SHERIDAN, WYOMING – July 21, 2025 – Sarepta Therapeutics has entered a critical phase of uncertainty after reports surfaced that the U.S. Food and Drug Administration (FDA) is considering requesting a halt to all shipments of its Duchenne muscular dystrophy (DMD) gene therapy, Elevidys. The potential move follows the disclosure of a third patient death associated with the company's underlying gene therapy platform.
FDA Scrutiny Intensifies After Third Death Linked to Platform
According to a Reuters report citing a source familiar with the matter, the FDA is expected to ask Sarepta to voluntarily suspend Elevidys shipments. The development marks an escalation in regulatory concerns after the death of a 51-year-old patient in a Phase I trial of Sarepta’s limb-girdle muscular dystrophy (LGMD) gene therapy SRP-9004—an experimental therapy built on the same platform technology as Elevidys. The patient, like two previously reported teenage recipients of Elevidys, died from acute liver failure.
In an email statement to BioSpace, a U.S. Department of Health and Human Services (HHS) representative confirmed the Reuters report. An HHS official further stated, “We are taking a hard look at pulling it from the market.”
FDA Commissioner Marty Makary added in a statement to Bloomberg News that regulators are actively evaluating whether Elevidys, first approved in June 2023, should remain available. Sarepta, meanwhile, told CNBC that it had not received any formal communication from the FDA on the matter.
Market Response Reflects Mounting Investor Concerns
Investor reaction was swift. Shares of Sarepta plunged nearly 37% on Friday, deepening the turmoil that has engulfed the biotech company in recent months. The latest sell-off followed Sarepta’s announcement Wednesday of a strategic pivot from its gene therapy programs to its siRNA platform, accompanied by the layoff of approximately 500 employees. The news initially spurred an 18% rebound in Sarepta’s stock as investors responded with cautious optimism.
However, confidence eroded sharply after the latest fatality became public. William Blair analysts wrote in a Friday investor note, “We think the LGMD patient death could amplify patient hesitancy to use commercial Elevidys and increase investor distrust since the company did not disclose the event on its call.”
BMO echoed the concern, adding in a separate investor note that the company only disclosed the LGMD patient death after its management call, citing the discontinuation of the SRP-9004 program as the reason for the delayed disclosure. The firm had warned earlier in the week, “The only risk we see moving forward is the death of a third Elevidys patient.”
A Defining Moment for Sarepta and the Gene Therapy Sector
This unfolding situation underscores critical challenges for the gene therapy industry, particularly concerning the safety of adeno-associated virus (AAV) vector–based therapies. With regulatory scrutiny intensifying, companies like Sarepta face mounting pressure to address both clinical risks and investor confidence.
- Regulatory Risks: The FDA’s pending decision could set a precedent for handling post-approval safety signals in the gene therapy sector.
- Market Impact: Sarepta’s valuation and market standing face significant threats, with investor sentiment turning sharply negative.
- Strategic Shifts: The company’s recent pivot toward siRNA-based assets reflects a broader industry trend of reassessing high-risk therapeutic platforms.
Learn more at www.sarepta.com