Sarepta, FDA and gene therapy
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Sarepta Therapeutics (SRPT) stock drops as the company faces potential FDA-mandated studies after safety concerns over its gene therapy, Elevidys. Read more here.
The FDA has placed multiple investigational gene therapy clinical trials on hold, signaling broader platform concerns.
Sarepta rebuffed a call from the Food and Drug Administration to halt all shipments of its gene therapy for Duchenne muscular dystrophy over safety issues, even as patients and investors expressed growing concern about the company’s decision-making.
Sarepta would need to conduct new analyses to validate the safety of Elevidys—which has had U.S. shipments paused by the company over a mounting string of controversies—to the FDA, Endpoints News reported Thursday, citing an unnamed senior FDA official.
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To suggest Sarepta Therapeutics may not survive its gene therapy crisis is a sobering claim to make, STAT's Adam Feuerstein writes. But it's an accurate reflection of the situation.
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Investor's Business Daily on MSNThe Hits Keep Coming For Sarepta. Why Its Latest Setback Creates A 'Dangerous Precedent' For Gene Therapies.Sarepta stock plunged again Thursday on a report that the Food and Drug Administration will require additional clinical testing to validate the safety profile of its controversial gene therapy, Elevidys.
Sarepta faces an “arduous and treacherous path” to try to get its Duchenne muscular dystrophy therapy Elevidys back onto the market