Sarepta Therapeutics Slides Amid FDA Safety Concerns for Gene Therapy Elevidys

Sarepta Therapeutics has resumed its sell-off after Endpoints News reported that the FDA would require new trials to address safety concerns and reintroduce its gene therapy, Elevidys. The therapy, developed in partnership with Roche, is for Duchenne muscular dystrophy. The FDA's request for new trials is a setback for Sarepta, which had expected the FDA to approve Elevidys based on existing data. The FDA's decision is a significant development for the company and the Duchenne muscular dystrophy community.

Sarepta Therapeutics has resumed its sell-off after Endpoints News reported that the FDA would require new trials to address safety concerns and reintroduce its gene therapy, Elevidys. The therapy, developed in partnership with Roche, is for Duchenne muscular dystrophy. The FDA's request for new trials is a setback for Sarepta, which had expected the FDA to approve Elevidys based on existing data. The FDA's decision is a significant development for the company and the Duchenne muscular dystrophy community.

Sarepta initially paused shipments of Elevidys in response to the FDA's request to temporarily halt distribution following multiple patient deaths. The FDA's concerns were heightened after the company reported the deaths of three patients, including two who were treated with Elevidys. The pause allowed Sarepta to work with the FDA to update the drug's safety labeling and incorporate a black box warning for acute liver injury.

Roche, which markets Elevidys outside the United States, also paused shipments in countries that rely on FDA rulings for local approvals. However, Roche will continue to supply Elevidys to countries that approved the gene therapy independently of the FDA, including Brazil and Japan. Roche's pause is limited to patients who can still walk.

The FDA's decision to require new trials for Elevidys is a significant setback for Sarepta and Roche. The companies had been optimistic that the FDA would approve Elevidys based on existing data. The new trials will require additional time and resources, potentially delaying the therapy's availability for Duchenne patients.

The FDA's decision is a reminder of the rigorous safety standards that gene therapies must meet. The pause in shipments and the requirement for new trials highlight the importance of thorough clinical testing and safety monitoring. The FDA's decision may also impact Sarepta's financial performance, as the company will need to invest in new trials and potentially delay revenue from Elevidys.

The Duchenne muscular dystrophy community is anxiously awaiting the outcome of the new trials. The therapy has the potential to significantly improve the lives of patients with the disease. However, the setback may lead to a delay in the availability of the therapy.

References:
[1] https://www.fiercepharma.com/pharma/sarepta-getting-back-fdas-good-side-pauses-shipments-duchenne-gene-therapy-elevidys
[2] https://www.statnews.com/2025/07/23/roche-sarepta-gene-therapy-elevidys-duchenne-safety/