Sarepta Therapeutics Faces Major Setback as FDA Halts Elevidys Shipments Amid Safety Concerns

Sarepta Therapeutics, a biotechnology company specializing in gene therapies, is facing significant challenges following a series of patient deaths and subsequent regulatory actions. The U.S. Food and Drug Administration (FDA) has paused shipments of Elevidys, Sarepta's gene therapy for Duchenne muscular dystrophy (DMD), and revoked the company's platform designation for its viral vector technology.

Patient Deaths Trigger FDA Investigation

Three patient deaths have been reported in connection with Sarepta's gene therapies. Two deaths were linked to Elevidys, leading to the addition of a black box warning for acute liver injury and acute liver failure. A third death occurred in a patient receiving an investigational gene therapy for limb girdle muscular dystrophy (LGMD).

The FDA's response to these incidents has been swift and severe. On July 22, 2025, the regulator paused all LGMD clinical trials, revoked Sarepta's platform designation for its viral vector technology, and requested the discontinuation of all Elevidys shipments in the United States.

Transparency Issues and Market Reaction

Sarepta's handling of the situation has raised concerns about transparency. The company initially failed to disclose the third patient death during a corporate restructuring announcement on July 16, claiming it was "neither material nor central to the topics at hand." This lack of transparency has damaged investor trust, with 85% of investors surveyed by BMO Capital Markets rating Sarepta's management credibility as 2 or lower on a scale of 1 to 10.

The market reaction to these events has been severe. Sarepta's stock price has plummeted from $125 at the start of the year to less than $13, with its market capitalization falling from $15 billion in June 2024 to just over $1.2 billion.

Uncertain Future for Elevidys and Sarepta

The FDA is now considering revoking Elevidys' approval altogether, citing concerns about the therapy's safety and efficacy. FDA Commissioner Marty Makary told Bloomberg News that the agency is evaluating this option, while Center for Biologics Evaluation and Research Director Vinay Prasad stated, "The FDA will not allow products whose harms are greater than benefits."

The Duchenne patient community is particularly affected by these developments. Parent Project Muscular Dystrophy (PPMD), an advocacy group, expressed deep concern and called for clarity and transparency from both Sarepta and the FDA.

As Sarepta navigates this crisis, the pharmaceutical industry watches closely. The company's ability to recover remains uncertain, with analysts predicting continued volatility and challenges in rebuilding trust with investors, patients, and regulators.