi will provide comprehensive patient support services and resources online and at 1-855-MyALTUVIIIO (855-692-5888).

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

Do not purchase or use Delsam Pharma’s Artificial Eye Ointment, the Food and Drug Administration warns.

The announcement released Wednesday adds to a previous warning issued earlier this month for EzriCare Artificial Tears or Delsam Pharma’s Artificial Tears because of potential bacterial contamination. All three products are manufactured by the same company, Global Pharma Healthcare, based in Tamilnadu, India.

Wikimedia Commons/FitzColinGerald/Creative Commons License

The FDA has faulted the company for multiple violations, including “lack of appropriate microbial testing” and “lack of proper controls concerning tamper-evident packaging,” and has banned imports to the United States.

The updated warning from the FDA did not give additional information about the over-the-counter eye ointment beyond potential bacterial contamination. 

On Feb. 1, the CDC issued an alert about an outbreak of a drug-resistant strain of bacteria, Pseudomonas aeruginosa, linked to artificial tear products. To date, 58 patients across 13 states have been identified, and the most commonly reported artificial tear brand was EzriCare Artificial Tears. Five patients had permanent vision loss, and one patient died.

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

Do not purchase or use Delsam Pharma’s Artificial Eye Ointment, the Food and Drug Administration warns.

The announcement released Wednesday adds to a previous warning issued earlier this month for EzriCare Artificial Tears or Delsam Pharma’s Artificial Tears because of potential bacterial contamination. All three products are manufactured by the same company, Global Pharma Healthcare, based in Tamilnadu, India.

Wikimedia Commons/FitzColinGerald/Creative Commons License

The FDA has faulted the company for multiple violations, including “lack of appropriate microbial testing” and “lack of proper controls concerning tamper-evident packaging,” and has banned imports to the United States.

The updated warning from the FDA did not give additional information about the over-the-counter eye ointment beyond potential bacterial contamination. 

On Feb. 1, the CDC issued an alert about an outbreak of a drug-resistant strain of bacteria, Pseudomonas aeruginosa, linked to artificial tear products. To date, 58 patients across 13 states have been identified, and the most commonly reported artificial tear brand was EzriCare Artificial Tears. Five patients had permanent vision loss, and one patient died.

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

Do not purchase or use Delsam Pharma’s Artificial Eye Ointment, the Food and Drug Administration warns.

The announcement released Wednesday adds to a previous warning issued earlier this month for EzriCare Artificial Tears or Delsam Pharma’s Artificial Tears because of potential bacterial contamination. All three products are manufactured by the same company, Global Pharma Healthcare, based in Tamilnadu, India.

Wikimedia Commons/FitzColinGerald/Creative Commons License

The FDA has faulted the company for multiple violations, including “lack of appropriate microbial testing” and “lack of proper controls concerning tamper-evident packaging,” and has banned imports to the United States.

The updated warning from the FDA did not give additional information about the over-the-counter eye ointment beyond potential bacterial contamination. 

On Feb. 1, the CDC issued an alert about an outbreak of a drug-resistant strain of bacteria, Pseudomonas aeruginosa, linked to artificial tear products. To date, 58 patients across 13 states have been identified, and the most commonly reported artificial tear brand was EzriCare Artificial Tears. Five patients had permanent vision loss, and one patient died.

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

Warming U.S. temperatures, the resumption of travel, and new knowledge about Zika’s long-term effects on children signal that Zika prevention and vaccine development should be on public health officials’, doctors’, and communities’ radar, even when community infection is not occurring.

“Although we haven’t seen confirmed Zika virus circulation in the continental United States or its territories for several years, it’s still something that we are closely monitoring, particularly as we move into the summer months,” Erin Staples, MD, PhD, medical epidemiologist at the Arboviral Diseases Branch of the Centers for Disease Control and Prevention in Fort Collins, Colo., told this news organization.

“This is because cases are still being reported in other countries, particularly in South America. Travel to these places is increasing following the pandemic, leaving more potential for individuals who might have acquired the infection to come back and restart community transmission.”
 

