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FDA OKs odevixibat for pruritus associated with rare liver disease
The U.S. Food and Drug Administration has approved odevixibat (Bylvay, Albireo Pharma), the first treatment for pruritus associated with all types of progressive familial intrahepatic cholestasis (PFIC).
PFIC is a rare disorder affecting an estimated one to two people per 100,000. The disorder usually appears within the first few months of life and causes progressive, life-threatening liver disease, often leading to cirrhosis and liver failure before age 10.
In PFIC, liver cells are unable to drain bile acids, leading to the buildup of toxic substances in the liver. While the precise cause of severe itching in patients with PFIC is unknown, it may involve increased levels of bile acids in the body and skin.
Odevixibat is a potent, nonsystemic ileal bile acid transport inhibitor that does not need refrigeration and is given as a once-daily capsule or opened and sprinkled onto soft foods, the company said in a news release announcing the approval.
There are at least three types of PFIC; all are inherited genetic conditions caused by gene mutations. Odevixibat is indicated to treat all subtypes.
“Treating children with PFIC can be difficult and frustrating given the current treatment options. Bylvay gives us a nonsurgical option and will change how we treat PFIC,” Richard Thompson, MD, principal investigator for the two trials that led to the approval, said in the news release.
“With this approval, my colleagues and I now have the opportunity to revisit how PFIC patients are being managed, and we are hopeful for better outcomes for these children,” said Dr. Thompson, professor of molecular hepatology at King’s College London.
The approval of odevixibat was supported by data from the PEDFIC 1 and PEDFIC 2 trials.
PEDFIC 1 enrolled 62 children with PFIC and severe itching, with 20 assigned to placebo and 42 to odevixibat, given once daily with a meal in the morning. Odevixibat met both of its primary endpoints, with the drug improving pruritus (P = .004) and reducing serum bile acid responses (P = .003).
In PEDFIC 2, a long-term, open-label extension study, the effects of odevixibat on pruritis and serum bile acids were sustained up to 48 weeks.
Odevixibat was well tolerated in both trials, with the most common treatment-related gastrointestinal adverse events being diarrhea/frequent stools. There were no serious treatment-related adverse events.
Children taking the drug should undergo liver test monitoring periodically during treatment, the FDA said when announcing the approval. Odevixibat may affect absorption of fat-soluble vitamins such as A, D, E, and K. Patients should be monitored for fat-soluble vitamin deficiency while taking the drug.
Full prescribing information is available online.
“Until now, invasive surgery was the only approved treatment option. With the approval of Bylvay, parents may find hope in having a less invasive treatment option available,” Emily Ventura, leader of the PFIC Advocacy and Resource Network and mother to a child with PFIC, said in the news release.
The company said it will launch odevixibat “immediately” to accelerate availability for patients and families affected by PFIC.
Odevixibat is also being studied in other rare pediatric cholestatic liver diseases, including biliary atresia and Alagille syndrome.
A version of this article first appeared on Medscape.com.
The U.S. Food and Drug Administration has approved odevixibat (Bylvay, Albireo Pharma), the first treatment for pruritus associated with all types of progressive familial intrahepatic cholestasis (PFIC).
PFIC is a rare disorder affecting an estimated one to two people per 100,000. The disorder usually appears within the first few months of life and causes progressive, life-threatening liver disease, often leading to cirrhosis and liver failure before age 10.
In PFIC, liver cells are unable to drain bile acids, leading to the buildup of toxic substances in the liver. While the precise cause of severe itching in patients with PFIC is unknown, it may involve increased levels of bile acids in the body and skin.
Odevixibat is a potent, nonsystemic ileal bile acid transport inhibitor that does not need refrigeration and is given as a once-daily capsule or opened and sprinkled onto soft foods, the company said in a news release announcing the approval.
There are at least three types of PFIC; all are inherited genetic conditions caused by gene mutations. Odevixibat is indicated to treat all subtypes.
“Treating children with PFIC can be difficult and frustrating given the current treatment options. Bylvay gives us a nonsurgical option and will change how we treat PFIC,” Richard Thompson, MD, principal investigator for the two trials that led to the approval, said in the news release.
“With this approval, my colleagues and I now have the opportunity to revisit how PFIC patients are being managed, and we are hopeful for better outcomes for these children,” said Dr. Thompson, professor of molecular hepatology at King’s College London.
The approval of odevixibat was supported by data from the PEDFIC 1 and PEDFIC 2 trials.
PEDFIC 1 enrolled 62 children with PFIC and severe itching, with 20 assigned to placebo and 42 to odevixibat, given once daily with a meal in the morning. Odevixibat met both of its primary endpoints, with the drug improving pruritus (P = .004) and reducing serum bile acid responses (P = .003).
In PEDFIC 2, a long-term, open-label extension study, the effects of odevixibat on pruritis and serum bile acids were sustained up to 48 weeks.
Odevixibat was well tolerated in both trials, with the most common treatment-related gastrointestinal adverse events being diarrhea/frequent stools. There were no serious treatment-related adverse events.
Children taking the drug should undergo liver test monitoring periodically during treatment, the FDA said when announcing the approval. Odevixibat may affect absorption of fat-soluble vitamins such as A, D, E, and K. Patients should be monitored for fat-soluble vitamin deficiency while taking the drug.
Full prescribing information is available online.
“Until now, invasive surgery was the only approved treatment option. With the approval of Bylvay, parents may find hope in having a less invasive treatment option available,” Emily Ventura, leader of the PFIC Advocacy and Resource Network and mother to a child with PFIC, said in the news release.
The company said it will launch odevixibat “immediately” to accelerate availability for patients and families affected by PFIC.
Odevixibat is also being studied in other rare pediatric cholestatic liver diseases, including biliary atresia and Alagille syndrome.
A version of this article first appeared on Medscape.com.
The U.S. Food and Drug Administration has approved odevixibat (Bylvay, Albireo Pharma), the first treatment for pruritus associated with all types of progressive familial intrahepatic cholestasis (PFIC).
PFIC is a rare disorder affecting an estimated one to two people per 100,000. The disorder usually appears within the first few months of life and causes progressive, life-threatening liver disease, often leading to cirrhosis and liver failure before age 10.
In PFIC, liver cells are unable to drain bile acids, leading to the buildup of toxic substances in the liver. While the precise cause of severe itching in patients with PFIC is unknown, it may involve increased levels of bile acids in the body and skin.
Odevixibat is a potent, nonsystemic ileal bile acid transport inhibitor that does not need refrigeration and is given as a once-daily capsule or opened and sprinkled onto soft foods, the company said in a news release announcing the approval.
There are at least three types of PFIC; all are inherited genetic conditions caused by gene mutations. Odevixibat is indicated to treat all subtypes.
“Treating children with PFIC can be difficult and frustrating given the current treatment options. Bylvay gives us a nonsurgical option and will change how we treat PFIC,” Richard Thompson, MD, principal investigator for the two trials that led to the approval, said in the news release.
“With this approval, my colleagues and I now have the opportunity to revisit how PFIC patients are being managed, and we are hopeful for better outcomes for these children,” said Dr. Thompson, professor of molecular hepatology at King’s College London.
The approval of odevixibat was supported by data from the PEDFIC 1 and PEDFIC 2 trials.
PEDFIC 1 enrolled 62 children with PFIC and severe itching, with 20 assigned to placebo and 42 to odevixibat, given once daily with a meal in the morning. Odevixibat met both of its primary endpoints, with the drug improving pruritus (P = .004) and reducing serum bile acid responses (P = .003).
In PEDFIC 2, a long-term, open-label extension study, the effects of odevixibat on pruritis and serum bile acids were sustained up to 48 weeks.
Odevixibat was well tolerated in both trials, with the most common treatment-related gastrointestinal adverse events being diarrhea/frequent stools. There were no serious treatment-related adverse events.
