Sarepta Defies FDA Order to Halt Gene Therapy Shipments Amid Rising Patient Deaths
Sarepta Defies FDA Order to Halt Gene Therapy Shipments Amid Rising Patient Deaths
Sarepta Therapeutics has publicly defied a rare request from the U.S. Food and Drug Administration (FDA) to immediately suspend all shipments of its gene therapy, Elevidys, following the confirmed death of a third patient. The biopharmaceutical company’s refusal marks a highly unusual standoff with the federal agency, intensifying scrutiny on its leading Duchenne muscular dystrophy treatment.
The FDA disclosed late Friday that it had met with Sarepta and formally requested a halt to sales, stating, “the company refused to do so.” While the FDA possesses the authority to forcibly remove drugs from the market, such processes are often protracted. Typically, companies comply with informal agency requests, making Sarepta’s stance a significant departure from standard practice.
FDA Commissioner Marty Makary emphasized the agency’s commitment to patient safety, declaring, “We believe in access to drugs for unmet medical needs but are not afraid to take immediate action when a serious safety signal emerges.”
Sarepta, however, maintains that its internal scientific review found “no new or changed safety signals” for younger Duchenne patients in early stages of the disease, for whom it intends to continue providing Elevidys. The company expressed its willingness for ongoing discussions and information sharing with the FDA.
Elevidys, the first U.S.-approved gene therapy for the fatal muscle-wasting disease, Duchenne muscular dystrophy, has faced controversy since its accelerated approval in 2023. This latest development follows the deaths of two teenage boys who had received the therapy, leading Sarepta to halt shipments for older Duchenne patients last month.
The newly confirmed third death involved a 51-year-old patient in an experimental gene therapy trial for a different form of muscular dystrophy. Sarepta reported this death to the FDA on June 20, prompting the agency to place that specific trial on hold. All three patient fatalities have been linked to liver injury, a known side effect listed in Sarepta’s prescribing information.
Adding to the company’s woes, earlier this week Sarepta announced a bold warning would be added to the drug’s label and a layoff of 500 employees, roughly a third of its workforce. Wall Street analysts have criticized the company for not disclosing the third patient death during these announcements. Sarepta’s shares plummeted over 35% on Friday, reflecting significant investor concern.
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