How Zika might reemerge

The Aedes aegypti mosquito is the vector by which Zika spreads, and “during the COVID pandemic, these mosquitoes moved further north in the United States, into southern California, and were identified as far north as Washington, D.C.,” said Neil Silverman, MD, professor of clinical obstetrics and gynecology and director of the Infections in Pregnancy Program at UCLA Medical Center in Los Angeles.

“On a population level, Americans have essentially no immunity to Zika from prior infection, and there is no vaccine yet approved. If individuals infected with Zika came into a U.S. region where the Aedes aegypti mosquito was present, that population could be very susceptible to infection spread and even another outbreak. This would be a confluence of bad circumstances, but that’s exactly what infectious disease specialists continue to be watchful about, especially because Zika is so dangerous for fetuses,” said Dr. Silverman.
 

How the public can prepare

The CDC recommends that pregnant women or women who plan to become pregnant avoid traveling to regions where there are currently outbreaks of Zika, but this is not the only way that individuals can protect themselves.

“The message we want to deliver to people is that in the United States, people are at risk for several mosquito-borne diseases every summer beyond just Zika,” Dr. Staples said. “It’s really important that people are instructed to make a habit of wearing EPA [U.S. Environmental Protection Agency)–registered insect repellents when they go outside. Right now, that is the single best tool that we have to prevent mosquito-borne diseases in the U.S.

“From a community standpoint, there are several emerging mosquito control methods that are being evaluated right now, such as genetic modification and irradiation of mosquitoes. These methods are aimed at producing sterile mosquitoes that are released into the wild to mate with the local mosquito population, which will render them infertile. This leads, over time, to suppression of the overall Aedes aegypti mosquito population – the main vector of Zika transmission,” said Dr. Staples.

Monica Gandhi, MD, MPH, professor of medicine and associate chief of the division of HIV, infectious diseases, and global medicine at the University of California, San Francisco, encourages her patients to wear mosquito repellent but cautioned that “there’s no antiviral that you can take for Zika. Until we have a vaccine, the key to controlling/preventing Zika is controlling the mosquitoes that spread the virus.”
 

Vaccines

The National Institute of Allergy and Infectious Diseases (NIAID) is currently investigating a variety of Zika vaccines, including a DNA-based vaccine, (phase 2), a purified inactivated virus vaccine (phase 1), live attenuated vaccines (phase 2), and mRNA vaccines (phase 2).

“I’m most excited about mRNA vaccines because they help patients produce a lot of proteins. The protein from a typical protein-adjuvant vaccine will break down, and patients can only raise an immune response to whatever proteins are left. On the other hand, mRNA vaccines provide the body [with] a recipe to make the protein from the pathogen in high amounts, so that a strong immune response can be raised for protection,” noted Dr. Gandhi.

Moderna’s mRNA-1893 vaccine was recently studied in a randomized, observer-blind, controlled, phase 1 trial among 120 adults in the United States and Puerto Rico, the results of which were published online in The Lancet. “The vaccine was found to be generally well tolerated with no serious adverse events considered related to vaccine. Furthermore, the vaccine was able to generate a potent immune response that was capable of neutralizing the virus in vitro,” said Brett Leav, MD, executive director of clinical development for public health vaccines at Moderna.

“Our mRNA platform technology ... can be very helpful against emerging pandemic threats, as we saw in response to COVID-19. What is unique in our approach is that if the genetic sequence of the virus is known, we can quickly generate vaccines to test for their capability to generate a functional immune response. In the case of the mRNA-1893 trial, the vaccine was developed with antigens that were present in the strain of virus circulating in 2016, but we could easily match whatever strain reemerges,” said Dr. Leav.

A phase 2 trial to confirm the dose of mRNA-1893 in a larger study population is underway.

Although it’s been demonstrated that Moderna’s mRNA vaccine is safe and effective, moving from a phase 2 to a phase 3 study presents a challenge, given the fact that currently, the disease burden from Zika is low. If an outbreak were to occur in the future, these mRNA vaccines could potentially be given emergency approval, as occurred during the COVID pandemic, according to Dr. Silverman.