Children taking the drug should undergo liver test monitoring periodically during treatment, the FDA said when announcing the approval. Odevixibat may affect absorption of fat-soluble vitamins such as A, D, E, and K. Patients should be monitored for fat-soluble vitamin deficiency while taking the drug.
Full prescribing information is available online.
“Until now, invasive surgery was the only approved treatment option. With the approval of Bylvay, parents may find hope in having a less invasive treatment option available,” Emily Ventura, leader of the PFIC Advocacy and Resource Network and mother to a child with PFIC, said in the news release.
The company said it will launch odevixibat “immediately” to accelerate availability for patients and families affected by PFIC.
Odevixibat is also being studied in other rare pediatric cholestatic liver diseases, including biliary atresia and Alagille syndrome.
A version of this article first appeared on Medscape.com.
FDA OKs spinal cord stimulation for diabetic neuropathy pain
The Food and Drug Administration has approved the first high-frequency spinal cord stimulation (SCS) therapy for treating painful diabetic neuropathy (PDN).
The approval is specific for the treatment of chronic pain associated with PDN using the Nevro’s Senza System with 10 kHz stimulation. It is intended for patients whose pain is refractory to, or who can’t tolerate, conventional medical treatment. According to the company, there are currently about 2.3 million individuals with refractory PDN in the United States.
The 10 kHz device, called HFX, involves minimally invasive epidural implantation of the stimulator device, which delivers mild electrical impulses to the nerves to interrupt pain signal to the brain. Such spinal cord stimulation “is a straightforward, well-established treatment for chronic pain that’s been used for over 30 years,” according to the company, although this is the first approval of the modality specifically for PDN.
Asked to comment, Rodica Pop-Busui MD, PhD, the Larry D. Soderquist Professor in Diabetes at the University of Michigan, Ann Arbor, said that “the approval of the Nevro 10kHz high-frequency spinal cord stimulation to treat pain associated with diabetic neuropathy has the potential for benefit for many patients with diabetes and painful diabetic peripheral neuropathy.”
She noted that, “although there are several other pharmacological agents that currently carry the FDA approval for PDN, this is a condition that is notoriously difficult to treat, particularly when taking into account the actual number needed to treat with a specific agent to achieve a clinically meaningful pain reduction, as well as the spectrum of side effects and drug-drug interactions in a patient population that require many other additional agents to manage diabetes and comorbidities on a daily basis. Thus, this new therapeutic approach besides effective pain reduction has the additional benefit of bypassing drug interactions.” Dr. Pop-Busui was the lead author on the American Diabetes Association’s 2017 position statement on diabetic neuropathy.
She also cautioned, on the other hand, that “it is not very clear yet how easy it will be for all eligible patients to have access to this technology, what will be the actual costs, the insurance coverage, or the acceptance by patients across various sociodemographic backgrounds from the at-large clinical care. However, given the challenges we encounter to treat diabetic neuropathy and particularly the pain associated with it, it is quite encouraging to see that the tools available to help our patients are now broader.”
Both 6-and 12-month results show benefit
The FDA approval was based on 6-month data from a prospective, multicenter, open-label randomized clinical trial published in JAMA Neurology.
Use of the 10-kHz SCS device was compared with conventional treatment alone in 216 patients with PDN refractory to gabapentinoids and at least one other analgesic class and lower limb pain intensity of 5 cm or more on a 10-cm visual analog scale.
The primary endpoint, percentage of participants reporting 50% pain relief or more without worsening of baseline neurologic deficits at 3 months, was met by 5 of 94 (5%) patients in the conventional group, compared with 75 of 95 (79%) with the 10-kHz SCS plus conventional treatment (P < .001).
Infections requiring device explant occurred in two patients in the 10-kHz SCS group (2%).
At 12 months, those in the original SCS group plus 86% of subjects given the option to cross over from the conventional treatment group showed “clear and sustained” benefits of the 10-kHz SCS with regard to lower-limb pain, pain interference with daily living, sleep quality, and activity, Erika Petersen, MD, director of the section of functional and restorative neurosurgery at the University of Arkansas for Medical Sciences, Little Rock , reported at the 2021 annual scientific sessions of the ADA.
Infection was the most common study-related adverse event, affecting 8 of 154 patients with the SCS implants (5.2%). Three resolved with conservative treatment and five (3.2%) required removal of the device.
The patients will be followed for a total of 24 months.
Commercial launch of HFX in the United States will begin immediately, the company said.
Dr. Pop-Busui has received consultant fees in the last 12 months from Averitas Pharma, Boehringer Ingelheim, Nevro, and Novo Nordisk. Dr. Petersen has financial relationships with Nevro, Medtronic, and several other neuromodulator makers.
The Food and Drug Administration has approved the first high-frequency spinal cord stimulation (SCS) therapy for treating painful diabetic neuropathy (PDN).
The approval is specific for the treatment of chronic pain associated with PDN using the Nevro’s Senza System with 10 kHz stimulation. It is intended for patients whose pain is refractory to, or who can’t tolerate, conventional medical treatment. According to the company, there are currently about 2.3 million individuals with refractory PDN in the United States.
The 10 kHz device, called HFX, involves minimally invasive epidural implantation of the stimulator device, which delivers mild electrical impulses to the nerves to interrupt pain signal to the brain. Such spinal cord stimulation “is a straightforward, well-established treatment for chronic pain that’s been used for over 30 years,” according to the company, although this is the first approval of the modality specifically for PDN.
Asked to comment, Rodica Pop-Busui MD, PhD, the Larry D. Soderquist Professor in Diabetes at the University of Michigan, Ann Arbor, said that “the approval of the Nevro 10kHz high-frequency spinal cord stimulation to treat pain associated with diabetic neuropathy has the potential for benefit for many patients with diabetes and painful diabetic peripheral neuropathy.”
She noted that, “although there are several other pharmacological agents that currently carry the FDA approval for PDN, this is a condition that is notoriously difficult to treat, particularly when taking into account the actual number needed to treat with a specific agent to achieve a clinically meaningful pain reduction, as well as the spectrum of side effects and drug-drug interactions in a patient population that require many other additional agents to manage diabetes and comorbidities on a daily basis. Thus, this new therapeutic approach besides effective pain reduction has the additional benefit of bypassing drug interactions.” Dr. Pop-Busui was the lead author on the American Diabetes Association’s 2017 position statement on diabetic neuropathy.
She also cautioned, on the other hand, that “it is not very clear yet how easy it will be for all eligible patients to have access to this technology, what will be the actual costs, the insurance coverage, or the acceptance by patients across various sociodemographic backgrounds from the at-large clinical care. However, given the challenges we encounter to treat diabetic neuropathy and particularly the pain associated with it, it is quite encouraging to see that the tools available to help our patients are now broader.”
Both 6-and 12-month results show benefit
The FDA approval was based on 6-month data from a prospective, multicenter, open-label randomized clinical trial published in JAMA Neurology.
Use of the 10-kHz SCS device was compared with conventional treatment alone in 216 patients with PDN refractory to gabapentinoids and at least one other analgesic class and lower limb pain intensity of 5 cm or more on a 10-cm visual analog scale.
The primary endpoint, percentage of participants reporting 50% pain relief or more without worsening of baseline neurologic deficits at 3 months, was met by 5 of 94 (5%) patients in the conventional group, compared with 75 of 95 (79%) with the 10-kHz SCS plus conventional treatment (P < .001).
Infections requiring device explant occurred in two patients in the 10-kHz SCS group (2%).
