Vyondys 53 (golodirsen)

Coverage Summary

Scope: This is a medical-benefit prior authorization policy for Vyondys 53 (golodirsen) for treatment of Duchenne muscular dystrophy in members with a confirmed DMD gene mutation amenable to exon 53 skipping. The policy summarizes initial and continuation criteria, dosing, limitations, and lists amenable exon deletions. Vyondys 53 is also available on the pharmacy benefit per the MassHealth Drug List.

Coverage stance: covered_with_criteria. Effective date: 06/01/2025. Last review: 05/15/2025.

Initial Therapy Criteria

Initial Authorization (Authorization may be granted when ALL of the following are met)

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

Initial Authorization criteria

Diagnosis and genetic eligibility: 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 include: 42-52, 45-52, 47-52, 48-52, 49-52, 50-52, 52, 54-58
Prescriber requirement: The prescribing physician is a neuromuscular neurologist or consult notes from a neuromuscular neurology office are provided
Ambulatory status (6MWT): The member is ambulatory as defined by a current six-minute walk test (6MWT - distance walked in six minutes in meters) of ≥ 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)6MWT >= 200 meters
Dosing is appropriate (30 mg/kg intravenously every week)30 mg/kg weekly
Corticosteroid use: Member has received a corticosteroid for at least 6 months prior and member will continue to use a corticosteroid in combination with the requested agent OR a demonstrated contraindication to corticosteroids
Document at least 6 months prior corticosteroid use or contraindication
Baseline timed function tests
- Timed tests list: Timed ten-meter walk/run (time in seconds)
- Timed tests list: Timed floor (supine) to stand (time in seconds)
- Timed tests list: Timed four-step descend (time in seconds)
- Timed tests list: Timed four-step climb (time in seconds)
- Timed tests list: Timed sit to stand (time in seconds)

Continuation Therapy Criteria / Reauthorization

Continuation of Therapy / Reauthorization (ALL of the following)

Reauthorizations may be approved when ALL the following is met:

Reauthorization criteria

Ambulatory status (6MWT): The member remains ambulatory as defined by a current 6MWT of ≥ 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)6MWT >= 200 meters
6MWT trajectory: 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 6MWTs results must be included)
Include pretreatment baseline and all interim results

Authorization for Members New to the Plan

Authorization for members new to the plan currently receiving treatment

Authorization may be reviewed on a case by case basis for members who are new to the plan currently receiving treatment with requested medication excluding when the product is obtained as samples or via manufacturer's patient assistance programs.

New-to-plan treatment review: Case-by-case review may be performed for members new to the plan currently receiving treatment (excludes products obtained as samples or via manufacturer's patient assistance programs)

Provider Actions

Prior Authorization

Prior authorization required (medical benefit)

Prior authorization is required for Vyondys 53 when billed to the medical benefit. Authorizations may be reviewed on a case-by-case basis for members new to the plan who are currently receiving treatment; this review excludes products obtained as samples or via the manufacturer's patient assistance programs.

Documentation Required

Documentation of mutation and neuromuscular specialist

Provide documentation of a confirmed out-of-frame DMD deletion that is amenable to exon 53 skipping. The prescribing physician must be a neuromuscular neurologist or you must supply consult notes from a neuromuscular neurology office.

Applicable Codes

Amenable Exon Deletions (clinical eligibility list)mixed

42-52	Amenable exon deletion range for exon 53 skipping
45-52	Amenable exon deletion range for exon 53 skipping
47-52	Amenable exon deletion range for exon 53 skipping
48-52	Amenable exon deletion range for exon 53 skipping
49-52	Amenable exon deletion range for exon 53 skipping
50-52	Amenable exon deletion range for exon 53 skipping
52	Amenable single exon deletion for exon 53 skipping
54-58	Amenable exon deletion range for exon 53 skipping

Amenable Exon Deletions (clinical eligibility list)mixed

42-52	Amenable exon deletion range for exon 53 skipping
45-52	Amenable exon deletion range for exon 53 skipping
47-52	Amenable exon deletion range for exon 53 skipping
48-52	Amenable exon deletion range for exon 53 skipping
49-52	Amenable exon deletion range for exon 53 skipping
50-52	Amenable exon deletion range for exon 53 skipping
52	Amenable single exon deletion for exon 53 skipping
54-58	Amenable exon deletion range for exon 53 skipping

Clinical Evidence & References

Key evidence and references: Prescribing information — Vyondys 53 (golodirsen) prescribing information, Sarepta Therapeutics Inc; Jun 2024.

Clinical study: Frank DE et al., Neurology 2020; 94:e2270 showed increased dystrophin production with golodirsen.

Regulatory note: The FDA granted accelerated approval for a targeted treatment for DMD mutations amenable to exon 53 skipping.

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. The policy applies to medical-benefit use; the drug is also available on the pharmacy benefit per the MassHealth Drug List.

Definitions: Ambulatory is defined by a current six-minute walk test (6MWT) distance of ≥ 200 meters (observed/completed by the treating provider or ordered and completed by a qualified medical practitioner). Amenable to exon 53 skipping includes DMD gene deletions: 42-52, 45-52, 47-52, 48-52, 49-52, 50-52, 52, 54-58.

6-Minute Walk Test>= 200 meters

Required dosing30 mg/kg weekly

Initial/Reauthorization Approval Duration3 months

Timed function tests required at baseline5

Revision History

05/15/2025administrativeLatest

Review updated formatting and references; no material clinical changes. Effective 06/01/2025.

02/08/2023policy_change

Material change: approval durations set to 3 months; added requirement for baseline measurements of timed function tests; prescribing physician specified as neuromuscular neurologist; requirement for appropriate dosing (30 mg/kg IV weekly). Effective 04/01/2023.