Updated July 18, 2025 at 8:35 PM ET

Sarepta Therapeutics has provided an update indicating that the company has received an informal request from the U.S. Food and Drug Administration (FDA) to voluntarily halt shipment of ELEVIDYS in the U.S.

According to Sarepta, based on data that shows no new or changed safety signals in the ambulant patient population, the company will continue to ship ELEVIDYS to the ambulant population.

In its press release, Sarepta acknowledged the recent death of a 51-year-old non-ambulant Limb-Girdle muscular dystrophy (LGMD) patient, clarifying that this event occurred following treatment of LGMD Type 2D with SRP-9004—not ELEVIDYS—using a different dose and manufacturing process than ELEVIDYS. Additionally, Sarepta noted that the company reported this acute liver failure event as a life-threatening case and subsequent death to FDA in a timely manner, in accordance with applicable law.

PPMD is continuing to monitor the situation and will keep the community informed as developments unfold and next steps become clear.

Read Sarepta’s community letter here.

++

Updated July 18, 2025 at 7:57 PM ET

The U.S. Food and Drug Administration (FDA) has announced it has placed Sarepta Therapeutics investigational gene therapy clinical trials for limb girdle muscular dystrophy on clinical hold and revoked Sarepta’s platform technology designation. According to the FDA, agency leadership met with Sarepta and requested it voluntarily stop all shipments of ELEVIDYS and the company refused to do so.

According to the FDA, continued approval for the use of ELEVIDYS in non-ambulatory patients living with Duchenne is contingent upon verification and description of clinical benefit in a confirmatory trial. Given new safety information, the FDA has notified the company that the indication should be restricted to use in ambulatory patients.

This update comes after what the FDA identifies as three deaths in non-ambulatory individuals that appear to have been a result of acute liver failure in individuals treated with ELEVIDYS or investigational gene therapy using the same AAVrh74 serotype that is used in ELEVIDYS. The FDA also decided to remove the platform technology designation for Sarepta’s AAVrh74 technology because early evidence didn’t show that it could be safely used in more than one drug.

++

PPMD acknowledges the media reports that the U.S. Food and Drug Administration (FDA) will request Sarepta Therapeutics stop shipping ELEVIDYS for all Duchenne patients. These reports are profoundly upsetting and raise serious concerns for our entire community. Families who have fought tirelessly for access to this therapy, those who have already received it, and those who are in line to receive it are now left with more questions than answers. 

ELEVIDYS was the first gene therapy approved for Duchenne and set an important precedent for the development and evaluation of future therapies. Its approval signaled both scientific and regulatory readiness to explore genetic approaches to treating Duchenne and underscored the importance of continued investment and collaboration in the rare disease space.

PPMD is urgently seeking answers and calling on both Sarepta and the FDA to provide clarity and transparency. Above all, we will remain steadfast in our commitment to the Duchenne community, to protect access, demand transparency, advocate for science-driven, patient-centered regulatory decisions, and ensure that individuals and families receive the answers and support they need and deserve.

PPMD is pulling together expert clinicians and community leaders to discuss next steps and assess the implications of this proposed halt in distribution for further investigation and determine the best path forward for families. We recognize the urgency and anxiety this announcement has created. Our expert partners will help us evaluate the medical, regulatory, and practical consequences of this development and ensure families receive accurate information and guidance. PPMD is committed to providing timely updates and actionable resources as this situation unfolds.

PPMD remains unwavering in our commitment to the Duchenne community. To every parent, patient, and caregiver: you are not alone. PPMD is mobilizing the full strength of our network, from clinical experts to legal advisors to get answers. Together, we will navigate this, as we always have, with resilience and urgency, protecting the safety of our community while continuing to drive forward the momentum of life-altering innovations. PPMD will continue to monitor this evolving situation closely and will provide timely updates to the community as new information becomes available.