At 12 months, those in the original SCS group plus 86% of subjects given the option to cross over from the conventional treatment group showed “clear and sustained” benefits of the 10-kHz SCS with regard to lower-limb pain, pain interference with daily living, sleep quality, and activity, Erika Petersen, MD, director of the section of functional and restorative neurosurgery at the University of Arkansas for Medical Sciences, Little Rock , reported at the 2021 annual scientific sessions of the ADA.
Infection was the most common study-related adverse event, affecting 8 of 154 patients with the SCS implants (5.2%). Three resolved with conservative treatment and five (3.2%) required removal of the device.
The patients will be followed for a total of 24 months.
Commercial launch of HFX in the United States will begin immediately, the company said.
Dr. Pop-Busui has received consultant fees in the last 12 months from Averitas Pharma, Boehringer Ingelheim, Nevro, and Novo Nordisk. Dr. Petersen has financial relationships with Nevro, Medtronic, and several other neuromodulator makers.
The Food and Drug Administration has approved the first high-frequency spinal cord stimulation (SCS) therapy for treating painful diabetic neuropathy (PDN).
The approval is specific for the treatment of chronic pain associated with PDN using the Nevro’s Senza System with 10 kHz stimulation. It is intended for patients whose pain is refractory to, or who can’t tolerate, conventional medical treatment. According to the company, there are currently about 2.3 million individuals with refractory PDN in the United States.
The 10 kHz device, called HFX, involves minimally invasive epidural implantation of the stimulator device, which delivers mild electrical impulses to the nerves to interrupt pain signal to the brain. Such spinal cord stimulation “is a straightforward, well-established treatment for chronic pain that’s been used for over 30 years,” according to the company, although this is the first approval of the modality specifically for PDN.
Asked to comment, Rodica Pop-Busui MD, PhD, the Larry D. Soderquist Professor in Diabetes at the University of Michigan, Ann Arbor, said that “the approval of the Nevro 10kHz high-frequency spinal cord stimulation to treat pain associated with diabetic neuropathy has the potential for benefit for many patients with diabetes and painful diabetic peripheral neuropathy.”
She noted that, “although there are several other pharmacological agents that currently carry the FDA approval for PDN, this is a condition that is notoriously difficult to treat, particularly when taking into account the actual number needed to treat with a specific agent to achieve a clinically meaningful pain reduction, as well as the spectrum of side effects and drug-drug interactions in a patient population that require many other additional agents to manage diabetes and comorbidities on a daily basis. Thus, this new therapeutic approach besides effective pain reduction has the additional benefit of bypassing drug interactions.” Dr. Pop-Busui was the lead author on the American Diabetes Association’s 2017 position statement on diabetic neuropathy.
She also cautioned, on the other hand, that “it is not very clear yet how easy it will be for all eligible patients to have access to this technology, what will be the actual costs, the insurance coverage, or the acceptance by patients across various sociodemographic backgrounds from the at-large clinical care. However, given the challenges we encounter to treat diabetic neuropathy and particularly the pain associated with it, it is quite encouraging to see that the tools available to help our patients are now broader.”
Both 6-and 12-month results show benefit
The FDA approval was based on 6-month data from a prospective, multicenter, open-label randomized clinical trial published in JAMA Neurology.
Use of the 10-kHz SCS device was compared with conventional treatment alone in 216 patients with PDN refractory to gabapentinoids and at least one other analgesic class and lower limb pain intensity of 5 cm or more on a 10-cm visual analog scale.
The primary endpoint, percentage of participants reporting 50% pain relief or more without worsening of baseline neurologic deficits at 3 months, was met by 5 of 94 (5%) patients in the conventional group, compared with 75 of 95 (79%) with the 10-kHz SCS plus conventional treatment (P < .001).
Infections requiring device explant occurred in two patients in the 10-kHz SCS group (2%).
At 12 months, those in the original SCS group plus 86% of subjects given the option to cross over from the conventional treatment group showed “clear and sustained” benefits of the 10-kHz SCS with regard to lower-limb pain, pain interference with daily living, sleep quality, and activity, Erika Petersen, MD, director of the section of functional and restorative neurosurgery at the University of Arkansas for Medical Sciences, Little Rock , reported at the 2021 annual scientific sessions of the ADA.
Infection was the most common study-related adverse event, affecting 8 of 154 patients with the SCS implants (5.2%). Three resolved with conservative treatment and five (3.2%) required removal of the device.
The patients will be followed for a total of 24 months.
Commercial launch of HFX in the United States will begin immediately, the company said.
Dr. Pop-Busui has received consultant fees in the last 12 months from Averitas Pharma, Boehringer Ingelheim, Nevro, and Novo Nordisk. Dr. Petersen has financial relationships with Nevro, Medtronic, and several other neuromodulator makers.
FDA approves intravenous immunoglobulin for dermatomyositis
, according to a statement from manufacturer Octapharma USA.
Dermatomyositis is a rare, idiopathic autoimmune disorder that affects approximately 10 out of every million people in the United States, mainly adults in their late 40s to early 60s, according to the company, but children aged 5-15 years can be affected. The disease is characterized by skin rashes, chronic muscle inflammation, progressive muscle weakness, and risk for mortality that is three times higher than for the general population.
There are no previously approved treatments for dermatomyositis prior to Octagam 10%, which also is indicated for chronic immune thrombocytopenic purpura in adults.
The approval for dermatomyositis was based on the results of a phase 3 randomized, double-blind, placebo-controlled clinical trial (the ProDERM trial) that included 95 adult patients at 36 sites worldwide, with 17 sites in the United States. In the trial, 78.7% of patients with dermatomyositis who were randomized to receive 2 g/kg of Octagam 10% every 4 weeks showed response at 16 weeks, compared with 43.8% of patients who received placebo. Response was based on the 2016 American College of Rheumatology/European Alliance of Associations for Rheumatology myositis response criteria. Placebo patients who switched to intravenous immunoglobulin (IVIG) during a trial extension had response rates at week 40 similar to the original patients at week 16.
“The study gives clinicians much more confidence in the efficacy and safety of intravenous immunoglobulin and provides valuable information about what type of patient is best suited for the treatment,” Rohit Aggarwal, MD, medical director of the Arthritis and Autoimmunity Center at the University of Pittsburgh and a member of the ProDERM study Steering Committee, said in the Octapharma statement.
Safety and tolerability were similar to profiles seen with other IVIG medications, according to the statement. The medication does carry a boxed warning from its chronic ITP approval, cautioning about the potential for thrombosis, renal dysfunction, and acute renal failure.
The most common adverse reactions reported by dermatomyositis patients in the ProDERM trial were headache, fever, nausea, vomiting, increased blood pressure, chills, musculoskeletal pain, increased heart rate, dyspnea, and reactions at the infusion sites.
Read the full prescribing information here.
, according to a statement from manufacturer Octapharma USA.
Dermatomyositis is a rare, idiopathic autoimmune disorder that affects approximately 10 out of every million people in the United States, mainly adults in their late 40s to early 60s, according to the company, but children aged 5-15 years can be affected. The disease is characterized by skin rashes, chronic muscle inflammation, progressive muscle weakness, and risk for mortality that is three times higher than for the general population.
There are no previously approved treatments for dermatomyositis prior to Octagam 10%, which also is indicated for chronic immune thrombocytopenic purpura in adults.
The approval for dermatomyositis was based on the results of a phase 3 randomized, double-blind, placebo-controlled clinical trial (the ProDERM trial) that included 95 adult patients at 36 sites worldwide, with 17 sites in the United States. In the trial, 78.7% of patients with dermatomyositis who were randomized to receive 2 g/kg of Octagam 10% every 4 weeks showed response at 16 weeks, compared with 43.8% of patients who received placebo. Response was based on the 2016 American College of Rheumatology/European Alliance of Associations for Rheumatology myositis response criteria. Placebo patients who switched to intravenous immunoglobulin (IVIG) during a trial extension had response rates at week 40 similar to the original patients at week 16.
