Exondys 51™ (eteplirsen)

EviCore does not recommend approval/coverage of Exondys 51 (eteplirsen) because clinical benefit has not been established despite FDA accelerated approval for DMD patients with a DMD gene mutation amenable to exon 51 skipping. If considered, documentation of a confirmed exon‑51‑amenable DMD mutation (molecular/genetic test) and compliance with EviCore’s specific coverage and safety criteria are required, and FDA approval remains conditional pending a confirmatory trial.

"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. (FDA accelerated appro..."

Sign up to see full coverage criteria, indications, and limitations.