Gene therapy maker Sarepta tells FDA it won't halt shipments despite patient deaths
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.
Wednesdays
Columnist Jen Zoratti looks at what's next in arts, life and pop culture.
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.
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
Winnipeg Free Press
6 hours ago
- Winnipeg Free Press
The US fertility rate reached a new low in 2024, CDC data shows
NEW YORK (AP) — The fertility rate in the U.S. dropped to an all-time low in 2024 with less than 1.6 kids per woman, new federal data released Thursday shows. The U.S. was once among only a few developed countries with a rate that ensured each generation had enough children to replace itself — about 2.1 kids per woman. But it has been sliding in America for close to two decades as more women are waiting longer to have children or never taking that step at all. The new statistic is on par with fertility rates in western European countries, according to World Bank data. Alarmed by recent drops, the Trump administration has taken steps to increase falling birth rates, like issuing an executive order meant to expand access to and reduce costs of in vitro fertilization and backing the idea of 'baby bonuses' that might encourage more couples to have kids. But there's no reason to be alarmed, according to Leslie Root, a University of Colorado Boulder researcher focused on fertility and population policy. 'We're seeing this as part of an ongoing process of fertility delay. We know that the U.S. population is still growing, and we still have a natural increase — more births than deaths,' she said. The U.S. Centers for Disease Control and Prevention released the statistic for the total fertility rate with updated birth data for 2024. In the early 1960s, the U.S. total fertility rate was around 3.5, but plummeted to 1.7 by 1976 after the Baby Boom ended. It gradually rose to 2.1 in 2007 before falling again, aside from a 2014 uptick. The rate in 2023 was 1.621, and inched down in 2024 to 1.599, according to the CDC's National Center for Health Statistics. Birth rates are generally declining for women in most age groups — and that doesn't seem likely to change in the near future, said Karen Guzzo, director of the Carolina Population Center at the University of North Carolina. People are marrying later and also worried about their ability to have the money, health insurance and other resources needed to raise children in a stable environment. 'Worry is not a good moment to have kids,' and that's why birth rates in most age groups are not improving, she said. Asked about birth-promoting measures outlined by the Trump administration, Guzzo said they don't tackle larger needs like parental leave and affordable child care. 'The things that they are doing are really symbolic and not likely to budge things for real Americans,' she said. Increase in births in new data The CDC's new report, which is based on a more complete review of birth certificates than provisional data released earlier this year, also showed a 1% increase in births — about 33,000 more — last year compared to the prior year. That brought the yearly national total to just over 3.6 million babies born. But this is different: The provisional data indicated birth rate increases last year for women in their late 20s and 30s. However, the new report found birth rate declines for women in their 20s and early 30s, and no change for women in their late 30s. What happened? CDC officials said it was due to recalculations stemming from a change in the U.S. Census population estimates used to compute the birth rate. That's plausible, Root said. As the total population of women of childbearing age grew due to immigration, it offset small increases in births to women in those age groups, she said. ___ 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.
