CurrentMass General Brigham Health PlanPolicy N/A

Viltepso (vitolarsen) — Prior Authorization and Coverage Criteria

Prior authorization and coverage criteria for Viltepso (vitolarsen) for treatment of Duchenne muscular dystrophy in members whose DMD gene mutation is amenable to exon 53 skipping; applies to Mass General Brigham Health Plan members and notes medical and pharmacy benefit considerations.

Policy Summary

PayerMass General Brigham Health Plan

PolicyViltepso (vitolarsen) — Prior Authorization and Coverage Criteria

Policy CodePolicy N/A

Change TypeNo material change

Effective DateJun 1, 2025

Next Review DateN/A

Key ActionObtain prior authorization and submit documentation including confirmed DMD diagnosis with genetic testing showing an out-of-frame deletion amenable to exon 53 skipping, prescriber specialty or consult notes, baseline and interim 6MWT and timed function test results, and evidence of corticosteroid use for ≥3 months or documented contraindication.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

3 monthsApproval duration

80 mg/kg wklyDose

6MWT ≥200mAmbulatory requirement

5 timed testsBaseline tests

≥3 months

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Coverage Criteria for Viltepso (vitolarsen)

Initial authorization — ALL required

Authorization may be granted when ALL of the following criteria are met:

ALL of the following

Diagnosis of Duchenne muscular dystrophy (DMD).
Confirmed out-of-frame deletion in the DMD gene that is amenable to exon 53 skipping.
Prescriber is a neuromuscular neurologist, or consult notes from a neuromuscular neurology office are provided.
Member is ambulatory as defined by a current six-minute walk test (6MWT) distance ≥ 200 meters. Tests must have been observed or completed by the treating provider, or ordered by the treating provider and completed by a qualified medical practitioner.≥ 200 meters
Dosing is appropriate: 80 mg/kg administered intravenously every week.
Member has received a corticosteroid for at least 3 months prior to initiation and documentation that the member will continue to use a corticosteroid in combination with the requested agent, or there is a demonstrated contraindication to corticosteroids.
- Timed 10-meter walk/run (time in seconds).
- Timed floor (supine) to stand (time in seconds).
- Timed 4-step descend (time in seconds).
- Timed 4-step climb (time in seconds).
- Timed sit-to-stand (time in seconds).

Reauthorization — ALL required

Reauthorization may be granted when ALL of the following criteria are met:

ALL of the following

Member remains ambulatory as defined by a current six-minute walk test (6MWT).
Member has a stable or improving pattern of 6MWT results documented in the medical record, including the pretreatment baseline and all interim results; all previous 6MWT results must be included.
Dosing remains appropriate (80 mg/kg IV weekly).
Member continues to take a corticosteroid in combination with the requested agent, or there is a demonstrated contraindication to corticosteroids.
- Timed 10-meter walk/run (time in seconds).

Authorization may be reviewed for members new to the plan who are currently receiving treatment with Viltepso; however, approvals do not apply when the product is obtained as samples or via the manufacturer's patient assistance program.

Initial approvals and reauthorizations will be granted for 3 months.

Functional Thresholds & Coding Details

Six-minute walk test (6MWT) — threshold

6MWT ambulatory thresholdMember must be ambulatory as defined by a current six-minute walk test (6MWT) with distance ≥ 200 meters; tests must be observed or completed by the treating provider or ordered and completed by a qualified medical practitioner.

Use for reauthorizationReauthorization requires the member remains ambulatory by current 6MWT and shows a stable or improving pattern of 6MWTs compared with baseline; include pretreatment baseline and all interim results.

Documentation requirementsAll 6MWT results must be included with dates and observation documentation (baseline and interim); tests may be ordered and completed by a qualified medical practitioner.

Provider Actions, Prior Authorization, and Documentation

Prior Authorization

Prior authorization required — medical vs pharmacy benefit

Prior authorization is required for Viltepso on the medical benefit; Viltepso is also available on the pharmacy benefit and may have coverage and prior authorization requirements per the MassHealth Drug List.

Medical benefit: prior authorization required for Viltepso.
Pharmacy benefit: Viltepso is available on the pharmacy benefit; see the MassHealth Drug List for coverage and criteria.

Step Therapy

Step therapy not required

No step therapy requirements are specified for Viltepso in this document.

Key Definitions

Amenable to exon 53 skipping — definition

DefinitionA confirmed out-of-frame deletion in the DMD gene that is specifically responsive to exon 53 skipping therapy (i.e., the DMD mutation is amenable to exon 53 skipping).

Required evidenceGenetic testing demonstrating a confirmed out-of-frame deletion in the DMD gene that is amenable to exon 53 skipping must be provided with the authorization request.

Therapy applicabilityViltepso (vitolarsen) is indicated for DMD patients whose DMD gene mutation is amenable to exon 53 skipping; eligibility hinges on this specific genetic finding.

Background

Duchenne muscular dystrophy (DMD) is a rapidly progressive neuromuscular disease caused by defects in the dystrophin gene. Viltepso (vitolarsen) is indicated for patients with a confirmed DMD gene mutation that is amenable to exon 53 skipping, and the policy applies to both medical- and pharmacy-benefit access pathways.

For authorization consideration, providers should document diagnosis and genetic testing demonstrating an out-of-frame deletion amenable to exon 53 skipping, confirm ambulatory status (with 6MWT ≥ 200 meters), verify corticosteroid use for ≥ 3 months or a contraindication, and follow the recommended dosing of 80 mg/kg IV weekly. Initial and reauthorization approvals are granted for 3 months.

Documentation Required

Required documentation to support authorization

Provide documentation that demonstrates: confirmed diagnosis and genotype, prescriber specialty, baseline and interim functional measures, corticosteroid use status, and dosing.

Confirmed diagnosis of Duchenne muscular dystrophy and genetic testing showing a confirmed out‑of‑frame deletion in the DMD gene amenable to exon 53 skipping.
Prescriber is a neuromuscular neurologist or provide neuromuscular neurology consult notes.
Baseline measurements and dates for ALL five timed function tests (timed 10‑meter walk/run; timed floor‑to‑stand; timed 4‑step descend; timed 4‑step climb; timed sit‑to‑stand); tests must be observed or completed by the treating provider or ordered and completed by a qualified medical practitioner.
Current six‑minute walk test (6MWT) result showing ambulatory status (≥ 200 meters) with test dates and all results.
Evidence member has received a corticosteroid for at least 3 months prior and will continue corticosteroid in combination with the requested agent, or documentation of a contraindication to corticosteroids.
Documentation of appropriate dosing: 80 mg/kg IV every week.