Vyondys 53™

Drug - Vyondys 53™ (golodirsen) [Sarepta Therapeutics Inc.]

April 2020

Therapeutic area - Duchene Muscular Dystrophy

Initial approval criteria

• Patient must have documentation of a confirmed diagnosis of Duchenne muscular dystrophy (DMD) with genetic testing demonstrating a mutation on the DMD gene that is amenable to exon 53 skipping AND
• Patient has been on a stable dose of corticosteroids, unless contraindicated or intolerance, for ≥ 6 months AND
• Patient retains meaningful voluntary motor function (patient is able to speak, manipulate objects using upper extremities, ambulate, etc.) AND
• Patient has documented ongoing physical therapy and/or occupational therapy AND
• Baseline documentation of ≥ 1 of the following:
  • Dystrophin level
  • 6-minute walk test (6WMT) or other timed function tests
  • Upper limb function (ULM) test
  • North Star Ambulatory Assessment (NSAA)
  • Forced Vital Capacity (FVC) % predicted
• Baseline glomerular filtration rate (GFR) by 24-hour urine collection and renal function monitoring plan must be submitted at time of request
• Initial approval is for 6 months

Renewal criteria

• Patient must continue to meet the above criteria AND
• Patient has demonstrated a response to therapy compared to pretreatment baseline in ≥ 1 of the following (not all-inclusive):
  • Increase in dystrophin level
  • Stability, improvement, or slowed rate of decline in 6MWT or other timed function tests
  • Stability, improvement, or slowed rate of decline in ULM test
  • Stability, improvement, or slowed rate of decline in NSAA
  • Stability, improvement, or slowed rate of decline in FVC% predicted
  • Improvement in quality of life AND
• Documentation of monthly renal function monitoring is submitted at time of request AND
• Patient has not experienced any treatment-restricting adverse effects (severe hypersensitivity reactions, renal toxicity/proteinuria, etc.)
• Renewal approval is for 6 months

Quantity limits

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

Background

A clinical benefit of VYONDYS 53 has not been established. VYONDYS 53 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 53 skipping. This indication is approved under accelerated approval based on an increase in dystrophin in skeletal muscle observed in some patients treated with VYONDYS 53. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.

VYONDYS 53 must be administered intravenously by a qualified healthcare professional. VYONDYS 53 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