Drug - Exondys 51™ (eteplirsen) [Sarepta Therapeutics, Inc.]
Therapeutic area - Duchene muscular dystrophy
Initial approval will be limited to 6 months in duration.
Renewal approval will be limited to 6 months in duration.
Patient is currently enrolled in clinical trials for Exondys 51
A clinical benefit of EXONDYS 51 has not been established. EXONDYS 51 is FDA-approved 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 approved under accelerated approval based on an increase in dystrophin in skeletal muscle observed in some patients treated with EXONDYS 51. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.
EXONDYS 51 must be administered intravenously by a qualified healthcare professional. EXONDYS 51 is not covered through the MHCP fee-for-service pharmacy benefit and must be submitted as a medical claim.
MHCP Provider Call Center 651-431-2700 or 800-366-5411