If approved, provisionally or through a traditional route, the vaccine would “accelerate the ability to tamp down any further outbreaks, because vaccine-based immunity could be made available to a large portion of the population who were pregnant or planning a pregnancy, not just in the U.S. but also in these endemic areas,” said Dr. Silverman.


 

Takeaways from the last Zika outbreak

Practical steps such as mosquito eradication and development of vaccines are not the only takeaway from the recent Zika epidemics inside and outside the United States. A clearer picture of the short- and long-term stakes of the disease has emerged.

According to the CDC, most people who become infected with Zika experience only mild symptoms, such as fever, rash, headache, and muscle pain, but babies conceived by mothers infected with Zika are at risk for stillbirth, miscarriage, and microcephaly and other brain defects.

Although a pregnant woman who tests positive for Zika is in a very high-risk situation, “data show that only about 30% of mothers with Zika have a baby with birth defects. If a pregnant woman contracted Zika, what would happen is we would just do very close screening by ultrasound of the fetus. If microcephaly in utero or fetal brain defects were observed, then a mother would be counseled on her options,” said Dr. Gandhi.

Dr. Silverman noted that “new data on children who were exposed in utero and had normal exams, including head measurements when they were born, have raised concerns. In recently published long-term follow-up studies, even when children born to mothers infected with Zika during pregnancy had normal head growth at least 3 years after birth, they were still at risk for neurodevelopmental delay and behavioral disorders, including impact on coordination and executive function.

“This is another good reason to keep the potential risks of Zika active in the public’s consciousness and in public health planning.”

Dr. Silverman, Dr. Gandhi, and Dr. Staples have disclosed no relevant financial relationships. Dr. Leav is an employee of Moderna and owns stock in the company.

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

Warming U.S. temperatures, the resumption of travel, and new knowledge about Zika’s long-term effects on children signal that Zika prevention and vaccine development should be on public health officials’, doctors’, and communities’ radar, even when community infection is not occurring.

“Although we haven’t seen confirmed Zika virus circulation in the continental United States or its territories for several years, it’s still something that we are closely monitoring, particularly as we move into the summer months,” Erin Staples, MD, PhD, medical epidemiologist at the Arboviral Diseases Branch of the Centers for Disease Control and Prevention in Fort Collins, Colo., told this news organization.

“This is because cases are still being reported in other countries, particularly in South America. Travel to these places is increasing following the pandemic, leaving more potential for individuals who might have acquired the infection to come back and restart community transmission.”
 

How Zika might reemerge

The Aedes aegypti mosquito is the vector by which Zika spreads, and “during the COVID pandemic, these mosquitoes moved further north in the United States, into southern California, and were identified as far north as Washington, D.C.,” said Neil Silverman, MD, professor of clinical obstetrics and gynecology and director of the Infections in Pregnancy Program at UCLA Medical Center in Los Angeles.

“On a population level, Americans have essentially no immunity to Zika from prior infection, and there is no vaccine yet approved. If individuals infected with Zika came into a U.S. region where the Aedes aegypti mosquito was present, that population could be very susceptible to infection spread and even another outbreak. This would be a confluence of bad circumstances, but that’s exactly what infectious disease specialists continue to be watchful about, especially because Zika is so dangerous for fetuses,” said Dr. Silverman.
 

How the public can prepare

The CDC recommends that pregnant women or women who plan to become pregnant avoid traveling to regions where there are currently outbreaks of Zika, but this is not the only way that individuals can protect themselves.

“The message we want to deliver to people is that in the United States, people are at risk for several mosquito-borne diseases every summer beyond just Zika,” Dr. Staples said. “It’s really important that people are instructed to make a habit of wearing EPA [U.S. Environmental Protection Agency)–registered insect repellents when they go outside. Right now, that is the single best tool that we have to prevent mosquito-borne diseases in the U.S.

