Sarepta Therapeutics Halts ELEVIDYS Shipments Due to Fatal Liver Failures in Non-Ambulatory DMD Patients

Sarepta Therapeutics has halted shipments of its gene therapy ELEVIDYS for non-ambulatory Duchenne muscular dystrophy patients due to two reported cases of fatal acute liver failure. The company has paused its ENVISION Phase 3 trial to update protocols and discuss with regulators. DMD primarily affects males, causing muscle loss and respiratory issues. Sarepta Therapeutics is a commercial-stage biopharmaceutical company discovering RNA-targeted therapeutics and gene therapies for rare diseases.

Sarepta Therapeutics, Inc. (SRPT) has announced a significant development affecting its gene therapy product, ELEVIDYS. The company has temporarily halted shipments of ELEVIDYS for non-ambulatory Duchenne muscular dystrophy (DMD) patients following the reported deaths of two patients who experienced acute liver failure after treatment with the drug. This decision comes amidst ongoing regulatory scrutiny and the company's efforts to enhance the safety profile of ELEVIDYS.

The two fatal cases were reported in June 2025, with the FDA issuing a safety communication on June 24, 2025, to investigate the potential risk of acute liver failure associated with ELEVIDYS [2]. In response, Sarepta paused its ENVISION Phase 3 trial to update protocols and consult with regulators. The company has also temporarily suspended distribution of ELEVIDYS for non-ambulatory patients until a new immunosuppressive regimen can be approved.

DMD is a rare, genetic disorder that primarily affects males, leading to progressive muscle weakness and respiratory issues. ELEVIDYS, an approved gene therapy for DMD, is designed to address the underlying cause of the disease by producing micro-dystrophin in skeletal muscle. The company's decision to pause shipments and update protocols underscores its commitment to ensuring the safety of its patients.

Sarepta Therapeutics is a commercial-stage biopharmaceutical company that specializes in RNA-targeted therapeutics and gene therapies for rare diseases. The company's stock price has been volatile in recent weeks, reflecting the market's reaction to these developments. As of July 2, 2025, Sarepta's stock price has fallen from its previous highs, indicating investor concern over the safety issues and regulatory uncertainties surrounding ELEVIDYS.

Investors and financial professionals should closely monitor the developments surrounding Sarepta Therapeutics and ELEVIDYS. The company's ability to address the safety concerns and maintain regulatory compliance will be critical to its long-term success.

References:
[1] https://www.tradingview.com/news/reuters.com,2025-07-14:newsml_GNX9xxYf3:0-investor-alert-pomerantz-law-firm-reminds-investors-with-losses-on-their-investment-in-sarepta-therapeutics-inc-of-class-action-lawsuit-and-upcoming-deadlines-srpt/
[2] https://www.marketbeat.com/stocks/NASDAQ/SRPT/fda-events/