VYONDYS 53 (golodirsen)

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