“From a community standpoint, there are several emerging mosquito control methods that are being evaluated right now, such as genetic modification and irradiation of mosquitoes. These methods are aimed at producing sterile mosquitoes that are released into the wild to mate with the local mosquito population, which will render them infertile. This leads, over time, to suppression of the overall Aedes aegypti mosquito population – the main vector of Zika transmission,” said Dr. Staples.

Monica Gandhi, MD, MPH, professor of medicine and associate chief of the division of HIV, infectious diseases, and global medicine at the University of California, San Francisco, encourages her patients to wear mosquito repellent but cautioned that “there’s no antiviral that you can take for Zika. Until we have a vaccine, the key to controlling/preventing Zika is controlling the mosquitoes that spread the virus.”
 

Vaccines

The National Institute of Allergy and Infectious Diseases (NIAID) is currently investigating a variety of Zika vaccines, including a DNA-based vaccine, (phase 2), a purified inactivated virus vaccine (phase 1), live attenuated vaccines (phase 2), and mRNA vaccines (phase 2).

“I’m most excited about mRNA vaccines because they help patients produce a lot of proteins. The protein from a typical protein-adjuvant vaccine will break down, and patients can only raise an immune response to whatever proteins are left. On the other hand, mRNA vaccines provide the body [with] a recipe to make the protein from the pathogen in high amounts, so that a strong immune response can be raised for protection,” noted Dr. Gandhi.

Moderna’s mRNA-1893 vaccine was recently studied in a randomized, observer-blind, controlled, phase 1 trial among 120 adults in the United States and Puerto Rico, the results of which were published online in The Lancet. “The vaccine was found to be generally well tolerated with no serious adverse events considered related to vaccine. Furthermore, the vaccine was able to generate a potent immune response that was capable of neutralizing the virus in vitro,” said Brett Leav, MD, executive director of clinical development for public health vaccines at Moderna.

“Our mRNA platform technology ... can be very helpful against emerging pandemic threats, as we saw in response to COVID-19. What is unique in our approach is that if the genetic sequence of the virus is known, we can quickly generate vaccines to test for their capability to generate a functional immune response. In the case of the mRNA-1893 trial, the vaccine was developed with antigens that were present in the strain of virus circulating in 2016, but we could easily match whatever strain reemerges,” said Dr. Leav.

A phase 2 trial to confirm the dose of mRNA-1893 in a larger study population is underway.

Although it’s been demonstrated that Moderna’s mRNA vaccine is safe and effective, moving from a phase 2 to a phase 3 study presents a challenge, given the fact that currently, the disease burden from Zika is low. If an outbreak were to occur in the future, these mRNA vaccines could potentially be given emergency approval, as occurred during the COVID pandemic, according to Dr. Silverman.

If approved, provisionally or through a traditional route, the vaccine would “accelerate the ability to tamp down any further outbreaks, because vaccine-based immunity could be made available to a large portion of the population who were pregnant or planning a pregnancy, not just in the U.S. but also in these endemic areas,” said Dr. Silverman.


 

Takeaways from the last Zika outbreak

Practical steps such as mosquito eradication and development of vaccines are not the only takeaway from the recent Zika epidemics inside and outside the United States. A clearer picture of the short- and long-term stakes of the disease has emerged.

According to the CDC, most people who become infected with Zika experience only mild symptoms, such as fever, rash, headache, and muscle pain, but babies conceived by mothers infected with Zika are at risk for stillbirth, miscarriage, and microcephaly and other brain defects.

Although a pregnant woman who tests positive for Zika is in a very high-risk situation, “data show that only about 30% of mothers with Zika have a baby with birth defects. If a pregnant woman contracted Zika, what would happen is we would just do very close screening by ultrasound of the fetus. If microcephaly in utero or fetal brain defects were observed, then a mother would be counseled on her options,” said Dr. Gandhi.

Dr. Silverman noted that “new data on children who were exposed in utero and had normal exams, including head measurements when they were born, have raised concerns. In recently published long-term follow-up studies, even when children born to mothers infected with Zika during pregnancy had normal head growth at least 3 years after birth, they were still at risk for neurodevelopmental delay and behavioral disorders, including impact on coordination and executive function.