“The study gives clinicians much more confidence in the efficacy and safety of intravenous immunoglobulin and provides valuable information about what type of patient is best suited for the treatment,” Rohit Aggarwal, MD, medical director of the Arthritis and Autoimmunity Center at the University of Pittsburgh and a member of the ProDERM study Steering Committee, said in the Octapharma statement.
Safety and tolerability were similar to profiles seen with other IVIG medications, according to the statement. The medication does carry a boxed warning from its chronic ITP approval, cautioning about the potential for thrombosis, renal dysfunction, and acute renal failure.
The most common adverse reactions reported by dermatomyositis patients in the ProDERM trial were headache, fever, nausea, vomiting, increased blood pressure, chills, musculoskeletal pain, increased heart rate, dyspnea, and reactions at the infusion sites.
Read the full prescribing information here.
, according to a statement from manufacturer Octapharma USA.
Dermatomyositis is a rare, idiopathic autoimmune disorder that affects approximately 10 out of every million people in the United States, mainly adults in their late 40s to early 60s, according to the company, but children aged 5-15 years can be affected. The disease is characterized by skin rashes, chronic muscle inflammation, progressive muscle weakness, and risk for mortality that is three times higher than for the general population.
There are no previously approved treatments for dermatomyositis prior to Octagam 10%, which also is indicated for chronic immune thrombocytopenic purpura in adults.
The approval for dermatomyositis was based on the results of a phase 3 randomized, double-blind, placebo-controlled clinical trial (the ProDERM trial) that included 95 adult patients at 36 sites worldwide, with 17 sites in the United States. In the trial, 78.7% of patients with dermatomyositis who were randomized to receive 2 g/kg of Octagam 10% every 4 weeks showed response at 16 weeks, compared with 43.8% of patients who received placebo. Response was based on the 2016 American College of Rheumatology/European Alliance of Associations for Rheumatology myositis response criteria. Placebo patients who switched to intravenous immunoglobulin (IVIG) during a trial extension had response rates at week 40 similar to the original patients at week 16.
“The study gives clinicians much more confidence in the efficacy and safety of intravenous immunoglobulin and provides valuable information about what type of patient is best suited for the treatment,” Rohit Aggarwal, MD, medical director of the Arthritis and Autoimmunity Center at the University of Pittsburgh and a member of the ProDERM study Steering Committee, said in the Octapharma statement.
Safety and tolerability were similar to profiles seen with other IVIG medications, according to the statement. The medication does carry a boxed warning from its chronic ITP approval, cautioning about the potential for thrombosis, renal dysfunction, and acute renal failure.
The most common adverse reactions reported by dermatomyositis patients in the ProDERM trial were headache, fever, nausea, vomiting, increased blood pressure, chills, musculoskeletal pain, increased heart rate, dyspnea, and reactions at the infusion sites.
Read the full prescribing information here.
FDA to revise statin pregnancy contraindication
The U.S. Food and Drug Administration (FDA) aims to update the labeling on all statins to remove the drugs’ blanket contraindication in all pregnant patients, the agency has announced. The change should reinforce for both physicians and patients that statin use in women with unrecognized pregnancy is unlikely to be harmful, it said.
“Because the benefits of statins may include prevention of serious or potentially fatal events in a small group of very high-risk pregnant patients, contraindicating these drugs in all pregnant women is not appropriate.”
The revision should emphasize for clinicians “that statins are safe to prescribe in patients who can become pregnant and help them reassure patients with unintended statin exposure in early pregnancy,” the FDA explained.
Removal of the broadly worded contraindication should “enable health care professionals and patients to make individual decisions about benefit and risk, especially for those at very high risk of heart attack or stroke." That includes women with homozygous familial hypercholesterolemia and those who are prescribed statins for secondary prevention, the agency said.
Clinicians “should discontinue statin therapy in most pregnant patients, or they can consider the ongoing therapeutic needs of the individual patient, particularly those at very high risk for cardiovascular events during pregnancy. Because of the chronic nature of cardiovascular disease, treatment of hyperlipidemia is not generally necessary during pregnancy.”
A version of this article first appeared on Medscape.com.
The U.S. Food and Drug Administration (FDA) aims to update the labeling on all statins to remove the drugs’ blanket contraindication in all pregnant patients, the agency has announced. The change should reinforce for both physicians and patients that statin use in women with unrecognized pregnancy is unlikely to be harmful, it said.
“Because the benefits of statins may include prevention of serious or potentially fatal events in a small group of very high-risk pregnant patients, contraindicating these drugs in all pregnant women is not appropriate.”
The revision should emphasize for clinicians “that statins are safe to prescribe in patients who can become pregnant and help them reassure patients with unintended statin exposure in early pregnancy,” the FDA explained.
Removal of the broadly worded contraindication should “enable health care professionals and patients to make individual decisions about benefit and risk, especially for those at very high risk of heart attack or stroke." That includes women with homozygous familial hypercholesterolemia and those who are prescribed statins for secondary prevention, the agency said.
Clinicians “should discontinue statin therapy in most pregnant patients, or they can consider the ongoing therapeutic needs of the individual patient, particularly those at very high risk for cardiovascular events during pregnancy. Because of the chronic nature of cardiovascular disease, treatment of hyperlipidemia is not generally necessary during pregnancy.”
A version of this article first appeared on Medscape.com.
The U.S. Food and Drug Administration (FDA) aims to update the labeling on all statins to remove the drugs’ blanket contraindication in all pregnant patients, the agency has announced. The change should reinforce for both physicians and patients that statin use in women with unrecognized pregnancy is unlikely to be harmful, it said.
“Because the benefits of statins may include prevention of serious or potentially fatal events in a small group of very high-risk pregnant patients, contraindicating these drugs in all pregnant women is not appropriate.”
The revision should emphasize for clinicians “that statins are safe to prescribe in patients who can become pregnant and help them reassure patients with unintended statin exposure in early pregnancy,” the FDA explained.
Removal of the broadly worded contraindication should “enable health care professionals and patients to make individual decisions about benefit and risk, especially for those at very high risk of heart attack or stroke." That includes women with homozygous familial hypercholesterolemia and those who are prescribed statins for secondary prevention, the agency said.
Clinicians “should discontinue statin therapy in most pregnant patients, or they can consider the ongoing therapeutic needs of the individual patient, particularly those at very high risk for cardiovascular events during pregnancy. Because of the chronic nature of cardiovascular disease, treatment of hyperlipidemia is not generally necessary during pregnancy.”
A version of this article first appeared on Medscape.com.
FDA approves new asparaginase product for leukemia
The new product is Jazz Pharmaceutical’s Rylaze (asparaginase erwinia chrysanthemi [recombinant]-rywn), and it is approved for use in the treatment of acute lymphoblastic leukemia and lymphoblastic lymphoma.
Asparaginase, an enzyme that helps kill blood cancer cells, is a key component of chemotherapy for both conditions.
The problem is that about 20% of patients become allergic to the standard option, asparaginase derived from Escherichia coli.
The only alternative until now has been Erwinaze (also distributed by Jazz Pharmaceuticals), which, like Rylaze, is derived from Erwinia chrysanthemi, a plant pathogen related to Escherichia coli.
However, Erwinaze has been bedeviled by manufacturing problems and has been in short supply since 2016.
The situation has been “extremely disconcerting to patients, families and providers,” and the hope is that Rylaze will “provide a consistently sourced alternative,” Gregory Reaman, MD, the FDA’s associate director of pediatric oncology, said in a press release.
Rylaze will hit the U.S. market in mid-July. Jazz has been a distributor of Erwinaze as well, but it released its last batch in May, according to a spokesperson.
