Latest news with #Marty Makary
The Independent
2 days ago
- Health
- The Independent
Gene therapy maker Sarepta tells FDA it won't halt shipments despite patient deaths
Drugmaker Sarepta Therapeutics said late Friday it won't comply with a request from the Food and Drug Administration to halt all shipments of its gene therapy following the death of a third patient receiving one of its treatments for muscular dystrophy. The highly unusual move is a latest in a string of events that have hammered the company's stock for weeks and recently forced it to lay off 500 employees. The company's decision not to comply with the FDA also places future availability of its leading therapy, called Elevidys, in doubt. The FDA said in a statement Friday night that officials met with Sarepta and requested it suspend all sales but 'the company refused to do so.' The agency has the authority to pull drugs from the market, but the cumbersome regulatory process can take months or even years. Instead, the agency usually makes an informal request and companies almost always comply. 'We believe in access to drugs for unmet medical needs but are not afraid to take immediate action when a serious safety signal emerges,' FDA Commissioner Marty Makary said in a statement. Elevidys is the first gene therapy approved in the U.S. for Duchenne's muscular dystrophy, the fatal muscle-wasting disease that affects males, though it has faced scrutiny since its clearance in 2023. The one-time treatment received accelerated approval against the recommendations of some FDA scientists who doubted its effectiveness. The FDA granted full approval last year and expanded the therapy's use to patients 4 years and older, including those who can no longer walk. Previously, it was only available for younger patients who were still walking. Sarepta said Friday that its scientific review showed 'no new or changed safety signals' for younger patients with Duchenne's who have earlier stages of the disease. The company said it plans to keep the drug available for those patients. 'We look forward to continued discussions and sharing of information with FDA,' the company said in a statement. Sarepta halted shipments last month of the therapy for older boys with Duchenne's, which gradually destroys muscle and skeletal strength, resulting in early death. The move followed the deaths of two teenage boys taking the therapy. The company also confirmed a third death Friday: a 51-year-old patient who was taking an experimental gene therapy in a trial for a different form of muscular dystrophy. Sarepta said it reported the death to the FDA on June 20. The FDA said Friday it placed that trial on hold. Sarepta noted that the gene therapy involved in the incident uses 'a different dose and is manufactured using a different process,' than Elevidys. All three patient deaths were linked to liver injury, a side effect noted in Sarepta's prescribing information. Earlier this week Sarepta announced it would add a bold warning to drug and lay off a third of its employees. The company did not mention the third patient death in its news release or conference call announcing those changes, sparking pointed criticism from Wall Street analysts. Company shares fell more than 35% Friday to close at $14.07. Cambridge, Massachusetts-based Sarepta has received FDA approval for three other Duchenne's drugs since 2016, none of which have been confirmed to work. The company has long been criticized for failing to complete several studies needed to secure full FDA approval of its drugs. ___ The Associated Press Health and Science Department receives support from the Howard Hughes Medical Institute's Department of Science Education and the Robert Wood Johnson Foundation. The AP is solely responsible for all content.
Yahoo
2 days ago
- Health
- Yahoo
Gene therapy maker Sarepta tells FDA it won't halt shipments despite patient deaths
WASHINGTON (AP) — Drugmaker Sarepta Therapeutics (SRPT) said late Friday it won't comply with a request from the Food and Drug Administration to halt all shipments of its gene therapy following the death of a third patient receiving one of its treatments for muscular dystrophy. The highly unusual move is a latest in a string of events that have hammered the company's stock for weeks and recently forced it to lay off 500 employees. The company's decision not to comply with the FDA also places future availability of its leading therapy, called Elevidys, in doubt. The FDA said in a statement Friday night that officials met with Sarepta and requested it suspend all sales but 'the company refused to do so.' The agency has the authority to pull drugs from the market, but the cumbersome regulatory process can take months or even years. Instead, the agency usually makes an informal request and companies almost always comply. 'We believe in access to drugs for unmet medical needs but are not afraid to take immediate action when a serious safety signal emerges,' FDA Commissioner Marty Makary said in a statement. Elevidys is the first gene therapy approved in the U.S. for Duchenne's muscular dystrophy, the fatal muscle-wasting disease that affects males, though it has faced scrutiny since its clearance in 2023. The one-time treatment received accelerated approval against the recommendations of some FDA scientists who doubted its effectiveness. The FDA granted full approval last year and expanded the therapy's use to patients 4 years and older, including those who can no longer walk. Previously, it was only available for younger patients who were still walking. Sarepta said Friday that its scientific review showed 'no new or changed safety signals' for younger patients with Duchenne's who have earlier stages of the disease. The company said it plans to keep the drug available for those patients. 'We look forward to continued discussions and sharing of information with FDA,' the company said in a statement. Sarepta halted shipments last month of the therapy for older boys with Duchenne's, which gradually destroys muscle and skeletal strength, resulting in early death. The move followed the deaths of two teenage boys taking the therapy. The company also confirmed a third death Friday: a 51-year-old patient who was taking an experimental gene therapy in a trial for a different form of muscular dystrophy. Sarepta said it reported the death to the FDA on June 20. The FDA said Friday it placed that trial on hold. Sarepta noted that the gene therapy involved in the incident uses 'a different dose and is manufactured using a different process,' than Elevidys. All three patient deaths were linked to liver injury, a side effect noted in Sarepta's prescribing information. Earlier this week Sarepta announced it would add a bold warning to drug and lay off a third of its employees. The company did not mention the third patient death in its news release or conference call announcing those changes, sparking pointed criticism from Wall Street analysts. Company shares fell more than 35% Friday to close at $14.07. Cambridge, Massachusetts-based Sarepta has received FDA approval for three other Duchenne's drugs since 2016, none of which have been confirmed to work. The company has long been criticized for failing to complete several studies needed to secure full FDA approval of its drugs. ___ The Associated Press Health and Science Department receives support from the Howard Hughes Medical Institute's Department of Science Education and the Robert Wood Johnson Foundation. The AP is solely responsible for all content.
