Currentsierra health and lifePolicy 2025D0088H

Vyondys 53 (golodirsen) — Coverage Criteria for Duchenne Muscular Dystrophy (exon 53)

Defines coverage criteria, requirements for initial and continued authorization, and coding for golodirsen (Vyondys 53) for members with DMD amenable to exon 53 skipping; applies to commercial/community plan benefit determinations.

Policy Summary

Payersierra health and life

PolicyVyondys 53 (golodirsen) — Coverage Criteria

Policy CodePolicy 2025D0088H

Change TypeCriteria added and functional thresholds clarified

Effective DateJul 1, 2025

Next Review DateN/A

Key ActionSubmit prior authorization with medical records documenting exon 53–amenable mutation and required baseline functional measures (e.g., 6MWT ≥300 m or ambulatory with NSAA>17 or TTR<7s).

SourceLink

POLICY UPDATE CHANGES

Added criterion requiring either no prior gene therapy or documentation of clinically meaningful functional decline since receiving gene replacement therapy for patients who previously received gene therapy.

Revised coverage criteria for initial therapy (updated ambulatory and functional assessment requirements).

Updated Benefit Considerations section to reflect most current information.

≈8%DMD patients amenable to exon 53 skipping

J1429HCPCS code

6MWT ≥300 mInitial functional threshold

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Coverage Criteria for Vyondys 53 (golodirsen)

Initial Therapy

Covered when ALL of the following are met for initial therapy:

Initial Therapy - core requirements: Diagnosis of Duchenne muscular dystrophy by, or in consultation with, a neurologist with expertise in the diagnosis of DMD; and submission of medical records confirming the DMD gene mutation is amenable to exon 53 skipping; and dosing per FDA labeling; and initial authorization for no more than 12 months.

Functional eligibility (one required): Either: submission of medical records confirming the patient has a 6‑Minute Walk Test (6MWT) ≥ 300 meters while walking independently prior to beginning Vyondys 53 therapy; OR both: submission of records confirming the patient is ambulatory without needing an assistive device AND one of the following: NSAA score > 17 OR time to rise (TTR) < 7 seconds.6MWT ≥ 300 m; NSAA > 17; TTR < 7 s

Gene therapy history (one required): Either: patient has not previously received gene therapy (e.g., Elevidys); OR both: patient previously received gene therapy and submission of medical records documenting a clinically meaningful functional decline (e.g., loss of a motor milestone) since receiving gene replacement therapy.

Added in 07/01/2025 revision

Therapy concurrency restriction: Vyondys 53 is not used concomitantly with other exon skipping therapies for DMD (e.g., Amondys, Exondys 51, Viltepso).

Continuation Therapy

Covered when ALL of the following are met for continuation (reauthorization):

Continuation Therapy - requirements: Submission of medical records confirming the patient is ambulatory without needing an assistive device; Vyondys 53 is not used concomitantly with other exon skipping therapies for DMD; prescribed by, or in consultation with, a neurologist with expertise in the treatment of DMD; dosing in accordance with FDA labeling; reauthorization for no more than 12 months.

Unproven / Not Medically Necessary

Unproven/Not medically necessary uses: Use of Vyondys 53 for other forms of muscular dystrophy (e.g., Becker muscular dystrophy) is unproven and not medically necessary.

Vyondys 53 (golodirsen) is not covered for patients whose DMD gene mutations are not amenable to exon 53 skipping or for other muscular dystrophies. The policy explicitly excludes use concomitantly with other exon‑skipping therapies for DMD (for example, Amondys, Exondys 51, Viltepso). Providers must document an exon 53‑amenable mutation and meet the policy’s specified functional and documentation requirements for coverage consideration.

Use of Vyondys 53 for other forms of muscular dystrophy, such as Becker muscular dystrophy, is considered not medically necessary and therefore not covered under this policy.

Applicable Codes and Functional Thresholds

Applicable HCPCS CodesHCPCSCovered

J1429

Injection, golodirsen, 10 mg.

Applicable Diagnosis CodesICD-10

G71.01

Duchenne or Becker muscular dystrophy

inv-08: 6-Minute Walk Test (6MWT) — initial therapy threshold and study inclusion reference

Initial authorization functional threshold6-Minute Walk Test (6MWT) ≥ 300 meters while walking independently prior to starting therapy (documented in medical records)

Clinical trial inclusion referenceSKIP‑NMD trial used baseline 6MWT > 250 meters as an inclusion criterion (Servais et al., 2022)

Alternative functional criteriaAmbulatory without assistive device plus either NSAA > 17 or time to rise (TTR) < 7 seconds (as alternative to 6MWT ≥ 300 m)

Prior Authorization, Documentation, and Concurrency Rules

Prior Authorization

Prior Authorization Required

Prior authorization is required for Vyondys 53. Initial authorization will be for no more than 12 months; reauthorization (continuation) will be for no more than 12 months. Prescriptions must be by, or in consultation with, a neurologist with expertise in the treatment of Duchenne muscular dystrophy (DMD).

Initial and continuation authorizations: up to 12 months each
Prescriber requirement: neurologist with DMD expertise
Authorization required prior to drug dispensing

Step Therapy

Concomitant Therapy Restriction

Vyondys 53 must not be used concomitantly with other exon skipping therapies for DMD. Examples of exon skipping therapies that must not be used at the same time include Amondys (casimersen), Exondys 51 (eteplirsen), and Viltepso (viltolarsen).

Concomitant use with other exon skipping therapies is prohibited: Amondys (casimersen), Exondys 51 (eteplirsen), Viltepso (viltolarsen)

Key Definitions and Functional Status

inv-14: Exon 53 amenable mutation — Definition

DefinitionA DMD gene deletion or mutation configuration predicted to allow restoration of the reading frame by skipping exon 53, enabling production of an internally truncated dystrophin protein.

Clinical applicabilityApplies to patients with confirmed DMD gene mutations demonstrated by genetic testing and documented as amenable to exon 53 skipping (≈8% of DMD patients)

Coverage requirementSubmission of medical records confirming the mutation is amenable to exon 53 skipping is required for authorization

inv-15: Ambulatory without assistive device — Definition

DefinitionPatient can walk independently without side‑by‑side assist, cane, walker, or wheelchair (i.e., ambulatory without needing an assistive device), as documented in medical records.

Clinical Background

Duchenne muscular dystrophy (DMD) is an X‑linked, progressive muscle‑wasting disorder caused by mutations in the dystrophin gene that lead to absent or defective dystrophin and loss of ambulation in childhood with progressive cardiopulmonary complications. Antisense oligonucleotide exon‑skipping therapies such as golodirsen (Vyondys 53) target specific mutations to restore the dystrophin reading frame; golodirsen is directed at mutations amenable to exon 53 skipping and is intended for use only when the patient’s genotype and functional status meet the policy’s criteria.

Policy Revision History

2025-07-01material_revisionLatest

Revised coverage criteria for initial therapy to add gene therapy history requirement: either no prior gene therapy (e.g., Elevidys) or documentation of clinically meaningful functional decline since prior gene replacement therapy; clarified functional eligibility thresholds (6MWT ≥300 m; NSAA >17; TTR <7s)

2025-07-01supporting_update

Updated Benefit Considerations section to reflect the most current information