The key difference between the two products is that the asparaginase in Erwinaze is derived directly from Erwinia chrysanthemi, whereas the asparaginase in Rylaze is a recombinant product produced by different bacteria that have been genetically altered with Erwinia chrysanthemi DNA.
The approval for Rylaze was based on a study involving 102 patients (median age, 10 years) who developed hypersensitivity to Escherichia coli–derived enzyme or “silent inactivation” from neutralizing antibodies. In the study, almost 94% of patients achieved asparaginase target activity levels at the approved dosage of 25 mg/m2 IM every 48 hours. The study is ongoing, and investigators are currently evaluating intravenous dosing.
The most common side effects are hypersensitivity reactions, blood clots, hemorrhage, and pancreatic and liver toxicity. There is also a risk for fetal harm, so labeling advises women to use effective nonhormonal contraception during treatment and for 3 months afterward.
A version of this article first appeared on Medscape.com.
The new product is Jazz Pharmaceutical’s Rylaze (asparaginase erwinia chrysanthemi [recombinant]-rywn), and it is approved for use in the treatment of acute lymphoblastic leukemia and lymphoblastic lymphoma.
Asparaginase, an enzyme that helps kill blood cancer cells, is a key component of chemotherapy for both conditions.
The problem is that about 20% of patients become allergic to the standard option, asparaginase derived from Escherichia coli.
The only alternative until now has been Erwinaze (also distributed by Jazz Pharmaceuticals), which, like Rylaze, is derived from Erwinia chrysanthemi, a plant pathogen related to Escherichia coli.
However, Erwinaze has been bedeviled by manufacturing problems and has been in short supply since 2016.
The situation has been “extremely disconcerting to patients, families and providers,” and the hope is that Rylaze will “provide a consistently sourced alternative,” Gregory Reaman, MD, the FDA’s associate director of pediatric oncology, said in a press release.
Rylaze will hit the U.S. market in mid-July. Jazz has been a distributor of Erwinaze as well, but it released its last batch in May, according to a spokesperson.
The key difference between the two products is that the asparaginase in Erwinaze is derived directly from Erwinia chrysanthemi, whereas the asparaginase in Rylaze is a recombinant product produced by different bacteria that have been genetically altered with Erwinia chrysanthemi DNA.
The approval for Rylaze was based on a study involving 102 patients (median age, 10 years) who developed hypersensitivity to Escherichia coli–derived enzyme or “silent inactivation” from neutralizing antibodies. In the study, almost 94% of patients achieved asparaginase target activity levels at the approved dosage of 25 mg/m2 IM every 48 hours. The study is ongoing, and investigators are currently evaluating intravenous dosing.
The most common side effects are hypersensitivity reactions, blood clots, hemorrhage, and pancreatic and liver toxicity. There is also a risk for fetal harm, so labeling advises women to use effective nonhormonal contraception during treatment and for 3 months afterward.
A version of this article first appeared on Medscape.com.
The new product is Jazz Pharmaceutical’s Rylaze (asparaginase erwinia chrysanthemi [recombinant]-rywn), and it is approved for use in the treatment of acute lymphoblastic leukemia and lymphoblastic lymphoma.
Asparaginase, an enzyme that helps kill blood cancer cells, is a key component of chemotherapy for both conditions.
The problem is that about 20% of patients become allergic to the standard option, asparaginase derived from Escherichia coli.
The only alternative until now has been Erwinaze (also distributed by Jazz Pharmaceuticals), which, like Rylaze, is derived from Erwinia chrysanthemi, a plant pathogen related to Escherichia coli.
However, Erwinaze has been bedeviled by manufacturing problems and has been in short supply since 2016.
The situation has been “extremely disconcerting to patients, families and providers,” and the hope is that Rylaze will “provide a consistently sourced alternative,” Gregory Reaman, MD, the FDA’s associate director of pediatric oncology, said in a press release.
Rylaze will hit the U.S. market in mid-July. Jazz has been a distributor of Erwinaze as well, but it released its last batch in May, according to a spokesperson.
The key difference between the two products is that the asparaginase in Erwinaze is derived directly from Erwinia chrysanthemi, whereas the asparaginase in Rylaze is a recombinant product produced by different bacteria that have been genetically altered with Erwinia chrysanthemi DNA.
The approval for Rylaze was based on a study involving 102 patients (median age, 10 years) who developed hypersensitivity to Escherichia coli–derived enzyme or “silent inactivation” from neutralizing antibodies. In the study, almost 94% of patients achieved asparaginase target activity levels at the approved dosage of 25 mg/m2 IM every 48 hours. The study is ongoing, and investigators are currently evaluating intravenous dosing.
The most common side effects are hypersensitivity reactions, blood clots, hemorrhage, and pancreatic and liver toxicity. There is also a risk for fetal harm, so labeling advises women to use effective nonhormonal contraception during treatment and for 3 months afterward.
A version of this article first appeared on Medscape.com.
FDA warns of potential mechanical concerns with MAGEC devices
MAGEC is a surgical magnetic rod system used to treat early-onset scoliosis (EOS) in children under 10 years of age. The magnetic system can help avoid invasive surgeries, as growth rods can be adjusted with an external remote control. MAGEC is the only FDA-approved pure distraction-based system for EOS and is the most-used technology for EOS treatment in the United States, Aakash Agarwal, PhD, director of research and clinical affairs at Spinal Balance in Swanton, Ohio, said in an interview.
According to the notice, there are reports of endcap separation and O-ring seal failure in the following six MAGEC devices:
- MAGEC Spinal Bracing and Distraction System
- MAGEC 2 Spinal Bracing and Distraction System
- MAGEC System
- MAGEC System Model X Device
- MAGEC System Model X Rod
- MAGEC System Rods
Endcap separation can potentially expose the patient’s tissue to internal components of the device that have not been completely tested for biocompatibility.
In February 2020, NuVasive recalled its MAGEC System Model X rods to address reports of endcap separation issues. The FDA cleared a modified version of the device designed to mitigate these events in July 2020. In April 2021, NuVasive informed providers of potential biocompatibility concerns and placed a voluntary shipping hold on the MAGEC device system. The shipping hold was lifted July 15, the company announced.
The FDA is currently not recommending removal of functioning MAGEC devices, noting that it is “in the best interest of patients” to continue to make the system available. The overall benefits of the device outweigh the known risks, and the restricted use for a 2-year implantation time for children under 10 years of age will further mitigate these risks, the FDA said in the statement.
To report adverse events related to MAGEC devices, patients, caregivers, and providers can submit a report through MedWatch, the FDA safety information and adverse event reporting program.
A version of this article first appeared on Medscape.com.
MAGEC is a surgical magnetic rod system used to treat early-onset scoliosis (EOS) in children under 10 years of age. The magnetic system can help avoid invasive surgeries, as growth rods can be adjusted with an external remote control. MAGEC is the only FDA-approved pure distraction-based system for EOS and is the most-used technology for EOS treatment in the United States, Aakash Agarwal, PhD, director of research and clinical affairs at Spinal Balance in Swanton, Ohio, said in an interview.
According to the notice, there are reports of endcap separation and O-ring seal failure in the following six MAGEC devices:
- MAGEC Spinal Bracing and Distraction System
- MAGEC 2 Spinal Bracing and Distraction System
- MAGEC System
- MAGEC System Model X Device
- MAGEC System Model X Rod
- MAGEC System Rods
Endcap separation can potentially expose the patient’s tissue to internal components of the device that have not been completely tested for biocompatibility.
In February 2020, NuVasive recalled its MAGEC System Model X rods to address reports of endcap separation issues. The FDA cleared a modified version of the device designed to mitigate these events in July 2020. In April 2021, NuVasive informed providers of potential biocompatibility concerns and placed a voluntary shipping hold on the MAGEC device system. The shipping hold was lifted July 15, the company announced.
