When three sufferers die after taking a cutting-edge drug, most corporations hit the brakes. Not Sarepta Therapeutics.
The Massachusetts-based biotech firm is below hearth for refusing a request from the U.S. Food and Drug Administration to cease promoting its Duchenne muscular dystrophy gene remedy, Elevidys. The AP reported that the FDA made the request after three sufferers who acquired gene therapies from Sarepta died, all from liver issues linked to therapy.
Sarepta stated no.
The firm claims its inner overview discovered no new security issues for youthful sufferers with early-stage Duchenne’s illness, and it plans to maintain distributing the drug to that group. Sarepta says it’s in ongoing talks with the FDA.
The FDA’s authority to drag a drug from the market exists, however the course of can drag on for months or years. In most circumstances, an organization will voluntarily adjust to the company’s request. That didn’t occur right here.
FDA Commissioner Marty Makary issued a blunt assertion saying the company helps entry to remedies for life-threatening circumstances, however received’t hesitate to behave when a transparent security danger emerges.
Elevidys first grabbed headlines in 2023 when it grew to become the primary gene remedy accredited for Duchenne’s muscular dystrophy within the U.S. Even then, some FDA scientists brazenly questioned whether or not it really labored.
The drug, which delivers a one-time genetic repair, was initially greenlit for youthful boys who may nonetheless stroll. Its use later expanded to older sufferers and people in later phases of the illness.
But after two teenage boys died whereas taking Elevidys, Sarepta pulled again shipments for older sufferers. On Friday, the corporate confirmed a 3rd loss of life—a 51-year-old man in a separate scientific trial for a distinct muscular dystrophy therapy. Sarepta says that trial is now on maintain.
All three deaths have been linked to liver harm, a identified danger listed within the drug’s official security info.
Facing mounting criticism, Sarepta not too long ago introduced plans so as to add a daring warning label to Elevidys. The firm additionally laid off a 3rd of its employees, strikes that solely fueled skepticism about its priorities.
At the guts of the controversy is a query many sufferers and households are asking: Should a drug that may save lives even be allowed to danger them?
Related: Doctors Just Helped Create Healthy Babies Using DNA From Three People
Discussion about this post