Sarepta Therapeutics is preparing to submit supplemental new drug applications (sNDAs) to the FDA for converting the accelerated approvals of AMONDYS 45 and VYONDYS 53 to traditional approvals, supported by data from the ESSENCE confirmatory study combined with real-world evidence.
Encouragingly, the FDA has confirmed acceptance of these data for the applications, with submission planned by the end of April.
Both therapies, approved for Duchenne muscular dystrophy (DMD) patients with specific exon mutations, have demonstrated favorable safety profiles, and the ESSENCE study evaluated their efficacy and safety in a global Phase 3 trial among children ages 6-13.
This is welcome news for the rare disease community, and an indication of the FDA’s flexibility and willingness to consider the totality of the data – especially in the case of DMD were longer term confirmatory trials are both challenging and complex.
Community Letter below:
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