“This is another good reason to keep the potential risks of Zika active in the public’s consciousness and in public health planning.”

Dr. Silverman, Dr. Gandhi, and Dr. Staples have disclosed no relevant financial relationships. Dr. Leav is an employee of Moderna and owns stock in the company.

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

Warming U.S. temperatures, the resumption of travel, and new knowledge about Zika’s long-term effects on children signal that Zika prevention and vaccine development should be on public health officials’, doctors’, and communities’ radar, even when community infection is not occurring.

“Although we haven’t seen confirmed Zika virus circulation in the continental United States or its territories for several years, it’s still something that we are closely monitoring, particularly as we move into the summer months,” Erin Staples, MD, PhD, medical epidemiologist at the Arboviral Diseases Branch of the Centers for Disease Control and Prevention in Fort Collins, Colo., told this news organization.

“This is because cases are still being reported in other countries, particularly in South America. Travel to these places is increasing following the pandemic, leaving more potential for individuals who might have acquired the infection to come back and restart community transmission.”
 

How Zika might reemerge

The Aedes aegypti mosquito is the vector by which Zika spreads, and “during the COVID pandemic, these mosquitoes moved further north in the United States, into southern California, and were identified as far north as Washington, D.C.,” said Neil Silverman, MD, professor of clinical obstetrics and gynecology and director of the Infections in Pregnancy Program at UCLA Medical Center in Los Angeles.

“On a population level, Americans have essentially no immunity to Zika from prior infection, and there is no vaccine yet approved. If individuals infected with Zika came into a U.S. region where the Aedes aegypti mosquito was present, that population could be very susceptible to infection spread and even another outbreak. This would be a confluence of bad circumstances, but that’s exactly what infectious disease specialists continue to be watchful about, especially because Zika is so dangerous for fetuses,” said Dr. Silverman.
 

How the public can prepare

The CDC recommends that pregnant women or women who plan to become pregnant avoid traveling to regions where there are currently outbreaks of Zika, but this is not the only way that individuals can protect themselves.

“The message we want to deliver to people is that in the United States, people are at risk for several mosquito-borne diseases every summer beyond just Zika,” Dr. Staples said. “It’s really important that people are instructed to make a habit of wearing EPA [U.S. Environmental Protection Agency)–registered insect repellents when they go outside. Right now, that is the single best tool that we have to prevent mosquito-borne diseases in the U.S.

“From a community standpoint, there are several emerging mosquito control methods that are being evaluated right now, such as genetic modification and irradiation of mosquitoes. These methods are aimed at producing sterile mosquitoes that are released into the wild to mate with the local mosquito population, which will render them infertile. This leads, over time, to suppression of the overall Aedes aegypti mosquito population – the main vector of Zika transmission,” said Dr. Staples.

Monica Gandhi, MD, MPH, professor of medicine and associate chief of the division of HIV, infectious diseases, and global medicine at the University of California, San Francisco, encourages her patients to wear mosquito repellent but cautioned that “there’s no antiviral that you can take for Zika. Until we have a vaccine, the key to controlling/preventing Zika is controlling the mosquitoes that spread the virus.”
 

Vaccines

The National Institute of Allergy and Infectious Diseases (NIAID) is currently investigating a variety of Zika vaccines, including a DNA-based vaccine, (phase 2), a purified inactivated virus vaccine (phase 1), live attenuated vaccines (phase 2), and mRNA vaccines (phase 2).

“I’m most excited about mRNA vaccines because they help patients produce a lot of proteins. The protein from a typical protein-adjuvant vaccine will break down, and patients can only raise an immune response to whatever proteins are left. On the other hand, mRNA vaccines provide the body [with] a recipe to make the protein from the pathogen in high amounts, so that a strong immune response can be raised for protection,” noted Dr. Gandhi.

