Eteplirsen (Exondys 51®)

Eteplirsen (Exondys 51) is FDA‑approved for DMD patients with a confirmed DMD gene mutation amenable to exon 51 skipping, but the policy does not recommend approval because clinical benefit has not been established and continued approval is contingent on confirmatory trials. Coverage is limited to the FDA‑approved indication and any applicable coverage/safety criteria (confirmed exon‑51‑amenable mutation required); no additional explicit documentation, prior‑therapy, or frequency limits are specified in this policy.

"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."

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