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    Exondys 51

    Drug  - Exondys 51™ (eteplirsen) [Sarepta Therapeutics, Inc.]

    June 2017

    Therapeutic area - Duchene muscular dystrophy

    Approval criteria

    • Patient must have a diagnosis of one of Duchene muscular dystrophy (DMD) AND
    • Patient must have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping AND
    • Must be prescribed by a provider specializing in genetics or neurology AND
    • Provider’s specialty must be provided at time of request
    • At time of request, prescriber must confirm whether or not the patient is currently enrolled in clinical trials for Exondys 51

    Quantity limit

    • 30 mg/kg once weekly
    • Patient’s most current weight (rounded to the nearest kg) must be provided at time of request

    Approvals

    Initial approval will be limited to 6 months in duration.
    Renewal approval will be limited to 6 months in duration. 

    • Renewals must be prescribed by a provider specializing in genetics or neurology AND
    • Provider’s specialty must be provided at time of request AND
    • Chart notes must be supplied at time of request showing patient is responsive to treatment defined as:
      • Maintain or increase in physical function from baseline OR
      • Progression has been slower than otherwise would have been expected in this patient population

    Denial criteria

    Patient is currently enrolled in clinical trials for Exondys 51

    Background information

    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.

    Questions?

    MHCP Provider Call Center 651-431-2700 or 800-366-5411

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