The FDA is currently not recommending removal of functioning MAGEC devices, noting that it is “in the best interest of patients” to continue to make the system available. The overall benefits of the device outweigh the known risks, and the restricted use for a 2-year implantation time for children under 10 years of age will further mitigate these risks, the FDA said in the statement.
To report adverse events related to MAGEC devices, patients, caregivers, and providers can submit a report through MedWatch, the FDA safety information and adverse event reporting program.
A version of this article first appeared on Medscape.com.
MAGEC is a surgical magnetic rod system used to treat early-onset scoliosis (EOS) in children under 10 years of age. The magnetic system can help avoid invasive surgeries, as growth rods can be adjusted with an external remote control. MAGEC is the only FDA-approved pure distraction-based system for EOS and is the most-used technology for EOS treatment in the United States, Aakash Agarwal, PhD, director of research and clinical affairs at Spinal Balance in Swanton, Ohio, said in an interview.
According to the notice, there are reports of endcap separation and O-ring seal failure in the following six MAGEC devices:
- MAGEC Spinal Bracing and Distraction System
- MAGEC 2 Spinal Bracing and Distraction System
- MAGEC System
- MAGEC System Model X Device
- MAGEC System Model X Rod
- MAGEC System Rods
Endcap separation can potentially expose the patient’s tissue to internal components of the device that have not been completely tested for biocompatibility.
In February 2020, NuVasive recalled its MAGEC System Model X rods to address reports of endcap separation issues. The FDA cleared a modified version of the device designed to mitigate these events in July 2020. In April 2021, NuVasive informed providers of potential biocompatibility concerns and placed a voluntary shipping hold on the MAGEC device system. The shipping hold was lifted July 15, the company announced.
The FDA is currently not recommending removal of functioning MAGEC devices, noting that it is “in the best interest of patients” to continue to make the system available. The overall benefits of the device outweigh the known risks, and the restricted use for a 2-year implantation time for children under 10 years of age will further mitigate these risks, the FDA said in the statement.
To report adverse events related to MAGEC devices, patients, caregivers, and providers can submit a report through MedWatch, the FDA safety information and adverse event reporting program.
A version of this article first appeared on Medscape.com.
Latest FDA pembrolizumab approval expands label to cutaneous SCCs
The
The July 6 approval for the programmed death–1 inhibitor follows a June FDA approval for pembrolizumab monotherapy in patients with recurrent or metastatic cSCC disease not curable by surgery or radiation. Both approvals, pembrolizumab’s first for cSCC, are based on findings from the second interim analysis of the phase 2, multicenter, open-label KEYNOTE-629 trial.
The objective response rate in the cohort of 54 patients with locally advanced disease was 50%, including a complete response rate of 17% and a partial response rate of 33%. Duration of response was 6 months or longer in 81% of the 27 responders, and 12 months or longer in 37% of responders. After a median follow-up of 13.4 months, median duration of response had not yet been reached.
Pembrolizumab has previously received FDA approvals, either as monotherapy or in combination with other agents, for the treatment of numerous cancer types, including certain melanomas, non–small cell lung cancers, head and neck SCCs, classical Hodgkin lymphomas, primary mediastinal large B-cell lymphomas, urothelial carcinomas, microsatellite instability–high or mismatch repair–deficient cancers, and gastric, esophageal, cervical, hepatocellular, Merkel cell, renal cell, tumor mutational burden–high, and triple-negative breast cancers.
Patients in the KEYNOTE-629 trial received pembrolizumab at a dose of 200 mg IV every 3 weeks for 24 months or until documented disease progression or unacceptable toxicity.
Adverse reactions occurring in patients with recurrent or metastatic cSCC or locally advanced cSCC in KEYNOTE-629 were similar to those observed in patients with melanoma or non–small cell lung cancer who were treated with pembrolizumab monotherapy in previous trials.
The checkpoint inhibitor can cause immune-mediated adverse reactions, which may be severe or fatal, according to Merck, the drug’s manufacturer. The reactions can occur in any organ system or tissue and can affect more than one body system simultaneously.
“Immune-mediated adverse reactions can occur at any time during or after treatment with Keytruda, including pneumonitis, colitis, hepatitis, endocrinopathies, nephritis, dermatologic reactions, solid organ transplant rejection, and complications of allogeneic hematopoietic stem cell transplantation,” Merck explained in a press release, noting that “early identification and management of immune-mediated adverse reactions are essential to ensure safe use of Keytruda.”
Depending on the severity of any reaction, treatment should be withheld or permanently discontinued, and corticosteroids administered if appropriate, Merck stated.
The
The July 6 approval for the programmed death–1 inhibitor follows a June FDA approval for pembrolizumab monotherapy in patients with recurrent or metastatic cSCC disease not curable by surgery or radiation. Both approvals, pembrolizumab’s first for cSCC, are based on findings from the second interim analysis of the phase 2, multicenter, open-label KEYNOTE-629 trial.
The objective response rate in the cohort of 54 patients with locally advanced disease was 50%, including a complete response rate of 17% and a partial response rate of 33%. Duration of response was 6 months or longer in 81% of the 27 responders, and 12 months or longer in 37% of responders. After a median follow-up of 13.4 months, median duration of response had not yet been reached.
Pembrolizumab has previously received FDA approvals, either as monotherapy or in combination with other agents, for the treatment of numerous cancer types, including certain melanomas, non–small cell lung cancers, head and neck SCCs, classical Hodgkin lymphomas, primary mediastinal large B-cell lymphomas, urothelial carcinomas, microsatellite instability–high or mismatch repair–deficient cancers, and gastric, esophageal, cervical, hepatocellular, Merkel cell, renal cell, tumor mutational burden–high, and triple-negative breast cancers.
Patients in the KEYNOTE-629 trial received pembrolizumab at a dose of 200 mg IV every 3 weeks for 24 months or until documented disease progression or unacceptable toxicity.
Adverse reactions occurring in patients with recurrent or metastatic cSCC or locally advanced cSCC in KEYNOTE-629 were similar to those observed in patients with melanoma or non–small cell lung cancer who were treated with pembrolizumab monotherapy in previous trials.
The checkpoint inhibitor can cause immune-mediated adverse reactions, which may be severe or fatal, according to Merck, the drug’s manufacturer. The reactions can occur in any organ system or tissue and can affect more than one body system simultaneously.
“Immune-mediated adverse reactions can occur at any time during or after treatment with Keytruda, including pneumonitis, colitis, hepatitis, endocrinopathies, nephritis, dermatologic reactions, solid organ transplant rejection, and complications of allogeneic hematopoietic stem cell transplantation,” Merck explained in a press release, noting that “early identification and management of immune-mediated adverse reactions are essential to ensure safe use of Keytruda.”
Depending on the severity of any reaction, treatment should be withheld or permanently discontinued, and corticosteroids administered if appropriate, Merck stated.
The
The July 6 approval for the programmed death–1 inhibitor follows a June FDA approval for pembrolizumab monotherapy in patients with recurrent or metastatic cSCC disease not curable by surgery or radiation. Both approvals, pembrolizumab’s first for cSCC, are based on findings from the second interim analysis of the phase 2, multicenter, open-label KEYNOTE-629 trial.
The objective response rate in the cohort of 54 patients with locally advanced disease was 50%, including a complete response rate of 17% and a partial response rate of 33%. Duration of response was 6 months or longer in 81% of the 27 responders, and 12 months or longer in 37% of responders. After a median follow-up of 13.4 months, median duration of response had not yet been reached.
Pembrolizumab has previously received FDA approvals, either as monotherapy or in combination with other agents, for the treatment of numerous cancer types, including certain melanomas, non–small cell lung cancers, head and neck SCCs, classical Hodgkin lymphomas, primary mediastinal large B-cell lymphomas, urothelial carcinomas, microsatellite instability–high or mismatch repair–deficient cancers, and gastric, esophageal, cervical, hepatocellular, Merkel cell, renal cell, tumor mutational burden–high, and triple-negative breast cancers.