Moderna’s mRNA-1893 vaccine was recently studied in a randomized, observer-blind, controlled, phase 1 trial among 120 adults in the United States and Puerto Rico, the results of which were published online in The Lancet. “The vaccine was found to be generally well tolerated with no serious adverse events considered related to vaccine. Furthermore, the vaccine was able to generate a potent immune response that was capable of neutralizing the virus in vitro,” said Brett Leav, MD, executive director of clinical development for public health vaccines at Moderna.

“Our mRNA platform technology ... can be very helpful against emerging pandemic threats, as we saw in response to COVID-19. What is unique in our approach is that if the genetic sequence of the virus is known, we can quickly generate vaccines to test for their capability to generate a functional immune response. In the case of the mRNA-1893 trial, the vaccine was developed with antigens that were present in the strain of virus circulating in 2016, but we could easily match whatever strain reemerges,” said Dr. Leav.

A phase 2 trial to confirm the dose of mRNA-1893 in a larger study population is underway.

Although it’s been demonstrated that Moderna’s mRNA vaccine is safe and effective, moving from a phase 2 to a phase 3 study presents a challenge, given the fact that currently, the disease burden from Zika is low. If an outbreak were to occur in the future, these mRNA vaccines could potentially be given emergency approval, as occurred during the COVID pandemic, according to Dr. Silverman.

If approved, provisionally or through a traditional route, the vaccine would “accelerate the ability to tamp down any further outbreaks, because vaccine-based immunity could be made available to a large portion of the population who were pregnant or planning a pregnancy, not just in the U.S. but also in these endemic areas,” said Dr. Silverman.


 

Takeaways from the last Zika outbreak

Practical steps such as mosquito eradication and development of vaccines are not the only takeaway from the recent Zika epidemics inside and outside the United States. A clearer picture of the short- and long-term stakes of the disease has emerged.

According to the CDC, most people who become infected with Zika experience only mild symptoms, such as fever, rash, headache, and muscle pain, but babies conceived by mothers infected with Zika are at risk for stillbirth, miscarriage, and microcephaly and other brain defects.

Although a pregnant woman who tests positive for Zika is in a very high-risk situation, “data show that only about 30% of mothers with Zika have a baby with birth defects. If a pregnant woman contracted Zika, what would happen is we would just do very close screening by ultrasound of the fetus. If microcephaly in utero or fetal brain defects were observed, then a mother would be counseled on her options,” said Dr. Gandhi.

Dr. Silverman noted that “new data on children who were exposed in utero and had normal exams, including head measurements when they were born, have raised concerns. In recently published long-term follow-up studies, even when children born to mothers infected with Zika during pregnancy had normal head growth at least 3 years after birth, they were still at risk for neurodevelopmental delay and behavioral disorders, including impact on coordination and executive function.

“This is another good reason to keep the potential risks of Zika active in the public’s consciousness and in public health planning.”

Dr. Silverman, Dr. Gandhi, and Dr. Staples have disclosed no relevant financial relationships. Dr. Leav is an employee of Moderna and owns stock in the company.

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

The Food and Drug Administration approved elacestrant (Orserdu, Stemline Therapeutics) for postmenopausal women or men with ER-positive, HER2-negative, ESR1-mutated advanced or metastatic breast cancer that progressed on at least one line of endocrine therapy.

The agency also approved the Guardant360 CDx assay as a companion diagnostic to identify breast cancer patients who meet the treatment requirements, according to the agency’s press release announcing the approval.

The novel oral selective estrogen receptor degrader was approved based on the phase 3 EMERALD trial, which included 478 postmenopausal women and men with ER-positive, HER2-negative advanced or metastatic breast cancer, about half of whom had ESR1 mutations. Patients had progressed on one or two prior lines of endocrine therapy, including one containing a CDK4/6 inhibitor. Participants could also have had one prior line of chemotherapy in the advanced or metastatic setting.

Participants were randomized 1:1 to either elacestrant 345 mg orally once daily or investigator’s choice of endocrine therapy, which included fulvestrant or an aromatase inhibitor.

In the 228 patients (48%) with ESR1 mutations, median progression-free survival (PFS) was 3.8 months with elacestrant versus 1.9 months in the fulvestrant or aromatase inhibitor arm (hazard ratio, 0.55; P = .0005). Investigators observed no statistically significant PFS difference between the treatment arms in patients who didn’t have the mutation.
 

