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.
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.
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.
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.
Meet the assholes behind this decision:
Sarepta Therapeutics thinks it can act like POTUS and just keep doing what is patently wrong.
And it is probably correct in that assumption. Just “donate” the potus some cash and its all good.
They’ll get fined but end up making way more money from this than the fine itself. This is why corporations don’t care about the ramifications.