Patients in the KEYNOTE-629 trial received pembrolizumab at a dose of 200 mg IV every 3 weeks for 24 months or until documented disease progression or unacceptable toxicity.
Adverse reactions occurring in patients with recurrent or metastatic cSCC or locally advanced cSCC in KEYNOTE-629 were similar to those observed in patients with melanoma or non–small cell lung cancer who were treated with pembrolizumab monotherapy in previous trials.
The checkpoint inhibitor can cause immune-mediated adverse reactions, which may be severe or fatal, according to Merck, the drug’s manufacturer. The reactions can occur in any organ system or tissue and can affect more than one body system simultaneously.
“Immune-mediated adverse reactions can occur at any time during or after treatment with Keytruda, including pneumonitis, colitis, hepatitis, endocrinopathies, nephritis, dermatologic reactions, solid organ transplant rejection, and complications of allogeneic hematopoietic stem cell transplantation,” Merck explained in a press release, noting that “early identification and management of immune-mediated adverse reactions are essential to ensure safe use of Keytruda.”
Depending on the severity of any reaction, treatment should be withheld or permanently discontinued, and corticosteroids administered if appropriate, Merck stated.
FDA to warn J&J that vaccine can increase Guillain-Barré risk: Media
as early as July 13, according to multiple media reports.
Although the FDA is projected to add the new warning to the labeling for the vaccine, the agency still calculates the benefit of vaccination with the J&J product continues to outweigh the risk. Benefits include protection against the Delta variant and serious COVID-19 outcomes.
More than 100 cases of Guillain-Barré reported to the Vaccine Adverse Event Reporting System, a federal program for reporting vaccine issues, spurred the FDA to act.
Men and people older than 50 appear to be at highest risk, according to reports of a July 12 Centers for Disease Control and Prevention statement. The CDC also revealed that most cases occur about 2 weeks following immunization.
Guillain-Barré syndrome often causes muscle weakness and sometimes temporary paralysis. Most people who develop the rare syndrome recover.
Such was not the case for a 57-year-old man, the New York Times reported July 12. He had a history of both a heart attack and stroke in the previous 4 years and died in April after vaccination with the J&J vaccine and developing Guillain-Barré.
The new warning comes in the wake of a number of setbacks for the company’s COVID-19 vaccine. On April 13, the FDA and CDC both recommended a 10-day pause on administration of the J&J vaccine after reports of rare blood clot events emerged. In mid-June, the FDA requested that Johnson and Johnson discard millions of vaccine doses produced at a manufacturing facility in Baltimore.
The mRNA vaccines from Pfizer/BioNTech and Moderna are not affected by the new FDA warning.
The Biden administration is expected to make a formal announcement of the new warning for the Johnson and Johnson vaccine as early as July 13, the Times reports.
A version of this article first appeared on Medscape.com.
as early as July 13, according to multiple media reports.
Although the FDA is projected to add the new warning to the labeling for the vaccine, the agency still calculates the benefit of vaccination with the J&J product continues to outweigh the risk. Benefits include protection against the Delta variant and serious COVID-19 outcomes.
More than 100 cases of Guillain-Barré reported to the Vaccine Adverse Event Reporting System, a federal program for reporting vaccine issues, spurred the FDA to act.
Men and people older than 50 appear to be at highest risk, according to reports of a July 12 Centers for Disease Control and Prevention statement. The CDC also revealed that most cases occur about 2 weeks following immunization.
Guillain-Barré syndrome often causes muscle weakness and sometimes temporary paralysis. Most people who develop the rare syndrome recover.
Such was not the case for a 57-year-old man, the New York Times reported July 12. He had a history of both a heart attack and stroke in the previous 4 years and died in April after vaccination with the J&J vaccine and developing Guillain-Barré.
The new warning comes in the wake of a number of setbacks for the company’s COVID-19 vaccine. On April 13, the FDA and CDC both recommended a 10-day pause on administration of the J&J vaccine after reports of rare blood clot events emerged. In mid-June, the FDA requested that Johnson and Johnson discard millions of vaccine doses produced at a manufacturing facility in Baltimore.
The mRNA vaccines from Pfizer/BioNTech and Moderna are not affected by the new FDA warning.
The Biden administration is expected to make a formal announcement of the new warning for the Johnson and Johnson vaccine as early as July 13, the Times reports.
A version of this article first appeared on Medscape.com.
as early as July 13, according to multiple media reports.
Although the FDA is projected to add the new warning to the labeling for the vaccine, the agency still calculates the benefit of vaccination with the J&J product continues to outweigh the risk. Benefits include protection against the Delta variant and serious COVID-19 outcomes.
More than 100 cases of Guillain-Barré reported to the Vaccine Adverse Event Reporting System, a federal program for reporting vaccine issues, spurred the FDA to act.
Men and people older than 50 appear to be at highest risk, according to reports of a July 12 Centers for Disease Control and Prevention statement. The CDC also revealed that most cases occur about 2 weeks following immunization.
Guillain-Barré syndrome often causes muscle weakness and sometimes temporary paralysis. Most people who develop the rare syndrome recover.
Such was not the case for a 57-year-old man, the New York Times reported July 12. He had a history of both a heart attack and stroke in the previous 4 years and died in April after vaccination with the J&J vaccine and developing Guillain-Barré.
The new warning comes in the wake of a number of setbacks for the company’s COVID-19 vaccine. On April 13, the FDA and CDC both recommended a 10-day pause on administration of the J&J vaccine after reports of rare blood clot events emerged. In mid-June, the FDA requested that Johnson and Johnson discard millions of vaccine doses produced at a manufacturing facility in Baltimore.
The mRNA vaccines from Pfizer/BioNTech and Moderna are not affected by the new FDA warning.
The Biden administration is expected to make a formal announcement of the new warning for the Johnson and Johnson vaccine as early as July 13, the Times reports.
A version of this article first appeared on Medscape.com.
FDA head calls for investigation into agency’s approval of aducanumab (Aduhelm)
After several weeks of outcry and heated debate over the Food and Drug Administration’s controversial approval of the Alzheimer’s drug aducanumab (Aduhelm), the head of the agency is now calling for a federal investigation into its own approval proceedings.
Janet Woodcock, MD, the FDA’s acting commissioner, sent a letter to the Office of the Inspector General on July 9, she announced in a tweet.
Dr. Woodcock is asking for an investigation into questionable meetings and other interactions between Biogen and FDA staff members prior to the drug’s approval that “may have occurred outside of the formal correspondence process.”
The letter explains that concerns around these issues “could undermine the public’s confidence in the FDA’s decision.” Therefore, an independent investigation is needed to determine whether anything occurred that was “inconsistent with FDA policies and procedures.”
Dr. Woodcock noted that she has “tremendous confidence in the integrity of the staff and leadership of the Center for Drug Evaluation and Research” involved in the review process.
However, “FDA is dedicated to scientific integrity, to reviewing data without bias, and to basing its regulatory decisions on data,” she wrote. “You have my personal commitment that the Agency will fully cooperate should your office undertake a review.”
Dr. Woodcock concluded by urging that a review be conducted as soon as possible, noting that “should such a review result in actionable items, you also have my commitment to addressing these issues.”
A version of this article first appeared on Medscape.com.
After several weeks of outcry and heated debate over the Food and Drug Administration’s controversial approval of the Alzheimer’s drug aducanumab (Aduhelm), the head of the agency is now calling for a federal investigation into its own approval proceedings.
Janet Woodcock, MD, the FDA’s acting commissioner, sent a letter to the Office of the Inspector General on July 9, she announced in a tweet.
