Read Sarepta’s letter to the community
Shared July 18, 2025
The FDA has revoked Sarepta’s platform technology designation and requested that Sarepta voluntarily stop all shipments of ELEVIDYS today. Sarepta has issued a statement indicating that they will continue to ship ELEVIDYS to the ambulant population.
Read FDA press release HERE
Shared July 18, 2025
Read Sarepta press release HERE
Shared July 18, 2025 at 7:39 PM EDT
Multiple news outlets are reporting that the FDA is going to request that Sarepta Therapeutics halt Elevidys treatments for all individuals with Duchenne. Sarepta has already paused treatment for non-ambulatory individuals after the death of 2 teenagers. Yesterday Sarepta also reported the death of a 51-year old man with LGMD who also died from acute liver failure after receiving a similar gene therapy for his disease.
We recognize the huge impact this news will have on our community, and that our families will have many questions during this period of tremendous uncertainty. Our hearts go out to all those who are impacted by this news, and we commit to sharing information as we learn more.
The unfolding events have cast a pall of uncertainty over the treatment’s future and ignited urgent calls for greater transparency within the industry.
Families and advocates who once championed Elevidys now grapple with a mix of disappointment and concern for those treated patients affected with severe liver failure. As regulatory agencies intensify their scrutiny, the path forward for Elevidys remains fraught with questions about safety, accountability and a reevaluation of its risk-benefit calculation.
The tension between innovation and patient safety has rarely felt so acute. Researchers and policymakers are now weighing whether current frameworks for reporting adverse events and monitoring long-term safety are robust enough. Families deserve access to all the available data in order to make appropriate decisions about Elevidys, as well as any treatment option or clinical trial opportunity.
Meanwhile, families awaiting treatment options must navigate a landscape where hope is tempered by the realities of medical complexity. It’s important to recognize that there are multiple FDA approved AAV therapies for a range of genetic diseases. We at CureDuchenne still believe in the promise of gene therapy for Duchenne, and while we appreciate the need for caution, we remain hopeful that AAV gene therapy programs will continue to advance with important learnings.
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