Yesterday afternoon, Sarepta issued a press release and community letter which included updates related to the non-ambulatory label for ELEVIDYS. While ELEVIDYS continues to be available for individuals living with Duchenne who are ambulant, Sarepta paused shipments for non-ambulant patients following a second safety event in June.
Key takeaways for the Duchenne community from this latest announcement include:
- A committee of neuromuscular specialists, hepatologists, hematologists, and immunologists has met to review cases of acute liver failure (ALF) and is proposing an enhanced immunosuppressive protocol with sirolimus for non-ambulant patients. According to Sarepta, the company plans to submit this protocol to the FDA for review and discuss a proposal to gather data on the regimen in a new cohort (Cohort 8) of the ENDEAVOR study (Study SRP-9001-103) as a pathway to re-establish dosing in non-ambulant patients.
- The FDA has requested that a black box warning for acute liver injury (ALI) and ALF be added to the ELEVIDYS label.
Yesterday’s announcement also included information on a strategic company restructuring—including a 36% workforce reduction. We have reached out to Sarepta and will share additional information with the community when available.
Read Sarepta’s community letter:
Dear US Duchenne Community,
Today, Sarepta issued a press release announcing a strategic company restructuring and pipeline prioritization plan to help ensure we remain a strong, sustainable company well into the future— able to continue delivering on our long-standing mission for the Duchenne community and other families with rare genetic diseases.
Our mission and purpose are rooted in your trust and continued transparency. That’s why we want to share what this news means, both in terms of difficult decisions and areas of progress that directly impact Duchenne families.
As part of the restructuring and implementation of a more focused strategy, we have made difficult but necessary changes— including reducing our workforce by 36%. These decisions are not made lightly, especially knowing the dedication of our team to the patients and families we serve. Many of you have gotten to know Sarepta team members over the years and know how hard this is.
Through all of this, our commitment to the Duchenne community remains unwavering. Sarepta’s exon-skipping therapies continue to serve many families and provide a stable foundation to continue supporting and advancing Duchenne care.
Update on Efforts Related to ELEVIDYS Safety
We also want to update you following previously communicated steps being taken to strengthen the ELEVIDYS safety profile. While ELEVIDYS continues to be available for those in the community who are ambulant, we paused shipments for non-ambulant patients while we took specific actions to inform and work with FDA immediately after learning of a second safety event in June. At that time, we shared that we would convene a panel of scientific experts to review the cases and align on proposed next steps. The committee of neuromuscular specialists, hepatologists, hematologists, and immunologists has met and proposed a clinical study to investigate an enhanced immunosuppressive regimen, and we are submitting this protocol to the FDA for review.
Additionally, while we are not in on-going discussions with FDA about the ELEVIDYS label, we do anticipate a black box warning for acute liver injury (ALI) and acute liver failure (ALF). We will share additional information with the community when possible.
The changes announced today are focused on protecting the future—for the Duchenne community and for others with serious unmet needs. With these decisions, we are positioning ourselves to continue delivering meaningful therapies and advancing promising new science, including through our next-generation siRNA programs.
We are here because of you, and we are staying the course with and for you. Our gratitude for your partnership is sincere.
With respect and commitment,
Wendy Erler
Senior Vice President, Patient Affairs Advocacy@sarepta.com
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