Sarepta Therapeutics Faces Controversy Over Gene Therapy
WASHINGTON — Sarepta Therapeutics announced late Friday that it will not adhere to the FDA’s request to halt shipments of its gene therapy, Elevidys, which is used to treat Duchenne muscular dystrophy. This decision comes after the unfortunate deaths of three patients who were undergoing treatment.
The move has raised alarms and is the latest in a series of challenges that have impacted the company’s stock, resulting in the recent layoff of 500 employees. The FDA had asked Sarepta to suspend its sales after three deaths, yet the company has chosen not to comply, casting doubt on the therapy’s future availability.
FDA Commissioner Marty Makary stated the agency often requests companies to pause drug sales informally, but Sarepta’s refusal is notable. “While we support access to innovative treatments, we must prioritize patient safety when serious concerns arise,” he added.
Elevidys is the first gene therapy approved in the U.S. for this severe muscle-wasting disease, which predominantly affects males. Its approval has been controversial since its clearance in 2023, particularly after initial doubts from some FDA scientists over its efficacy. Although it received full approval last year, criticism remains regarding the lack of conclusive evidence supporting the drug’s effectiveness.
Sarepta claims its internal review indicated no new safety concerns for younger patients currently taking the therapy. The company has reiterated its commitment to keeping Elevidys available for these patients, stating it looks forward to ongoing discussions with the FDA.
Despite the assurances, the deaths of two teenage boys using the therapy and a 51-year-old patient involved in a different experimental trial linked to liver injury present serious concerns. Following these incidents, Sarepta announced it would implement a stronger warning on the drug and went on to lay off a significant portion of its workforce.
Investors reacted negatively, with Sarepta’s shares plummeting over 35% on Friday, closing at $14.07. Critics have long pointed out that the company has faced multiple challenges in completing studies for FDA approval of its other Duchenne therapies, leaving questions about the overall reliability of its products.
As discussions continue, the future of Elevidys hangs in the balance, raising crucial questions about patient safety and the responsibilities of pharmaceutical companies to ensure their products are both safe and effective.
