Today (23rd of July), Roche issued a community update regarding its Duchenne program, specifically addressing the availability of delandistrogene moxeparvovec (Elevidys). This follows a recent directive from the U.S. Food and Drug Administration (FDA) requesting Sarepta to halt all shipments of this treatment within the United States.
In alignment with the FDA’s request, Roche has decided to voluntarily and temporarily suspend all new orders of delandistrogene moxeparvovec for countries that depend on FDA approval, as well as in regions where the product is distributed through the Named Patient Supply (NPS) program. This pause applies to ambulatory Duchenne muscular dystrophy (DMD) patients, as access for non-ambulatory patients had previously been discontinued.
Roche has reiterated its commitment to patient safety, emphasising that, based on current data, the benefits of Elevidys outweigh the risks for ambulatory DMD patients. Accordingly, in countries such as Brazil and Japan, where Elevidys is approved independently of the FDA, Roche will continue to supply and ship the treatment as per local regulations.
Roche is in ongoing communication to clarify the reasons behind the FDA’s request and to determine subsequent actions. The company recognises the potential impact of this development on the Duchenne community, particularly for families awaiting treatment. The company has provided an FAQ section in its full community letter to address initial concerns and encourages anyone with further questions to reach out to Action Duchenne for additional support.
Frequently Asked Questions
I’ve already received treatment with delandistrogene moxeparvovec. Should I be worried?
- The clinical trial programme for delandistrogene moxeparvovec has shown that the treatment can support stabilisation or slowing of disease progression alongside improvements in motor function, with a consistent and manageable safety profile. The meaningful value that can be derived from treatment with delandistrogene moxeparvovec has led to numerous regulatory approvals around the world.
- Please contact your local treating physician with any questions that you might have relating to previous treatment with delandistrogene moxeparvovec.
What happens if treatment is planned but we change our minds about having it?
- We encourage you to discuss your situation with your healthcare provider as they are in the best position to work together with you and your family to decide what is best for you.
How long do you expect this pause to continue? Will you resume accepting new orders for treatment with delandistrogene moxeparvovec?
- At this time we do not have an answer for how long this pause will continue.
- What we can tell you is that we will share important updates and announcements with both the Duchenne community and physicians as we have them.
This update follows the news announced in April, that Roche would be placing a temporary hold on three clinical studies (NCT06241950, NCT06128564, NCT05881408) across the EU and UK, following a request from the European Medicines Agency.
If you would like to talk about the topic discussed in this article, please do reach out to us at info@actionduchenne.org.
