Olecranon, I share your general concern about Sarepta.
I think for EXONDYS 51 it is highly unlikely the FDA will ask for a confirmatory trial. MISSION, the current post-marketing commitment, which the FDA specified, is not a confirmatory trial; it is a dose-ranging study.
CEO Ingram said this during Monday's Q3 conference call:
I believe the readout date for MISSION is 2026. So to your very good point, MISSION is a post-marketing commitment that we have with respect to EXONDYS, but unlike ESSENCE and unlike VYONDYS and AMONDYS, it is not a confirmatory study. So there is no confirmatory study for EXONDYS and that was purposeful. The FDA in some of their memos indicated that what they really wanted to see was not a confirmation study, but rather a dose-ranging study. So there's -- essentially, it's dose ranging between 30 mg per kg, 100 mg per kg, even up to 200 mg per kg. And so the result of that is really a dose. Is 30 mg per kg the optimal dose or is 100 mg per kg the optimal dose? So that's the consequence of that study. It's not a confirmatory study, and that will read out in 2026, and we'll go from there. In terms of expanding the Elevidys label to non-ambulatory patients (again), if they are able to conclude discussions with the FDA and submit the protocol using sirolimus, Rodino-Klapac said
So as soon as we get that study started, we are ready to enroll quickly. Patients are lined up. And so by -- as you mentioned, in the first half of next year, we'll start to have data on the effectiveness and then in the second half of the year is when we'll have the full data set for the trial based on starting the trial soon. So we're hoping to get that initiated in the near term. They have a small amount of data already on the use of sirolimus, which they have said is positive. But they wouldn't submit an application for label expansion to the non-ambulatory population until the end of 2026 at the earliest. |
