Sarepta's Elevidys Faces Setbacks in Europe Amid Ongoing Safety Concerns

In a significant blow to Sarepta Therapeutics, the European Union's Committee for Medicinal Products for Human Use (CHMP) has issued a negative opinion on the company's gene therapy Elevidys for ambulatory patients with Duchenne muscular dystrophy (DMD). This decision comes amid mounting safety concerns and regulatory challenges for the embattled drug.

European Regulatory Hurdles

The CHMP, operating under the European Medicines Agency (EMA), declined to endorse Elevidys for use in ambulatory DMD patients aged 3 to 7 years. Roche, which holds the rights to Elevidys outside the United States, submitted the application for EU approval in June 2024.

The committee's decision was based on the failure of Elevidys to demonstrate significant improvement in patients' ability to move after one year in the key Embark study. Moreover, the EMA review panel concluded that the drug's primary biological effect – the production of a shortened form of dystrophin – could not be definitively linked to functional improvements.

Levi Garraway, Roche's Chief Medical Officer, expressed disappointment with the decision, citing the "urgent need for disease-modifying therapies for children in the EU living with Duchenne." Roche plans to continue working with the EMA to explore potential paths forward, emphasizing the drug's performance on secondary study objectives and long-term data pooled from multiple trials.

Safety Concerns and U.S. Distribution Halt

The negative opinion from Europe compounds recent setbacks for Elevidys in the United States. Sarepta has suspended shipments of the gene therapy following multiple patient deaths and subsequent scrutiny from the U.S. Food and Drug Administration (FDA).

In March 2025, Sarepta reported the death of a non-ambulatory DMD patient from acute liver failure after treatment with Elevidys. A second non-ambulatory patient death in June prompted the company to suspend shipments to this patient group. The situation escalated further with the announcement of a third patient death related to SPR-9004, an investigational gene therapy using the same vector technology as Elevidys.

In response to these events, Sarepta has added a black box warning to Elevidys and, at the FDA's request, suspended all shipments of the therapy. Roche has followed suit, temporarily halting deliveries to non-ambulatory patients in countries that base their approvals on FDA decisions.

Financial Implications and Future Outlook

The ongoing challenges surrounding Elevidys pose significant financial risks for Sarepta. The gene therapy, which received controversial FDA approval in 2023 and expanded clearance in 2024, was expected to generate hundreds of millions of dollars in revenue crucial for meeting Sarepta's debt obligations.

The negative opinion in Europe further jeopardizes Sarepta's financial future by potentially eliminating near- and medium-term milestone and royalty payments from Roche. This development, coupled with the uncertain status of Elevidys in the U.S. market, may force Sarepta to consider additional cost-cutting measures beyond its recent layoffs and restructuring efforts.

As the pharmaceutical industry closely watches these developments, the future of Elevidys and Sarepta's position in the gene therapy market remain uncertain. The company faces the challenging task of addressing safety concerns, navigating regulatory hurdles, and maintaining financial stability in the wake of these setbacks.