CurrentEmblemHealthPolicy N/A

Vyondys 53 (golodirsen) injection

Clinical coverage policy governing prior authorization, medical necessity, dosing, renewal, and monitoring requirements for Vyondys 53 (golodirsen) for members with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping.

Policy Summary

PayerEmblemHealth

PolicyVyondys 53 (golodirsen) injection

Policy CodePolicy N/A

Change TypeMaterial updates to limitations/exclusions and authorization length

Effective Date

Next Review DateMar 2, 2025

Key ActionObtain prior authorization and document confirmed DMD mutation amenable to exon 53 skipping plus required baseline tests (including renal monitoring) before initiating therapy.

SourceLink

POLICY UPDATE CHANGES

Addition to limitations/exclusions: Patient is not on concomitant therapy with delandistrogene moxeparvovec-rokl; AND Serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio (UPCR) are measured prior to starting therapy and periodically during treatment.

Updated length of authorization from 12 months to 6 months.

Initial criteria: requirement for stable corticosteroid dose for at least 6 months, retention of meaningful voluntary motor function, receipt of physical/occupational therapy, and baseline documentation of at least one functional or biomarker measure (e.g., dystrophin level, timed function tests, NSAA, FVC).

30 mg/kg weeklydose

6 monthsauthorization length

J1429applicable J-code

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Coverage and Medical Necessity Criteria

Initial Therapy (Medical Necessity Criteria)

Covered when ALL of the following are met:

DMD diagnosis and mutation: Patient has Duchenne muscular dystrophy with a confirmed DMD gene mutation amenable to exon 53 skipping.

Corticosteroid requirement: Patient has been on a stable dose of corticosteroids for at least 6 months unless contraindicated or intolerant.>=6 months stable dose

Functional status: Patient retains meaningful voluntary motor function (e.g., able to speak, manipulate objects using upper extremities, ambulate, etc.) and is receiving physical and/or occupational therapy.

Baseline documentation of ONE or more of the following: dystrophin level; timed function tests (6-minute walk test [6MWT], time to stand [TTSTAND], time to run/walk 10 meters [TTRW], time to climb 4 stairs [TTCLIMB], etc.); upper limb function test; North Star Ambulatory Assessment (NSAA) score; Forced Vital Capacity (FVC) percent predicted.>=1 measure

Renewal Criteria

Renewal covered when ALL of the following are met:

Continuation of initial criteria: Patient continues to meet the initial approval criteria.

Absence of unacceptable toxicity: No unacceptable toxicity from the drug (examples: anaphylaxis, thrombosis, coagulopathy, severe hepatotoxicity, pancreatitis).

Response to therapy: Patient has responded to therapy compared to pretreatment baseline in one or more of the following: increase in dystrophin level; improvement in quality of life; or stability, improvement, or slowed rate of decline in timed function tests (e.g., TTSTAND, TTRW, TTCLIMB), upper limb function tests, NSAA score, or FVC percent predicted.>=1 outcome

Coverage decisions require clinically supported indications. Indications that are not supported by CMS-recognized compendia or acceptable peer‑reviewed literature may be deemed not approvable and not reimbursable. The policy also notes there are no clinical studies demonstrating use in the geriatric population (≥65 years), and therefore use in that population is unsupported. In addition, the policy requires baseline and periodic renal monitoring (see provider actions) because creatinine may be an unreliable marker of renal function in DMD patients.

Vyondys 53 is not considered medically necessary if the patient demonstrates disease progression while on the drug. Requests documenting continued clinical decline despite therapy do not meet the renewal/continued coverage requirements and therefore are not approvable under this policy.

Billing and Coding

Applicable Procedure CodesHCPCS

J1429

Injection, golodirsen, 10 mg (VYONDYS 53)

Applicable NDCsNDC

60923-0465-02

Vyondys, single use vial; 50 mg/mL powder for injection

ICD-10 DiagnosesICD-10

G71.01

Duchenne or Becker muscular dystrophy

inv-08: Maximum dose per administration — dose and billable unit summary

Maximum dose (mg/kg)30 mg/kg once weekly

Billable units per dose350 billable units per administration

Total mg per dosing interval3500 mg every 7 days

Provider Requirements and Actions

Prior Authorization

Prior Authorization Required

Prior Authorization Required — requests for Vyondys must include documentation that the member meets the medical necessity criteria and evidence that the DMD gene mutation is amenable to exon 53 skipping. Without submitted clinical evidence and mutation documentation, EmblemHealth cannot properly review the request.

Submit clinical evidence supporting diagnosis and criteria (see Initial Approval Criteria).
Provide genetic testing/report confirming DMD mutation amenable to exon 53 skipping.

Documentation Required

Documentation Requirement for Review

Documentation supporting the request must be submitted with the prior authorization and at renewal. EmblemHealth requires baseline and follow-up clinical measures to evaluate response and ongoing medical necessity.

Baseline documentation of one or more: dystrophin level; timed function tests (6MWT, TTSTAND, TTRW, TTCLIMB); upper limb function (ULM) test; North Star Ambulatory Assessment (NSAA) score; Forced Vital Capacity (FVC) % predicted.

Background

Vyondys 53 (golodirsen) is an antisense oligonucleotide designed to bind exon 53 of dystrophin pre‑mRNA to induce exon 53 skipping and permit production of an internally truncated dystrophin protein for patients with DMD mutations amenable to exon 53 skipping. Duchenne muscular dystrophy is a progressive neuromuscular disorder primarily affecting children and young adults; monitoring of motor and respiratory function and documentation of baseline biomarker or functional measures are used to assess disease status and treatment response.

Definitions

inv-15: Vyondys (golodirsen) — design and mechanism key-value summary

Mechanism of actionAntisense oligonucleotide that binds exon 53 of dystrophin pre-mRNA to induce exon 53 skipping

Intended molecular effectExclusion of exon 53 during mRNA processing to allow production of an internally truncated dystrophin protein

Target populationPatients with DMD who have genetic mutations amenable to exon 53 skipping

Revision History

2025-04-02material updateLatest

Added limitations/exclusions: patient must not be on concomitant delandistrogene moxeparvovec-rokl and renal monitoring (serum cystatin C, urine dipstick, and UPCR) required prior to starting therapy and periodically during treatment.

2024-01-02material update

Updated length of authorization from 12 months to 6 months and added initial criteria requiring stable corticosteroid dose for at least 6 months, retention of meaningful voluntary motor function, receipt of physical/occupational therapy, and baseline documentation of at least one functional or biomarker measure.