Yahoo
2 days ago
- Health
- Yahoo
Gene therapy maker Sarepta tells FDA it won't halt shipments despite patient deaths
WASHINGTON (AP) — Drugmaker Sarepta Therapeutics said late Friday it won't comply with a request from the Food and Drug Administration to halt all shipments of its gene therapy following the death of a third patient receiving one of its treatments for muscular dystrophy. The highly unusual move is a latest in a string of events that have hammered the company's stock for weeks and recently forced it to lay off 500 employees. The company's decision not to comply with the FDA also places future availability of its leading therapy, called Elevidys, in doubt. The FDA said in a statement Friday night that officials met with Sarepta and requested it suspend all sales but 'the company refused to do so.' The agency has the authority to pull drugs from the market, but the cumbersome regulatory process can take months or even years. Instead, the agency usually makes an informal request and companies almost always comply. 'We believe in access to drugs for unmet medical needs but are not afraid to take immediate action when a serious safety signal emerges,' FDA Commissioner Marty Makary said in a statement. Elevidys is the first gene therapy approved in the U.S. for Duchenne's muscular dystrophy, the fatal muscle-wasting disease that affects males, though it has faced scrutiny since its clearance in 2023. The one-time treatment received accelerated approval against the recommendations of some FDA scientists who doubted its effectiveness. The FDA granted full approval last year and expanded the therapy's use to patients 4 years and older, including those who can no longer walk. Previously, it was only available for younger patients who were still walking. Sarepta said Friday that its scientific review showed 'no new or changed safety signals' for younger patients with Duchenne's who have earlier stages of the disease. The company said it plans to keep the drug available for those patients. 'We look forward to continued discussions and sharing of information with FDA,' the company said in a statement. Sarepta halted shipments last month of the therapy for older boys with Duchenne's, which gradually destroys muscle and skeletal strength, resulting in early death. The move followed the deaths of two teenage boys taking the therapy. The company also confirmed a third death Friday: a 51-year-old patient who was taking an experimental gene therapy in a trial for a different form of muscular dystrophy. Sarepta said it reported the death to the FDA on June 20. The FDA said Friday it placed that trial on hold. Sarepta noted that the gene therapy involved in the incident uses 'a different dose and is manufactured using a different process,' than Elevidys. All three patient deaths were linked to liver injury, a side effect noted in Sarepta's prescribing information. Earlier this week Sarepta announced it would add a bold warning to drug and lay off a third of its employees. The company did not mention the third patient death in its news release or conference call announcing those changes, sparking pointed criticism from Wall Street analysts. Company shares fell more than 35% Friday to close at $14.07. Cambridge, Massachusetts-based Sarepta has received FDA approval for three other Duchenne's drugs since 2016, none of which have been confirmed to work. The company has long been criticized for failing to complete several studies needed to secure full FDA approval of its drugs. ___ The Associated Press Health and Science Department receives support from the Howard Hughes Medical Institute's Department of Science Education and the Robert Wood Johnson Foundation. The AP is solely responsible for all content. Matthew Perrone, The Associated Press
Associated Press
2 days ago
- Health
- Associated Press
Gene therapy maker Sarepta tells FDA it won't halt shipments despite patient deaths
WASHINGTON (AP) — Drugmaker Sarepta Therapeutics said late Friday it won't comply with a request from the Food and Drug Administration to halt all shipments of its gene therapy following the death of a third patient receiving one of its treatments for muscular dystrophy. The highly unusual move is a latest in a string of events that have hammered the company's stock for weeks and recently forced it to lay off 500 employees. The company's decision not to comply with the FDA also places future availability of its leading therapy, called Elevidys, in doubt. The FDA said in a statement Friday night that officials met with Sarepta and requested it suspend all sales but 'the company refused to do so.' The agency has the authority to pull drugs from the market, but the cumbersome regulatory process can take months or even years. Instead, the agency usually makes an informal request and companies almost always comply. 'We believe in access to drugs for unmet medical needs but are not afraid to take immediate action when a serious safety signal emerges,' FDA Commissioner Marty Makary said in a statement. Elevidys is the first gene therapy approved in the U.S. for Duchenne's muscular dystrophy, the fatal muscle-wasting disease that affects males, though it has faced scrutiny since its clearance in 2023. The one-time treatment received accelerated approval against the recommendations of some FDA scientists who doubted its effectiveness. The FDA granted full approval last year and expanded the therapy's use to patients 4 years and older, including those who can no longer walk. Previously, it was only available for younger patients who were still walking. Sarepta said Friday that its scientific review showed 'no new or changed safety signals' for younger patients with Duchenne's who have earlier stages of the disease. The company said it plans to keep the drug available for those patients. 