Eteplirsen (Exondys 51®)

Although Exondys 51 (eteplirsen) has FDA accelerated approval for DMD with a DMD gene mutation amenable to exon 51 skipping, this policy does not recommend approval because clinical benefit has not been established. If considered, approval requires documented genetic confirmation of an exon 51–amenable mutation and clinical records showing the patient meets the policy’s coverage and applicable safety criteria.

"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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