Dr. Woodcock is asking for an investigation into questionable meetings and other interactions between Biogen and FDA staff members prior to the drug’s approval that “may have occurred outside of the formal correspondence process.”
The letter explains that concerns around these issues “could undermine the public’s confidence in the FDA’s decision.” Therefore, an independent investigation is needed to determine whether anything occurred that was “inconsistent with FDA policies and procedures.”
Dr. Woodcock noted that she has “tremendous confidence in the integrity of the staff and leadership of the Center for Drug Evaluation and Research” involved in the review process.
However, “FDA is dedicated to scientific integrity, to reviewing data without bias, and to basing its regulatory decisions on data,” she wrote. “You have my personal commitment that the Agency will fully cooperate should your office undertake a review.”
Dr. Woodcock concluded by urging that a review be conducted as soon as possible, noting that “should such a review result in actionable items, you also have my commitment to addressing these issues.”
A version of this article first appeared on Medscape.com.
After several weeks of outcry and heated debate over the Food and Drug Administration’s controversial approval of the Alzheimer’s drug aducanumab (Aduhelm), the head of the agency is now calling for a federal investigation into its own approval proceedings.
Janet Woodcock, MD, the FDA’s acting commissioner, sent a letter to the Office of the Inspector General on July 9, she announced in a tweet.
Dr. Woodcock is asking for an investigation into questionable meetings and other interactions between Biogen and FDA staff members prior to the drug’s approval that “may have occurred outside of the formal correspondence process.”
The letter explains that concerns around these issues “could undermine the public’s confidence in the FDA’s decision.” Therefore, an independent investigation is needed to determine whether anything occurred that was “inconsistent with FDA policies and procedures.”
Dr. Woodcock noted that she has “tremendous confidence in the integrity of the staff and leadership of the Center for Drug Evaluation and Research” involved in the review process.
However, “FDA is dedicated to scientific integrity, to reviewing data without bias, and to basing its regulatory decisions on data,” she wrote. “You have my personal commitment that the Agency will fully cooperate should your office undertake a review.”
Dr. Woodcock concluded by urging that a review be conducted as soon as possible, noting that “should such a review result in actionable items, you also have my commitment to addressing these issues.”
A version of this article first appeared on Medscape.com.
‘Long haul’ COVID recovery worse than cancer rehab for some: CDC
People experiencing ongoing or “long-haul” symptoms after COVID-19 illness were more likely to report pain, challenges with physical activities, and “substantially worse health,” compared with people needing rehabilitation because of cancer, lead author Jessica Rogers-Brown, PhD, and colleagues report.
The study was published online July 9 in Morbidity and Mortality Weekly Report (MMWR).
The CDC investigators compared the self-reported physical and mental health symptoms, physical endurance, and use of health services of 1,295 outpatients recovering from COVID-19 and a control group of another 2,395 outpatients rehabilitating from a previous or current cancer diagnosis who had not experienced COVID-19.
Researchers used electronic health record data from January 2020 to March 2021 in the Select Medical network of outpatient clinics. The study included patients from 36 states and the District of Columbia.
Compared with people referred for cancer rehabilitation, those with COVID-19 symptoms lasting beyond 4 weeks were 2.3 times more likely to report pain, 1.8 times more likely to report worse physical health, and 1.6 times more likely to report difficulty with physical activities, an adjusted odds ratio analysis reveals.
The COVID-19 rehabilitation group also performed significantly worse on a 6-minute walk test, suggesting less physical endurance than people recovering from cancer (P < .001). They also used more rehabilitation services overall than the control group.
The researchers suggest services tailored to the unique physical and mental health rehabilitation needs of the post–COVID-19 patient population could be warranted.
The study does not suggest all people recovering with COVID-19 will fare worse than people recovering from cancer, the authors caution. They note that “these results should not be interpreted to mean that post–COVID-19 patients overall had poorer physical and mental health than patients with cancer.”
“Instead, results indicate that post–COVID-19 patients specifically referred to a large physical rehabilitation network had poorer health measures than those referred for cancer, which indicates that some patients recovering from COVID-19 had substantial rehabilitation needs.”
A version of this article first appeared on Medscape.com.
People experiencing ongoing or “long-haul” symptoms after COVID-19 illness were more likely to report pain, challenges with physical activities, and “substantially worse health,” compared with people needing rehabilitation because of cancer, lead author Jessica Rogers-Brown, PhD, and colleagues report.
The study was published online July 9 in Morbidity and Mortality Weekly Report (MMWR).
The CDC investigators compared the self-reported physical and mental health symptoms, physical endurance, and use of health services of 1,295 outpatients recovering from COVID-19 and a control group of another 2,395 outpatients rehabilitating from a previous or current cancer diagnosis who had not experienced COVID-19.
Researchers used electronic health record data from January 2020 to March 2021 in the Select Medical network of outpatient clinics. The study included patients from 36 states and the District of Columbia.
Compared with people referred for cancer rehabilitation, those with COVID-19 symptoms lasting beyond 4 weeks were 2.3 times more likely to report pain, 1.8 times more likely to report worse physical health, and 1.6 times more likely to report difficulty with physical activities, an adjusted odds ratio analysis reveals.
The COVID-19 rehabilitation group also performed significantly worse on a 6-minute walk test, suggesting less physical endurance than people recovering from cancer (P < .001). They also used more rehabilitation services overall than the control group.
The researchers suggest services tailored to the unique physical and mental health rehabilitation needs of the post–COVID-19 patient population could be warranted.
The study does not suggest all people recovering with COVID-19 will fare worse than people recovering from cancer, the authors caution. They note that “these results should not be interpreted to mean that post–COVID-19 patients overall had poorer physical and mental health than patients with cancer.”
“Instead, results indicate that post–COVID-19 patients specifically referred to a large physical rehabilitation network had poorer health measures than those referred for cancer, which indicates that some patients recovering from COVID-19 had substantial rehabilitation needs.”
A version of this article first appeared on Medscape.com.
People experiencing ongoing or “long-haul” symptoms after COVID-19 illness were more likely to report pain, challenges with physical activities, and “substantially worse health,” compared with people needing rehabilitation because of cancer, lead author Jessica Rogers-Brown, PhD, and colleagues report.
The study was published online July 9 in Morbidity and Mortality Weekly Report (MMWR).
The CDC investigators compared the self-reported physical and mental health symptoms, physical endurance, and use of health services of 1,295 outpatients recovering from COVID-19 and a control group of another 2,395 outpatients rehabilitating from a previous or current cancer diagnosis who had not experienced COVID-19.
Researchers used electronic health record data from January 2020 to March 2021 in the Select Medical network of outpatient clinics. The study included patients from 36 states and the District of Columbia.
Compared with people referred for cancer rehabilitation, those with COVID-19 symptoms lasting beyond 4 weeks were 2.3 times more likely to report pain, 1.8 times more likely to report worse physical health, and 1.6 times more likely to report difficulty with physical activities, an adjusted odds ratio analysis reveals.
The COVID-19 rehabilitation group also performed significantly worse on a 6-minute walk test, suggesting less physical endurance than people recovering from cancer (P < .001). They also used more rehabilitation services overall than the control group.
The researchers suggest services tailored to the unique physical and mental health rehabilitation needs of the post–COVID-19 patient population could be warranted.
The study does not suggest all people recovering with COVID-19 will fare worse than people recovering from cancer, the authors caution. They note that “these results should not be interpreted to mean that post–COVID-19 patients overall had poorer physical and mental health than patients with cancer.”
“Instead, results indicate that post–COVID-19 patients specifically referred to a large physical rehabilitation network had poorer health measures than those referred for cancer, which indicates that some patients recovering from COVID-19 had substantial rehabilitation needs.”
A version of this article first appeared on Medscape.com.