Viltepso™

Drug - Viltepso™ (viltolarsen injection) [NS Pharma Inc.]

March 2021

Therapeutic area - Duchene Muscular Dystrophy (DMD)

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 ≥ 3 months AND
Patient retains meaningful voluntary motor function (e.g., patient is able to speak, manipulate objects using upper extremities, ambulate) AND
Patient should be receiving physical therapy and/or occupational therapy AND
Patient is not on concomitant therapy with other DMD-directed antisense oligonucleotides (e.g., eteplirsen, golodirsen) AND
Patient does not have symptomatic cardiomyopathy AND
Patient serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio has been measured prior to the start of therapy AND
Prescriber attestation that serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio will be measured and during treatment (monthly urine dipstick with serum cystatin C and urine protein-to creatinine ratio every 3 months) AND
Baseline documentation of ≥ 1 of the following:
- 6-minute walk test (6MWT) or other timed function tests OR
- Upper limb function (ULM) test OR
- North Star Ambulatory Assessment (NSAA)
Initial approval is for 6 months

Renewal criteria

Patient must continue to meet above criteria AND
Patient has demonstrated a response to therapy compared to pretreatment baseline in ≥ 1 of the following (not all-inclusive):
- Stability, improvement, or slowed rate of decline in 6MWT or other timed function tests OR
- Stability, improvement, or slowed rate of decline in ULM test OR
- Stability, improvement, or slowed rate of decline in NSAA OR
- Improvement in quality of life AND
Patient has not experienced any treatment-restricting adverse effects (e.g., renal toxicities, proteinuria)
Renewal approval is for 6 months

Quantity limits

80 mg/kg once weekly
Patient’s weight (in kg) must be supplied at time of request

Billing for Viltepso

Viltepso must be billed as a medical claim.

Background

Viltepso 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 production in skeletal muscle observed in patients treated with Viltepso. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

Questions?

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