Sarepta has issued a series of press releases and community letters with updates concerning the label and shipments for ELEVIDYS. They have also released information about plans to include a stronger immunosuppression protocol involving sirolimus for patients who are non-ambulatory.
Learn more and read their full press release here and community letter below.
You can find earlier press releases with more information here: https://investorrelations.sarepta.com/press-releases.
July 19, 2025
Dear Duchenne Community,
We understand that there was a lot of uncertainty around the status of ELEVIDYS following reports of the FDA requesting Sarepta voluntarily halt shipments for all patients. We are writing to clarify what happened, when we learned of this request, what Sarepta is doing about it, and most importantly, what it means for the community.
First and foremost, ELEVIDYS remains available and Sarepta will continue to ship ELEVIDYS for ambulant patients. In June, we voluntarily halted shipments of ELEVIDYS for non-ambulatory patients to allow for an expert committee to evaluate the recommendation of an added immunosuppressive regimen and for necessary discussions with the FDA to take place. The pause on shipments for non-ambulant patient doses is still in place.
What happened yesterday, July 18?
We learned about the FDA’s intent to request Sarepta halt shipment of ELEVIDYS through media reports, at the same time as you did. Before engaging with all of you in Duchenne community, our process is to make sure that we have information that is accurate, truthful, and confirmed through official channels. Later in the afternoon, FDA did informally request a voluntary halt on shipment. A full timeline of the communication and interactions is detailed in our press release.
Second, media reports linked a recent patient death from a clinical trial of SRP-9004 to ELEVIDYS. This caused unnecessary confusion. This tragic event occurred in a Phase 1 clinical trial for an investigational gene therapy called SRP-9004. SRP-9004 is a clinical stage therapy that is intended to treat a different disease, LGMD Type 2D, is administered using a different dose, and is manufactured using a different process. When the patient’s condition was deemed life threatening, Sarepta reported it to the FDA on June 20, 2025, and when the patient passed, Sarepta reported the death to the FDA on July 3, 2025. Clinicians and leaders of patient advocacy organizations were also informed of the patient’s passing. We recognize that the death of any patient is heartbreaking, and our hearts go out to the patient’s family, friends, and everyone involved in his care.
Third, we declined FDA’s request to voluntarily stop shipping ELEVIDYS to ambulatory patients as there is no new or changed safety information for the ambulant patient population. Additionally, as we promised we would do, we assembled a panel of experts including physicians who specialize in neuromuscular disease, liver specialists, immune system experts and hepatologists to inform a protocol for evaluating the use of additional immunosuppression to further mitigate the risk of acute liver failure. The output from this committee has been shared with the FDA and we will continue to actively dialogue with the FDA on the role of immunosuppression in the interest of patients with Duchenne. The recommendations from the expert committee will also be shared with the Duchenne community as soon as possible.
Most importantly, the choice to be treated with ELEVIDYS should remain with patients, families, and their doctors. Their decisions should be informed by accurate information. Our goal at Sarepta is to preserve access to ELEVIDYS so that patients, their families, and their doctors can have well informed discussions and make their own choice about treatment. Advancing novel therapies in rare diseases requires a huge commitment to scientific integrity and data. The body of evidence supporting ELEVIDYS continues to grow and almost 1000 patients have been treated. We promise to provide all updates, and new scientific information in a timely manner.
With gratitude and commitment,
Wendy Erler
Senior Vice President, Patient Affairs
[email protected]
