ELEVIDYS UPDATE

Breaking News: Sarepta to resume shipments of ELEVIDYS for ambulatory individuals with Duchenne

07/28/25 5:09 PM EDT

We are pleased to report that the US FDA has recommended that Sarepta remove the voluntary hold for Elevidys for ambulatory patients, after concluding that the death of an 8-year-old boy in Brazil was unrelated to the gene therapy treatment. Sarepta has resumed shipments of its gene therapy drug for ambulatory individuals with Duchenne.

We applaud the FDA for moving quickly to evaluate available information while listening to and responding to the community impacted by Duchenne, and urge Sarepta and the FDA to continue to work together to better understand the risk-benefit profile for non-ambulant individuals with Duchenne.

We recognize that it has been a trying time for the Duchenne community, and especially for individuals who already had appointments to be treated or are hoping to receive this treatment.  We encourage you to reach out to your medical provider for updated information. 

As always, CureDuchenne is committed to working together with the entire community and the FDA to ensure that families and individuals with Duchenne have safe, effective treatments and are able to make informed decisions about treatment options. 

Link to press release HERE

Read the attached community letter

Read the FDA press release HERE

Breaking news:  Sarepta will voluntarily pause all shipments of Elevidys in the US after the close of business on Tuesday, 7/22/2025

07/21/25 7:40 PM EDT

Sarepta Therapeutics just announced that it is voluntarily and temporarily pausing Elevidys treatments in the US, in order to work with the FDA to complete the update to the Elevidys safety label.  Sarepta had already paused treatments for non-ambulatory individuals, but today’s announcement means that ambulatory individuals will not be dosed until further notice. 

We appreciate that many in our community will be impacted by this news, and especially those who were expecting to be dosed soon.  Our hearts are with you at this extremely difficult time.  The past week has already been very trying, and we at CureDuchenne are committed to providing updates as we learn more. 

While we can’t predict how long Elevidys will remain unavailable in the US, we fully expect Sarepta and the FDA will work diligently to agree an amendment to the Elevidys label that evaluates the use of an enhanced immunosuppression regimen to mitigate the risk of acute liver failure.

CureDuchenne is committed to working together with the entire community to ensure that, with more data and appropriate safety protocols, all individuals with Duchenne will have access to safe and effective treatments. 

Link to press release HERE
Community letter attached:

Read Sarepta’s letter to the community
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
July 18, 2025

Read Sarepta press release HERE
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.