Toronto Sun
18 hours ago
- Toronto Sun
8 children taken to hospitals after seizure-like symptoms at Massachusetts church concert
Published Jul 23, 2025 • 1 minute read In this still image taken from video, and ambulance with working lights is parked in a street in Cambridge, Mass., on Wednesday, July 23, 2025. Photo by AP CAMBRIDGE, Mass. — Eight children at a church near Harvard University where a French youth choir was holding a concert suffered seizure-like symptoms and were taken to hospitals, officials said. This advertisement has not loaded yet, but your article continues below. THIS CONTENT IS RESERVED FOR SUBSCRIBERS ONLY Subscribe now to read the latest news in your city and across Canada. Unlimited online access to articles from across Canada with one account. Get exclusive access to the Toronto Sun ePaper, an electronic replica of the print edition that you can share, download and comment on. Enjoy insights and behind-the-scenes analysis from our award-winning journalists. Support local journalists and the next generation of journalists. Daily puzzles including the New York Times Crossword. SUBSCRIBE TO UNLOCK MORE ARTICLES Subscribe now to read the latest news in your city and across Canada. Unlimited online access to articles from across Canada with one account. Get exclusive access to the Toronto Sun ePaper, an electronic replica of the print edition that you can share, download and comment on. Enjoy insights and behind-the-scenes analysis from our award-winning journalists. Support local journalists and the next generation of journalists. Daily puzzles including the New York Times Crossword. REGISTER / SIGN IN TO UNLOCK MORE ARTICLES Create an account or sign in to continue with your reading experience. Access articles from across Canada with one account. Share your thoughts and join the conversation in the comments. Enjoy additional articles per month. Get email updates from your favourite authors. THIS ARTICLE IS FREE TO READ REGISTER TO UNLOCK. Create an account or sign in to continue with your reading experience. Access articles from across Canada with one account Share your thoughts and join the conversation in the comments Enjoy additional articles per month Get email updates from your favourite authors Don't have an account? Create Account The symptoms were not life-threatening, the Cambridge Fire Department said in a news release. About 70 other people in attendance at the concert Tuesday evening at St. Paul's Parish in Harvard Square were not affected. Crews first received a call about a child suffering from a seizure. When firefighters arrived, the child was sitting outside of the church but was not actively having a seizure, Fire Chief Thomas Cahill said. 'That quickly escalated into seven other people having seizure-like symptoms,' Cahill told WCVB-TV. The department's hazmat team 'completed a thorough survey of the St. Paul buildings utilizing several air sampling meters to ensure that no hazardous conditions were present,' the news release said. 'Results were negative and the buildings were ventilated.' This advertisement has not loaded yet, but your article continues below. St. Paul's Facebook Page said a French youth choir, the Chœur d'Enfants d'Ile-de-France, was offering a free concert at the church on Tuesday night as part of its 2025 U.S. tour. 'Founded in 1970, the youth choir has travelled extensively and performed with some of the world's top conductors and soloists,' St. Paul's said. 'The concert will feature and exciting mix of sacred and secular repertoire, as well as popular French songs.' Messages seeking comment were emailed to St. Paul's and to the Harvard Catholic Center, the Catholic chaplaincy for Harvard University students and other academic institutions in the area. Canada Golf Toronto & GTA Ontario Sunshine Girls
Winnipeg Free Press
19 hours ago
- Winnipeg Free Press
What to know about Parkinson's disease after Ozzy Osbourne's death
Heavy metal rocker Ozzy Osbourne died this week at 76, just weeks after his farewell show and about five years after announcing he had Parkinson's disease. Here's what to know about the disease: What is Parkinson's? Parkinson's is a neurologic disease that robs people of control over their movements. It typically starts with tremors and is characterized by slow movement, a shuffling gait, stiff limbs, balance problems and slurred speech. Who gets it? About 1 million Americans are living with Parkinson's, and 10 million people worldwide, the Parkinson's Foundation. estimates. It usually appears after age 60, although sometimes it can develop before age 50. What causes Parkinson's? The exact cause isn't known, but Parkinson's develops when cells that produce one of the brain's chemical messengers, called dopamine, begin to deteriorate and die. Dopamine transports signals to parts of the brain that control movement. Parkinson's symptoms appear after enough dopamine-producing cells die that there's too little of this neurotransmitter in the brain. According to the foundation, most experts believe genetic and environmental factors are behind the disease. Dozens of gene mutations linked to Parkinson's have been discovered and genetics account for 10% to 15% of all cases. Other factors suspected of increasing the risk include head injuries, exposure to pesticides and herbicides and where you live. Is there a cure? There is no cure but there are treatments, including medications that affect dopamine levels and a surgically implanted tremor-blocking device. Patients also can benefit from physical and occupational therapy. Wednesdays Columnist Jen Zoratti looks at what's next in arts, life and pop culture. What's the prognosis? Symptoms worsen over time, usually slowly. The severity of symptoms and how quickly they progress varies widely between patients. In advanced cases, people may be unable to walk or care for themselves. They also can suffer from depression as well as memory and thinking problems. While Parkinson's itself isn't considered fatal, people can die from complications of the disease, including lung problems as muscle weakness impedes the ability to cough and to swallow. ___ 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.
