The much-anticipated 48-week results on eteplirsen, a drug designed to skip exon 51, were presented today by Sarepta Therapeutics on a webinar and press release. On the webinar, Sarepta’s Chief Executive Chris Garabedian presented data about the dystrophin found in study participants’ muscle biopsies and the 6-minute walk results.
To read some background about this clinical trial, click here.
An overview of the data reported today:
What's next?
For more details about the findings, read Sarepta's news release.
Also read Beyond Exon 51, a blog from PPMD's Senior Research Director, Sharon Hesterlee, explaining how an exon 51 success can benefit all people with Duchenne, regardless of mutation.
We are hopeful and very enthusiastic about these promising findings. We will keep you updated on what we learn at the World Muscle Society meeting, and please tune in for PPMD’s webinar with Sarepta on October 23rd at 1:00 pm EST.
Learn more:
Holly Peay, Vice President, Education & Outreach
Read more PPMD Staff Blogs
Comment
Has Sarepta published anything more specific than the mean delta values? Would data from individuals in the study typically be shown at something like the muscle conference?
Hello Keith, thank you for that clarification. You are right, it is 47% of normal; I apologize for the typo. I can't speak specifically to the downstream issues with the FDA and the phase III study, except to say that on the Oct 3 webinar, Serepta's CEO described very cautious optimism and an intent to discuss accelerated approval with the FDA. We are eager to know how the newly passed version of PDUFA (Prescription Drug User Fee Act) legislation with its modernization of accelerated approval provision will affect those decisions. Of course it will be in the FDA's hands and I don't think anyone knows whether the odds are good or bad. Personally, I’m interested in hearing from Serepta about the more detailed analysis from this study, which will be presented at the World Muscle Society meeting next week. We’ll keep the community updated as more information comes in.
Point of clarification: The Sarepta release says it was an increase to 47% of normal, not a 47% increase.
Can you talk a little about what the accelerated approval process Sarepta is applying for means, in terms of what additional clinical trials would or would not be required, what the endpoints might be, how fast the drug could concievably be available to people, and how insurance coverage is impacted?
Also, any information available on how large a phase III / confirmatory trial Sarepta is planning for?
Thanks.
the prospects for accelerated approval? Could an accelerated approval be granted solely on the basis of the Phase IIb results, with a post approval confirmatory trial? If they're granted accelerated approval, does the drug go only to those in the confirmatory trial, or anyone that wants it?
Hey Jen! I received the confirmation. you are all signed up.There was a small bug I just fixed. The confirmation page was not redirecting correctly. Ryan
I'm trying to sign up..its not letting me jennmcnary@yahoo.com
© 2021 Created by PPMD.
Powered by
Badges | Report an Issue | Privacy Policy | Terms of Service
You need to be a member of PPMD Community to add comments!
Join PPMD Community