Fair comparison?

In June, experts raised concerns about the adequacy of the “standard of care” control arm in EMERALD, particularly that single agents were used at a time when combination therapy is becoming more common.

“The expression ‘standard of care’ is applied generously, as the control arm is restricted” to single agents and no combinations, which “may have led to a substandard” comparison group, Timothée Olivier, MD, Geneva University Hospital, and Vinay Prasad, MD, MPH, University of California, San Francisco, said in an editorial quoted in the piece.

EMERALD investigators acknowledged that there were issues with the control group, noting that in the “United States and Europe, combination therapy with fulvestrant” – instead of single agents – “is increasingly being used as the second-line [standard of care] treatment.”

However, the goal of the study “was to compare a novel endocrine therapy vs. currently available endocrine therapies,” not combination regimens, the investigators said.

Also, “the benefit of elacestrant over fulvestrant and AIs [aromatase inhibitors] in our monotherapy trial ... suggests that incorporating elacestrant as the preferred endocrine therapy backbone in future earlier-line combination studies is a promising strategy.”
 

Lipid monitoring necessary

The most common adverse events with elacestrant, occurring in 10% or more of patients, are musculoskeletal pain, nausea, increased cholesterol, increased AST, increased triglycerides, fatigue, decreased hemoglobin, vomiting, increased ALT, decreased sodium, increased creatinine, decreased appetite, diarrhea, headache, constipation, abdominal pain, hot flush, and dyspepsia, according to labeling.

Labeling warns that elacestrant “may cause hypercholesterolemia and hypertriglyceridemia. Monitor lipid profile prior to starting treatment and periodically thereafter.”

The recommended elacestrant dose is the trial dose, 345 mg orally with food once daily until disease progression or unacceptable toxicity.

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

The Food and Drug Administration approved elacestrant (Orserdu, Stemline Therapeutics) for postmenopausal women or men with ER-positive, HER2-negative, ESR1-mutated advanced or metastatic breast cancer that progressed on at least one line of endocrine therapy.

The agency also approved the Guardant360 CDx assay as a companion diagnostic to identify breast cancer patients who meet the treatment requirements, according to the agency’s press release announcing the approval.

The novel oral selective estrogen receptor degrader was approved based on the phase 3 EMERALD trial, which included 478 postmenopausal women and men with ER-positive, HER2-negative advanced or metastatic breast cancer, about half of whom had ESR1 mutations. Patients had progressed on one or two prior lines of endocrine therapy, including one containing a CDK4/6 inhibitor. Participants could also have had one prior line of chemotherapy in the advanced or metastatic setting.

Participants were randomized 1:1 to either elacestrant 345 mg orally once daily or investigator’s choice of endocrine therapy, which included fulvestrant or an aromatase inhibitor.

In the 228 patients (48%) with ESR1 mutations, median progression-free survival (PFS) was 3.8 months with elacestrant versus 1.9 months in the fulvestrant or aromatase inhibitor arm (hazard ratio, 0.55; P = .0005). Investigators observed no statistically significant PFS difference between the treatment arms in patients who didn’t have the mutation.
 

Fair comparison?

In June, experts raised concerns about the adequacy of the “standard of care” control arm in EMERALD, particularly that single agents were used at a time when combination therapy is becoming more common.

“The expression ‘standard of care’ is applied generously, as the control arm is restricted” to single agents and no combinations, which “may have led to a substandard” comparison group, Timothée Olivier, MD, Geneva University Hospital, and Vinay Prasad, MD, MPH, University of California, San Francisco, said in an editorial quoted in the piece.

EMERALD investigators acknowledged that there were issues with the control group, noting that in the “United States and Europe, combination therapy with fulvestrant” – instead of single agents – “is increasingly being used as the second-line [standard of care] treatment.”

However, the goal of the study “was to compare a novel endocrine therapy vs. currently available endocrine therapies,” not combination regimens, the investigators said.

