Exondys 51 (eteplirsen)

Exondys 51 is FDA‑approved for treating Duchenne muscular dystrophy with a confirmed DMD gene mutation amenable to exon 51 skipping, but EviCore does not recommend approval/coverage because clinical benefit has not been established and continued FDA approval is contingent on confirmatory trials. Prior authorization requires documentation of a DMD diagnosis, genetic testing confirming an exon‑51–skippable mutation, and meeting EviCore’s specific coverage and safety criteria (requests failing these criteria may be denied).

"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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