We are heartbroken to learn of the loss of another member of our community following treatment with ELEVIDYS. According to Sarepta, this represents the second reported case of acute liver failure in a non-ambulatory patient resulting in death following treatment with ELEVIDYS. 

Our hearts ache for this family. The second tragic loss following treatment with gene therapy is a devastating reminder of both the promise and the complexity of this emerging therapeutic landscape. As we mourn alongside the community, we must also work toward a better understanding of the risks involved and improvement of safety monitoring protocols to ensure that the well-being of every individual remains top priority. 

In response to this news, Sarepta has taken the following steps: 

• Suspending all commercial shipments of ELEVIDYS for non-ambulatory patients while an enhanced safety protocol is reviewed. 
• Convening an independent panel of experts in Duchenne and liver health to evaluate additional safety measures, including the possible use of sirolimus alongside corticosteroids to help prevent liver complications.
• Pausing the ENVISION clinical trial (SRP-9001-303), which includes non-ambulatory and older ambulatory individuals, to allow time for protocol updates and regulatory discussions.

There are no changes to the protocol for ambulatory patients, and the existing corticosteroid regimen remains in place. Families that may have questions or concerns about this news should consult with their clinicians, who can provide individual guidance.  

Sarepta’s ex-U.S. partner for ELEVIDYS, Roche, has immediately paused all dosing in non-ambulatory individuals globally and is working with regulators and clinicians to reassess safety protocols, while treatment for ambulatory patients remains unchanged. You can read Roche’s release here.

Our hearts are heavy today, and our entire community grieves alongside this family and their loved ones. We stand united, determined to make Duchenne therapies as safe and effective as possible. 

PPMD will work with Sarepta to provide any additional information and to help answer questions as we learn more. We look forward to coming together as a community this week at our Annual Conference, where Sarepta will be available to meet with families and individuals, as well. 

Read Sarepta’s community letter here.

Read Roche’s community letter here.