Exondys 51™ (eteplirsen)

Exondys 51 (eteplirsen) has an FDA accelerated approval for DMD patients with a confirmed DMD gene mutation amenable to exon 51 skipping, but eviCore does not recommend approval because clinical benefit has not been established. Coverage would require documentation of DMD diagnosis and genetic confirmation of an exon‑51–amenable mutation, adherence to specific coverage/safety criteria, and continued approval may depend on confirmatory trial results.

"Exondys 51 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. This indication is app..."

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