With the recent news of Sarepta's promising 48-week data, many have asked how accelerated approval may play into the FDA review of eteplirsen. Let's first define what accelerated approval means.
Because it often takes years to assess whether patients truly benefit/improve from a particular drug or if the drug shows a "meaningful clinical outcome", in 1992 the FDA instituted accelerated approval allowing approval based on a surrogate end point. This applies to diseases with an unmet medical need, like Duchenne. Approval of a drug based on such endpoints is given on the condition that post marketing clinical trials verify the anticipated clinical benefit.
FDA rules state that accelerated approval indicates that acceptable/validated clinical endpoints have been met, that the trial(s) was adequate/well controlled, that FDA has received NDA application/reviewed it/agreed with sponsor analysis, AND that sponsor has agreed to post approval trial to confirm outcome (not just repeating surrogate findings).
Questions obviously remain. How will the newest version of PDUFA (Prescription Drug User Fee Act) factor into this review? The reauthorized bill (an important piece of FDA legislation that the community rallied behind this year) contained provisions strengthening accelerated review. It also contained a provision to work on speeding up the development of "breakthrough therapies" that demonstrate early promise. How these are applied remains to be seen. PPMD and others in the community are wanting answers and will work tirelessly to get them so we have as much clarity as possible on the path forward.
*Be sure to check out our upcoming webinar with Serepta where participants have the opportunity to ask questions directly with the company. We encourage you to submit questions prior to the webinar via email (firstname.lastname@example.org).
This is an exciting time for the Duchenne community. On their recent call with investors on October 3, Sarepta's CEO Chris Garabedian stated their intent to discuss accelerated approval with the FDA at their meeting later this year. Though the community is anxious to move things along faster, much of this will depend on that meeting and how the FDA views the data.
We all eagerly await the results from the full data presentation by Sarepta at World Muscle Society next week. And we will make sure you stay abreast of the role you can play.
Ryan Fischer, Director of Outreach & Advocacy
Read more PPMD Staff Blogs