Today, Sarepta Therapeutics announced information regarding their ENVISION study for SRP-9001.
ENVISION is a 72-week placebo control design with crossover study of SRP-9001 (delandistrogene moxeparvovec) in older ambulatory and non-ambulatory individuals living with Duchenne muscular dystrophy.
We learned that it is Sarepta’s intent to enroll a limited number of U.S. participants into the non-ambulatory cohort for a limited time, and not enroll anyone into the older ambulatory cohort in the U.S.
Given the limited time for U.S. enrollment, they expect that some study sites that were planning to participate in the study may not open and some families who were interested in participating in the U.S. will not have the chance to enroll.
Sarepta shared reasoning behind this decision, stating that their hope is the FDA will consider the broadest possible label if there is a positive read-out of the EMBARK study early next year. If the FDA agrees to a broad label, it may occur during the enrollment phase of the 72-week placebo controlled ENVISION study. The risk is that if participants drop out of ENVISION because the therapy is approved in the U.S. for a broader population, it reduces the chances of study success and may lessen the opportunity to bring SRP-9001 forward for a broad population in the U.S. and across the world.
We recognize that families have been anxiously awaiting news around the opening of sites for the ENVISION study focused on late ambulatory/non-ambulatory patients. While this news is incredibly disappointing, we are keenly aware TIME is MUSCLE and are counting the days until the FDA decision regarding SRP-9001 is announced. We will continue to advocate for broad access for all individuals.
On June 14, 2023, PPMD hosted a webinar to discuss the details of the ENVISION study. Watch the webinar recording below.