This evening, Sarepta Therapeutics issued a press release stating that it has voluntarily and temporarily paused all shipments of ELEVIDYS for Duchenne in the United States, effective close of business tomorrow, July 22, 2025. According to Sarepta, this pause will provide time for the company to respond to any requests for information and allow Sarepta and FDA to complete the ELEVIDYS safety labeling supplement process.
The events of the past several days have been unsettling for the Duchenne community. The news that Sarepta has now temporarily halted all shipments of ELEVIDYS, including to the ambulatory population covered under the current traditionally approved label, has understandably intensified concerns and uncertainty.
We deeply value the importance of data-driven decision-making and responsible regulatory oversight. We urge both Sarepta and the FDA to communicate with urgency, clarity, and transparency about the path forward.
PPMD has led the way for Patient Preference work in Duchenne. During this process, we trust the FDA will meaningfully consider this research, which demonstrates that families are willing to accept substantial risk in exchange for therapies that may slow progression.
We also hope that the principles of patient-focused drug development and regulatory flexibility for rare disease are upheld. These frameworks are designed to ensure that regulatory decisions are informed not only by clinical data, but by the lived experiences and needs of rare disease communities.
We recognize that scientific rigor and patient safety must remain paramount. But these values must also be balanced with transparency, trust, and a deep understanding of the urgency felt by families facing the realities of Duchenne each and every day. We are hopeful this pause will provide sufficient time to analyze available data, ensuring patients, families, and clinicians have the information needed to make informed decisions.
PPMD remains committed to working with Sarepta, the FDA, and all stakeholders to rely on data-driven decision making and protect the progress our community has worked so hard to achieve.
What Does This Mean for Individuals and Families?
We understand this is confusing and distressing, especially for individuals and families who are scheduled to receive ELEVIDYS and those who have already received the therapy. Your clinical team will be available to answer questions, help you understand how this may impact you or your child’s care, and offer guidance on making decisions about treatments and care going forward.
PPMD will provide updates as we learn more. We are committed to sharing accurate and timely information as it becomes available.
Read Sarepta’s press release here.
Read Sarepta’s community letter:
July 21, 2025
Dear Duchenne Community,
This evening, Sarepta has made the difficult decision to voluntarily and temporarily pause all U.S. shipments of ELEVIDYS effective at the end of the day tomorrow, including for ambulatory patients. This step will provide Sarepta with time to fully respond to any requests from the agency as we seek to update the label for ELEVIDYS. We believe this approach will provide a forum for a collaborative, science-driven review process with the agency.
You can read more in our statement here.
Please know that this decision was not made lightly. We know some of you were scheduled or taking steps to receive therapy. We share as much information as possible in real time with your physicians and this community. We are doing all we can to ensure the future availability of this therapy because we believe that patients and families should have choices and we hear the stories of the real difference it’s making for your loved ones.
Regards,
Wendy Erler
Senior Vice President, Patient Affairs
advocacy@sarepta.com
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