Sarepta Therapeutics Shares Fall Amid FDA Request to Halt Gene Therapy Shipments

Sarepta Therapeutics shares fell as the FDA reportedly plans to request the company to voluntarily halt all shipments of its gene therapy, Elevidys, developed with Roche. The FDA is allegedly weighing the request due to concerns about the therapy's efficacy and potential risks. The move could impact the treatment's approval and availability in the US.

Sarepta Therapeutics' shares have taken a significant dip following reports that the FDA plans to request the company to voluntarily halt all shipments of its gene therapy, Elevidys. The FDA's move is sparked by growing concerns over the therapy's efficacy and potential risks, which could significantly impact its approval and availability in the US.

The FDA has reportedly requested Sarepta Therapeutics to suspend the distribution of Elevidys, its approved gene therapy for Duchenne muscular dystrophy (DMD), as well as pause clinical trials testing the company's gene therapy agents. The decision comes after three patient deaths were linked to liver failure, all of whom were treated with Elevidys. The FDA's action is part of an ongoing evaluation of the therapy's safety and efficacy, particularly in non-ambulatory patients.

Sarepta Therapeutics has faced scrutiny since the first patient death was reported in June 2025. In response to the incidents, Roche, which collaborated with Sarepta on the development of Elevidys, imposed new dosing restrictions for the gene therapy, limiting its use in non-ambulatory patients with DMD. Additionally, Sarepta paused its phase 3 ENVISION trial, which was testing the gene therapy in older ambulatory and non-ambulatory patients with DMD.

The FDA's request for a voluntary halt on Elevidys shipments comes as the agency seeks to understand whether the incidents are related to the product and to avoid future unreasonable illness or injury. The decision underscores the FDA's commitment to patient safety and its willingness to take immediate action when serious safety signals emerge.

Sarepta Therapeutics has stated that it is working to mitigate any risks related to Elevidys and has convened an expert committee to review patient cases. The company is also planning to discuss a proposal with the FDA to gather data on the regimen in a new cohort of the ENDEAVOR study as a pathway to re-establish dosing in the non-ambulatory setting.

The potential halt on Elevidys shipments could have significant financial implications for Sarepta Therapeutics. The gene therapy, which received traditional approval in 2024 for ambulatory patients with DMD, is the first and only marketed gene therapy specific to patients with DMD. The disease affects approximately 20,000 individuals in the United States.

References:
[1] FDA Requests Sarepta Therapeutics Suspend Distribution of Elevidys and Places Clinical Trials on Hold for Multiple Gene Therapy Products Following 3 Deaths. News release. FDA. July 18, 2025. Accessed July 18, 2025. https://www.fda.gov/news-events/press-announcements/fda-requests-sarepta-therapeutics-suspend-distribution-elevidys-and-places-clinical-trials-hold
[2] UPDATED: Analysts demand transparency after Sarepta's roundabout disclosure of 3rd patient death. Fierce Biotech. July 18, 2025. Accessed July 18, 2025. https://www.fiercebiotech.com/biotech/3rd-patient-dies-following-treatment-sarepta-gene-therapy-reports
[3] Sarepta Therapeutics Crashes After A Third Gene Therapy Patient Dies. Investor’s Business Daily. July 18, 2025. Accessed July 18, 2025. https://www.investors.com/news/technology/sarepta-stock-patient-death-gene-therapy-restructuring/
[4] [Ad hoc announcement pursuant to Art. 53 LR] Roche provides safety update on Elevidys™ gene therapy for Duchenne muscular dystrophy in non-ambulatory patients. Roche. June 15, 2025. Accessed July 18, 2025. https://www.globenewswire.com/news-release/2025/06/15/3099406/0/en/Ad-hoc-announcement-pursuant-to-Art-53-LR-Roche-provides-safety-update-on-Elevidys-gene-therapy-for-Duchenne-muscular-dystrophy-in-non-ambulatory-patients.html
[5] Sarepta Provides Safety Update for ELEVIDYS and Initiates Steps to Strengthen Safety in Non-Ambulatory Individuals with Duchenne. June 15, 2025. Accessed July 18, 2025. https://www.businesswire.com/news/home/20250614701775/en/Sarepta-Provides-Safety-Update-for-ELEVIDYS-and-Initiates-Steps-to-Strengthen-Safety-in-Non-Ambulatory-Individuals-with-Duchenne
[6] Sarepta Therapeutics Announces Strategic Restructuring and Pipeline Prioritization Plan to Maintain Long-term, Sustainable Growth and Provides Update on ELEVIDYS Label. News release. Sarepta Therapeutics. July 16, 2025. Accessed July 18, 2025.
[7] Sarepta Therapeutics Announces Expanded US FDA Approval of ELEVIDYS to Duchenne Muscular Dystrophy Patients Ages 4 and Above. News Release. Sarepta Therapeutics. Published June 20, 2024. Accessed July 18, 2025. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-expanded-us-fda-approval-elevidys?_ga=2.261745871.332981042.1718920455-330115727.1718920455