FDA Reassesses Sarepta’s Elevidys After Third Patient Death

Highlights

• The FDA is reconsidering whether Sarepta’s Elevidys should remain on the U.S. market following three patient deaths linked to the company's gene therapies.
• The agency has requested a voluntary halt to all Elevidys shipments, not limited to non-ambulatory patients.
• Sarepta’s stock plummeted over 35% after the news broke, with broader biotech indexes also taking a hit.
• Elevidys received accelerated FDA approval in June 2023, with a $3.2 million price tag, for the treatment of Duchenne muscular dystrophy.
• Two patient deaths were directly linked to Elevidys; a third occurred with a similar therapy using the same AAV vector.
• The company is under criticism for transparency issues following its failure to disclose the third fatality during major restructuring announcements.
• Elevidys’ FDA approval faced internal resistance and remains controversial within regulatory and medical circles.

Regulatory Scrutiny Intensifies

Sarepta Therapeutics’ gene therapy, Elevidys, is facing renewed regulatory scrutiny following the death of a third patient associated with one of the company’s gene therapies. Although the therapy had previously avoided a market withdrawal thanks to a newly added black box warning, the U.S. Food and Drug Administration (FDA) is now taking a more serious stance.

According to Bloomberg News, FDA Commissioner Dr. Martin Makary is currently evaluating whether Elevidys should continue to be available in the U.S. market. A federal official confirmed with Fierce Pharma that the agency is “taking a hard look at pulling it from the market.”

FDA Requests Shipment Suspension

In a move signaling growing concern, the FDA is now requesting Sarepta to voluntarily suspend all shipments of Elevidys—not just those designated for non-ambulatory patients. The news was initially reported by Reuters and confirmed by sources close to the matter, as shared with Fierce Pharma. As of the latest reports, Sarepta has not issued a public comment in response.

Market Fallout and Stock Performance

The controversy has had an immediate impact on Sarepta’s market position. As of 2:30 p.m. ET on the day of the announcement, the company’s stock had dropped by more than 35%, reaching $13.9 per share. The broader biotech index XBI also experienced a decline of 1.9%, mirroring Sarepta’s losses.

Background on Elevidys Approval

Elevidys received accelerated approval from the FDA in June 2023 for the treatment of Duchenne muscular dystrophy (DMD), a rare genetic disorder. Designed as a one-time gene therapy, it entered the market with a list price of $3.2 million. Initially approved for ambulatory 4- and 5-year-old patients, the label was expanded in June to include all DMD patients aged 4 and above, including non-ambulatory individuals.

Patient Fatalities and Safety Investigations

Concerns surrounding Elevidys intensified in March when a patient died from acute liver failure (ALF) following treatment. At the time, a company spokesperson stated, “We are collecting additional information, but based on the current information from the treating physician, including the time since treatment and the clinical course, we cannot rule out the possibility that Elevidys was a contributing factor.”

A second death was reported in June, also involving a non-ambulatory teenage boy who developed ALF after receiving Elevidys. In response, Sarepta suspended the commercial use of the therapy in non-ambulatory patients and began seeking FDA approval for an enhanced liver toxicity mitigation strategy using the immunosuppressant sirolimus.

Just over a week after restricting Elevidys use, the FDA opened a formal investigation into the two deaths. The situation escalated further when a third patient death, this time unrelated to DMD but involving a similar gene therapy candidate coded SRP-9004, was disclosed. The patient had received the therapy for a subtype of limb-girdle muscular dystrophy. Both Elevidys and SRP-9004 utilize the same adeno-associated virus (AAV) vector, which has been linked to liver toxicity.

Ongoing Transparency Concerns

Sarepta paused development of SRP-9004 last Wednesday, but did not initially disclose the fatality. During a subsequent investor call, CEO Douglas Ingram claimed the decision was unrelated to the patient death, a stance that has drawn criticism from industry analysts. Many expressed frustration that such a critical detail was omitted during a week in which the company also announced a major restructuring involving 500 layoffs and the implementation of a black box warning for Elevidys.

Controversy from the Start

Elevidys’ approval has been mired in controversy from the outset. Peter Marks, M.D., Ph.D., the former director of the FDA’s Center for Biologics Evaluation and Research (CBER), overruled negative reviews from his own team to approve the therapy. His successor, Dr. Vinay Prasad, has since criticized the decision.

In a March post on X, Prasad wrote, “[Marks] overturned 3 reviewers to approve a [Duchenne] gene therapy that seems to be killing children and blowing their livers up.” The comment came shortly after Marks’ surprise departure from the agency.