CurrentMass General Brigham Health PlanPolicy N/A

Duchenne Muscular Dystrophy Agents Vyondys 53 (golodirsen) Effective 06/01/2025

Pharmacy benefit prior authorization policy for Vyondys 53 (golodirsen) for treatment of Duchenne muscular dystrophy in members with DMD gene mutations amenable to exon 53 skipping; includes initial and continuation criteria, dosing, required functional testing, approval durations, and eligible exon deletions.

Policy Summary

PayerMass General Brigham Health Plan

PolicyDuchenne Muscular Dystrophy Agents Vyondys 53 (golodirsen) Effective 06/01/2025

Policy CodePolicy N/A

Change TypeFormatting and reference updates (non-clinical)

Effective DateJun 1, 2025

Next Review Date

Key ActionPrior authorization required; provide documentation supporting criteria (genetic confirmation, baseline timed function tests, specialist consult and appropriate dosing) and note approvals are granted for 3 months.

SourceLink

POLICY UPDATE CHANGES

Reviewed and updated for P&T. Updated formatting and references. Effective 6/1/25.

Updated approval durations to 3 months, added requirement of baseline measurements, updated neurologist requirement to neuromuscular neurologist, and requirement of appropriate dosing (effective 4/1/23).

8Amenable Exon Deletions

3 moApproval Duration (initial/reauth)

>=200 m6MWT Ambulatory Threshold

Coverage Summary

Scope: Pharmacy benefit prior authorization policy for Vyondys 53 (golodirsen) for treatment of Duchenne muscular dystrophy in patients with DMD gene mutations amenable to exon 53 skipping. Policy includes initial and continuation criteria, dosing, required functional testing, approval durations, and eligible exon deletions. (Mass General Brigham Health Plan; Policy title: Duchenne Muscular Dystrophy Agents — Vyondys 53 (golodirsen); Effective date: 2025-06-01).

Coverage stance: Covered with criteria — authorization may be granted when all specified criteria are met or reviewed case-by-case for members new to the plan currently receiving treatment.

Payer/policy note: This policy aligns with MassHealth UPPL and references the Vyondys 53 prescribing information and pivotal clinical data in support of coverage criteria.

Quick facts and key thresholds

6MWT thresholdAmbulatory defined as six-minute walk test (6MWT) >= 200 meters

Dosing30 mg/kg intravenously every week

Approval / Reauthorization durationInitial approvals and reauthorizations granted for 3 months

Amenable exon deletions (exon 53 skipping)42-52; 45-52; 47-52; 48-52; 49-52; 50-52; 52; 54-58

IndicationFor treatment of Duchenne muscular dystrophy with DMD gene mutations amenable to exon 53 skipping

Documentation requirementsConfirmed out-of-frame DMD deletion amenable to exon 53 skipping and observed/completed baseline timed function tests

Initial Authorization Criteria

Initial Authorization

Authorization may be granted when ALL the following criteria are met, and documentation is provided:

ALL of the following

Diagnosis of Duchenne Muscular Dystrophy
Genetic confirmation: Documentation of a confirmed out of frame deletion in the DMD that is amenable to exon 53 skipping
Amenable exon deletions listed in policy (e.g., 42-52, 45-52, 47-52, 48-52, 49-52, 50-52, 52, 54-58)
Prescriber: The prescribing physician is a neuromuscular neurologist or consult notes from a neuromuscular neurology office are provided
Ambulatory status: The member is ambulatory as defined by a current six-minute walk test (6MWT distance walked in six minutes in meters) of ≥ 200 meters>= 200 meters
Test must have been observed or completed by the treating provider, or ordered by the treating provider and completed by a qualified medical practitioner
Corticosteroid use: Member has received corticosteroids for at least 6 months prior and member will continue to use a corticosteroid in combination with the requested agent OR there is a demonstrated contraindication to corticosteroids
Dosing is appropriate (30 mg/kg intravenously every week)30 mg/kg IV weekly
Baseline timed function tests
- Timed tests list
  - Timed ten-meter walk/run (time in seconds)
  - Timed floor to stand: Timed floor (supine) to stand (time in seconds)
  - Timed four-step descend (time in seconds)
  - Timed four-step climb (time in seconds)
  - Timed sit to stand (time in seconds)

Continuation (Reauthorization) Criteria

Reauthorizations may be approved when ALL the following is met:

ALL of the following

Test documentation: Documentation of observed or completed tests by the treating provider, or ordered by the treating provider and completed by a qualified medical practitioner
All results with dates must be provided; tests must be observed/completed as noted
6MWT pattern: The member has a stable or improving pattern of 6MWTs as shown in medical records with results of a pretreatment baseline and all interim results (all previous 6MWT results must be included)
Dosing remains appropriate (30 mg/kg intravenously every week)30 mg/kg IV weekly
Corticosteroid use: The member continues to utilize corticosteroids in combination with the requested agent OR there is a demonstrated contraindication to corticosteroids
Functional performance
- At least two timed tests
  - Timed ten-meter walk/run (time in seconds)
  - Timed floor to stand: Timed floor (supine) to stand (time in seconds)
  - Timed four-step climb (time in seconds)
  - Timed four-step descend (time in seconds)
  - Timed sit to stand (time in seconds)

Billing Rule

Approval duration

Initial approvals and reauthorizations will be granted for 3 months. Authorization may be reviewed case-by-case for members who are new to the plan and are currently receiving the medication.

Initial and reauthorization = 3 months
Case-by-case review for members already receiving medication

Provider Actions / Requirements

Prior Authorization

Prior Authorization Requirement

Prior authorization is required. Documentation supporting the criteria must be provided. Authorization may be reviewed on a case-by-case basis for members who are new to the plan and are currently receiving the medication.

Documentation Required

Documentation of genetic mutation

Provide documentation of a confirmed out-of-frame deletion in the DMD gene that is amenable to exon 53 skipping. The policy provides a list of amenable deletions.

Documentation Required

Baseline and ongoing timed function tests

Include baseline measurements for all five timed function tests. For reauthorization, include all prior and interim 6MWT and timed test results with dates. Tests must have been observed or completed by the treating provider, or ordered by the treating provider and completed by a qualified medical practitioner.

Documentation Required

Specialist prescriber or consult

The prescribing physician must be a neuromuscular neurologist or consult notes from a neuromuscular neurology office must be provided.

Clinical Evidence & References

Evidence: Prescribing information — Vyondys 53 (golodirsen) prescribing information, Sarepta Therapeutics Inc; Jun 2024.

Clinical study citation: Frank DE et al. Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy: Neurology 2020; 94:e2270.

Policy alignment: This policy aligns with MassHealth UPPL and references the prescribing information and pivotal data noted above.

Note: Do not duplicate metrics already presented in policy statistics (e.g., amenable exon deletions list, 6MWT threshold, dosing) — see policy sections for those details.

Background

Background: Vyondys 53 (golodirsen) is an antisense oligonucleotide indicated for treatment of Duchenne muscular dystrophy in patients with a confirmed DMD gene mutation that is amenable to exon 53 skipping.

Policy purpose: This is a pharmacy benefit prior authorization policy that establishes clinical and documentation requirements for initial and continuation approvals for Vyondys 53, including specialist prescriber requirements, dosing, baseline and ongoing functional testing, and approval durations.

Included elements: Initial and reauthorization criteria (diagnosis, confirmed amenable out-of-frame deletion, neuromuscular neurologist prescriber or consult, ambulatory status defined by a baseline 6MWT ≥ 200 meters, corticosteroid use or contraindication, and appropriate dosing 30 mg/kg IV weekly), requirement for baseline measurements on five timed function tests, reauthorization requirements for stability or improvement on 6MWT and at least two of five timed tests, and an amenable exon deletions list (exon 53 skipping deletions).

Approval duration and logistics: Initial approvals and reauthorizations are granted for 3 months. The policy may be reviewed case-by-case for members new to the plan who are currently receiving the medication.

Documentation and prescriber requirements: Documentation of a confirmed out-of-frame DMD deletion amenable to exon 53 skipping and baseline/ongoing observed timed function tests (observed/completed by treating provider or qualified medical practitioner) must be provided; prescribing physician must be a neuromuscular neurologist or provide consult notes.

Ambulatory (policy-specific) definition

Ambulatory (policy-specific) definitionDefined as a current six-minute walk test (6MWT) distance >= 200 meters

Observation/completion requirement6MWT must be observed or completed by the treating provider, or ordered by the treating provider and completed by a qualified medical practitioner

Revision History

2025-06-01effective updateLatest

Reviewed and updated for P&T. Updated formatting and references. Effective 6/1/25.

2023-02-08prior material clinical changes

Policy Summary

PayerMass General Brigham Health Plan

PolicyDuchenne Muscular Dystrophy Agents Vyondys 53 (golodirsen) Effective 06/01/2025

Policy CodePolicy N/A

Change TypeFormatting and reference updates (non-clinical)

Effective DateJun 1, 2025

Next Review Date

SourceLink

On This Page

OpenPayer

Duchenne Muscular Dystrophy Agents Vyondys 53 (golodirsen) Effective 06/01/2025

Coverage Summary

Initial Authorization Criteria

Continuation (Reauthorization) Criteria

Provider Actions / Requirements

Applicable Codes

Clinical Evidence & References

Background

Revision History