VYONDYS 53 (golodirsen)

OFFICE ADMINISTRATION - IV

Indications for Prior Authorization
  • 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 53 skipping.

Patient must meet the following criteria for the indication(s) above:
  • Patient is 6 years of age or older (age ≤ 15 years at therapy initiation), AND
  • Diagnosis of DMD with mutation amenable to exon 53 skipping confirmed by genetic testing (lab results from genetic testing are required), AND
  • Prescribed by or in consultation with a neurologist, AND
  • Patient is ambulatory as documented by the 6-minute walk test or North Star ambulatory assessment (average distance of at least 250 m while walking independently)
Reauthorization criteria:
  • Patient meets one of the following criteria sets:
    • All of the following:
      • Patient has been on therapy for less than 12 months, AND
      • Patient is tolerating therapy, AND
      • Medical records (e.g., chart notes, laboratory values) documenting the patient is maintaining ambulatory status (e.g., 6-minute walk test, North Star ambulatory assessment), OR
    • All of the following:
      • Patient has been on therapy for 12 months or more, AND
      • Patient has experienced a benefit from therapy (e.g., disease amelioration compared to untreated patients), AND
      • Patient is tolerating therapy, AND
      • Medical records (e.g., chart notes, laboratory values) documenting the patient is maintaining ambulatory status (e.g., 6-minute walk test, North Star ambulatory assessment)
Dosing:
  • 30 mg/kg intravenous infusion administered once weekly
  • Dose will not exceed 30 mg/kg infused once weekly
Approval:
  • Initial: 6 months
  • Reauthorization: 1 year

 

Last review date: April 13, 2021

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