Also, “the benefit of elacestrant over fulvestrant and AIs [aromatase inhibitors] in our monotherapy trial ... suggests that incorporating elacestrant as the preferred endocrine therapy backbone in future earlier-line combination studies is a promising strategy.”
 

Lipid monitoring necessary

The most common adverse events with elacestrant, occurring in 10% or more of patients, are musculoskeletal pain, nausea, increased cholesterol, increased AST, increased triglycerides, fatigue, decreased hemoglobin, vomiting, increased ALT, decreased sodium, increased creatinine, decreased appetite, diarrhea, headache, constipation, abdominal pain, hot flush, and dyspepsia, according to labeling.

Labeling warns that elacestrant “may cause hypercholesterolemia and hypertriglyceridemia. Monitor lipid profile prior to starting treatment and periodically thereafter.”

The recommended elacestrant dose is the trial dose, 345 mg orally with food once daily until disease progression or unacceptable toxicity.

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

The Food and Drug Administration approved elacestrant (Orserdu, Stemline Therapeutics) for postmenopausal women or men with ER-positive, HER2-negative, ESR1-mutated advanced or metastatic breast cancer that progressed on at least one line of endocrine therapy.

The agency also approved the Guardant360 CDx assay as a companion diagnostic to identify breast cancer patients who meet the treatment requirements, according to the agency’s press release announcing the approval.

The novel oral selective estrogen receptor degrader was approved based on the phase 3 EMERALD trial, which included 478 postmenopausal women and men with ER-positive, HER2-negative advanced or metastatic breast cancer, about half of whom had ESR1 mutations. Patients had progressed on one or two prior lines of endocrine therapy, including one containing a CDK4/6 inhibitor. Participants could also have had one prior line of chemotherapy in the advanced or metastatic setting.

Participants were randomized 1:1 to either elacestrant 345 mg orally once daily or investigator’s choice of endocrine therapy, which included fulvestrant or an aromatase inhibitor.

In the 228 patients (48%) with ESR1 mutations, median progression-free survival (PFS) was 3.8 months with elacestrant versus 1.9 months in the fulvestrant or aromatase inhibitor arm (hazard ratio, 0.55; P = .0005). Investigators observed no statistically significant PFS difference between the treatment arms in patients who didn’t have the mutation.
 

Fair comparison?

In June, experts raised concerns about the adequacy of the “standard of care” control arm in EMERALD, particularly that single agents were used at a time when combination therapy is becoming more common.

“The expression ‘standard of care’ is applied generously, as the control arm is restricted” to single agents and no combinations, which “may have led to a substandard” comparison group, Timothée Olivier, MD, Geneva University Hospital, and Vinay Prasad, MD, MPH, University of California, San Francisco, said in an editorial quoted in the piece.

EMERALD investigators acknowledged that there were issues with the control group, noting that in the “United States and Europe, combination therapy with fulvestrant” – instead of single agents – “is increasingly being used as the second-line [standard of care] treatment.”

However, the goal of the study “was to compare a novel endocrine therapy vs. currently available endocrine therapies,” not combination regimens, the investigators said.

Also, “the benefit of elacestrant over fulvestrant and AIs [aromatase inhibitors] in our monotherapy trial ... suggests that incorporating elacestrant as the preferred endocrine therapy backbone in future earlier-line combination studies is a promising strategy.”
 

Lipid monitoring necessary

The most common adverse events with elacestrant, occurring in 10% or more of patients, are musculoskeletal pain, nausea, increased cholesterol, increased AST, increased triglycerides, fatigue, decreased hemoglobin, vomiting, increased ALT, decreased sodium, increased creatinine, decreased appetite, diarrhea, headache, constipation, abdominal pain, hot flush, and dyspepsia, according to labeling.

Labeling warns that elacestrant “may cause hypercholesterolemia and hypertriglyceridemia. Monitor lipid profile prior to starting treatment and periodically thereafter.”

The recommended elacestrant dose is the trial dose, 345 mg orally with food once daily until disease progression or unacceptable toxicity.

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