Dateline: CAMBRIDGE, July 19, 2025 – Sarepta Therapeutics (SRPT.O) refused FDA’s request to halt Elevidys gene therapy shipments after three patients died from liver failure1.
The defiance raises regulatory enforcement questions and threatens the company’s $3 billion muscular dystrophy treatment franchise amid mounting safety concerns.
- FDA requested voluntary halt after three liver failure deaths
- Company refused, citing no new safety information
- Sarepta already cutting 500 jobs, adding black-box warning
Market reaction & context
Sarepta shares fell in after-hours trading Friday following news of the FDA’s shipment halt request2. The biotech company had already announced 500 job cuts and agreed to add a black-box warning to Elevidys earlier this week3.
Gene therapy stocks have faced increased scrutiny this year as regulators tighten oversight following several high-profile safety incidents across the sector.
Detailed analysis
The FDA met with Sarepta officials Friday and requested the company suspend all sales of Elevidys for ambulatory patients, but “the company refused to do so,” according to an FDA statement4. The regulator’s request came after three patients died from liver failure while receiving the treatment5.
Sarepta had previously halted shipments last month of the therapy for older boys with Duchenne muscular dystrophy, a condition that gradually destroys muscle and skeletal strength6. The company has now resumed some shipments while maintaining its position against the FDA’s broader halt request.
Management response
In a community letter, Sarepta said it “declined FDA’s request to voluntarily stop shipping ELEVIDYS to ambulatory patients as there is no new or changed safety information”7. The company argued that the therapy’s benefits continue to outweigh its risks for appropriate patients.
The unprecedented public dispute between Sarepta and the FDA highlights the tension between patient access to potentially life-saving treatments and evolving safety data in rare disease therapies.
Regulatory implications
The FDA’s inability to immediately force a voluntary halt demonstrates the limits of regulatory influence when companies resist cooperation. The agency could potentially pursue more formal enforcement actions, including mandatory recalls or market withdrawal orders.
Elevidys received accelerated approval for Duchenne muscular dystrophy, a rare genetic disorder affecting approximately 15,000 patients in the United States. The therapy represents a significant revenue opportunity for Sarepta, with peak sales estimates exceeding $2 billion annually.
Outlook
The standoff could escalate regulatory scrutiny of Elevidys and potentially delay or complicate approvals for other Sarepta treatments. Investors will watch for any formal FDA enforcement actions and their impact on the company’s broader gene therapy portfolio.
The dispute also raises questions about the adequacy of current clinical trial designs for detecting rare but serious adverse events in gene therapies, potentially influencing future regulatory requirements across the sector.
Not investment advice. For informational purposes only.
References
1 “Sarepta says it won’t comply with FDA request to stop shipping gene therapy Elevidys” (July 19, 2025). Reuters. Retrieved July 19, 2025.
2 “Sarepta stock falls on FDA gene therapy Elevidys comment” (July 18, 2025). CNBC. Retrieved July 19, 2025.
3 “Sarepta to cut 500 jobs, add black-box warning on gene therapy” (July 16, 2025). Reuters. Retrieved July 19, 2025.
4 “Gene therapy maker Sarepta tells FDA it won’t halt shipments” (July 19, 2025). Associated Press. Retrieved July 19, 2025.
5 “Sarepta Refuses FDA Request to Stop Shipping Muscular Dystrophy” (July 19, 2025). New York Times. Retrieved July 19, 2025.
6 “Gene therapy maker Sarepta tells FDA it won’t halt shipments” (July 19, 2025). Financial Post. Retrieved July 19, 2025.
7 “Community Letter: Update regarding ELEVIDYS” (July 18, 2025). Sarepta Therapeutics. Retrieved July 19, 2025.