- The approval is based on ESSENCE trial assessing Amondys 45 vs PBO in patients with DMD with a confirmed mutation amenable to exon 45 skipping. The study is expected to conclude in 2024
- The study resulted in a significant increase in dystrophin production in skeletal muscle observed in patients treated with Amondys 45. Consistent with the accelerated approval, the continued approval of Amondys 45 may contingent on confirmation of clinical benefit in a confirmatory trial.
- Amondys 45 is the third RNA exon-skipping treatment for DMD approved in the US. The company will initiate the commercial distribution of therapy in the US imminently
Click here to read full press release/ article | Ref: Sarepta | Image: Sarepta