'We look forward to continued discussions and sharing of information with FDA,' the company said in a statement. Sarepta halted shipments last month of the therapy for older boys with Duchenne's, which gradually destroys muscle and skeletal strength, resulting in early death. The move followed the deaths of two teenage boys taking the therapy. The company also confirmed a third death Friday: a 51-year-old patient who was taking an experimental gene therapy in a trial for a different form of muscular dystrophy. Sarepta said it reported the death to the FDA on June 20. The FDA said Friday it placed that trial on hold. Sarepta noted that the gene therapy involved in the incident uses 'a different dose and is manufactured using a different process,' than Elevidys. All three patient deaths were linked to liver injury, a side effect noted in Sarepta's prescribing information. Earlier this week Sarepta announced it would add a bold warning to drug and lay off a third of its employees. The company did not mention the third patient death in its news release or conference call announcing those changes, sparking pointed criticism from Wall Street analysts. Company shares fell more than 35% Friday to close at $14.07. Cambridge, Massachusetts-based Sarepta has received FDA approval for three other Duchenne's drugs since 2016, none of which have been confirmed to work. The company has long been criticized for failing to complete several studies needed to secure full FDA approval of its drugs. ___ The Associated Press Health and Science Department receives support from the Howard Hughes Medical Institute's Department of Science Education and the Robert Wood Johnson Foundation. The AP is solely responsible for all content.
Yahoo
3 days ago
- Health
- Yahoo
Exclusive-US FDA to ask Sarepta to stop shipments of gene therapy Elevidys, source says
By Bhanvi Satija (Reuters) -The U.S. Food and Drug Administration is planning to request Sarepta Therapeutics to voluntarily stop all shipments of its gene therapy, Elevidys, a source familiar with the matter told Reuters on Friday. Sarepta's stock tumbled over 40% to $12.81, its lowest level in more than nine years, after being briefly halted. A company spokesperson said the drugmaker has yet to receive a formal request. The agency's scrutiny of Sarepta has sharpened since the deaths of two teenage boys earlier this year who had received Elevidys, a gene therapy approved in the United States to treat a muscle-wasting condition called Duchenne muscular dystrophy. Earlier on Friday, Sarepta disclosed the death of a third patient - a 51-year-old man with limb-girdle muscular dystrophy who had received its experimental gene therapy SRP-9004. Like the two teenagers, he died from acute liver failure and was non-ambulatory, meaning unable to walk independently. In June, Sarepta stopped shipments of the therapy to such patients. Wall Street analysts have said the third death could amplify patient hesitancy to use Elevidys, given both the therapies use the same delivery vehicle known as adeno-associated virus vector. The company is in the process of changing its label for Elevidys, and the FDA will see the change through, the source told Reuters on Friday. Earlier in the day, FDA Commissioner Marty Makary told Bloomberg News in an interview that he was "taking a hard look" at whether a gene therapy from Sarepta should remain on the market. MANAGEMENT CREDIBILITY On an investor call on Friday, Sarepta faced pointed questions from analysts over why it had not disclosed the recent patient death earlier in the week. CEO Doug Ingram said the matter was "neither material, nor central" to Wednesday's update that had focused on the company's restructuring. "This event occurred in a trial that was otherwise completed with all dosing," said Ingram, adding that the decision to not proceed with the study was made independently of the patient's death. The company also said liver issues were not a new safety signal in the study, which is part of its now-halted limb-girdle muscular dystrophy (LGMD) gene therapy programs. On Wednesday, Sarepta had announced 500 layoffs as well as cuts to its LGMD programs, citing financial reasons. It had not mentioned the patient death then, despite analyst inquiries about safety. Sarepta reiterated the financial basis of its decision on Friday's investor call, but some analysts, including those at BMO Capital Markets, warned it could damage management credibility. At least two analysts asked whether other deaths had occurred in Sarepta's gene therapy programs. The company said it was not aware of any beyond the three that were disclosed. "We are, historically, a very transparent organization. If there was a change in the risk profile of Elevidys, we would first and foremost, share with physicians and patients, and then, of course, we would share it with investors," Ingram said. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
