CurrentEmblemHealthPolicy MG.MM.PH.349

Duchenne Muscular Dystrophy Drugs (Amondys 45, Exondys 51, Vyondys 53, Viltepso)

Defines medical necessity and prior authorization requirements for Amondys 45 (casimersen), Exondys 51 (eteplirsen), Vyondys 53 (golodirsen), and Viltepso (viltolarsen) for treatment of Duchenne muscular dystrophy for EmblemHealth members.

Policy Summary

PayerEmblemHealth

PolicyDuchenne Muscular Dystrophy Drugs (Amondys 45, Exondys 51, Vyondys 53, Viltepso)

Policy CodePolicy MG.MM.PH.349

Change TypeNo material changes

Effective DateFeb 28, 2022

Next Review DateMar 26, 2025

Key ActionSubmit prior authorization with clinical evidence including genetic testing confirming an exon 45, 51, or 53–amenable DMD mutation and baseline safety testing.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

12 mostandard approval length

weeklyadministration frequency

Exon 45/51/53eligible mutations

J1426–J1429procedure codes

Coverage Criteria for Exon-Skipping Therapies

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Initial Therapy

Covered when ALL of the following are met

Initial Approval Criteria: A. The patient must have a diagnosis of Duchenne muscular dystrophy (DMD);

B. Genetic testing documents the patient’s DMD gene mutation is amenable to exon 45, 51, or 53 skipping;

C. The patient is on a stable dose of corticosteroids prior to starting therapy OR there is a documented reason the patient is not on corticosteroids;

D. Baseline kidney function testing has been performed prior to starting therapy (except for eteplirsen, for which this is not required);

E. The patient is not concurrently being treated with another exon-skipping therapy for DMD.

Continuation Therapy / Renewal

Renewal is covered when ALL of the following are met

Renewal Criteria: A. Stabilization of disease or absence of disease progression;

B. Absence of unacceptable toxicity from the drug.

Concurrent treatment with another exon-skipping therapy for Duchenne muscular dystrophy is not permitted; patients must not be receiving more than one exon-skipping agent at the same time. This policy lists no other explicit general exclusions beyond that concurrent-therapy prohibition.

No additional not-medically-necessary statements are explicitly provided in the document.

Billing Codes, Dosing, and Diagnostic Codes

Applicable Procedure CodesHCPCS

J1429	Injection, golodirsen, 10 mg.
J1426	Injection, casimersen, 10 mg.
J1428	Injection, eteplirsen, 10 mg.
J1427	Injection, viltolarsen; 10 mg

Applicable NDCsNDC

60923-0465-02	Vyondys,single use vial; 50 mg/mL powder for injection.
60923-0227-02	Amondys Injection,100 mg/2 mL (50 mg/mL) solution in a single-dose vial.
73292-0011-01
60923-0363-02	Exondys 51 100 mg/2 mL Solution J1428 Injection Eteplirsen 10 mg.
60923-0284-10	Exondys 51 50 mg/10 mL Solution J1428 Injection Eteplirsen 10 mg.

ICD-10 DiagnosesICD-10

G71.01

Duchenne or Becker muscular dystrophy

Per-drug dosing limits

Amondys 45 (casimersen)30 mg/kg once weekly

Exondys 51 (eteplirsen)30 mg/kg once weekly

Vyondys 53 (golodirsen)30 mg/kg once weekly

Viltepso (viltolarsen)80 mg/kg once weekly

Prior Authorization, Documentation, and Provider Requirements

Prior Authorization

Prior Authorization Required

Prior authorization required. Approval for therapy will be granted for a 12-month period. Requests must be reviewed and approved by EmblemHealth prior to initiation of therapy.

Length of authorization: 12 months
Prior authorization required before treatment initiation

Denial Risk

Concurrent Therapy Prohibited

Concurrent therapy prohibition. The patient must not be concurrently treated with another exon-skipping therapy for Duchenne muscular dystrophy (DMD). Concurrent use of two exon-skipping therapies is not permitted and will result in denial of the request.

No concurrent treatment with another exon-skipping therapy for DMD

Documentation Required

Documentation Required for Review

Required clinical documentation. The treating provider must submit clinical evidence to support medical necessity. Failure to provide complete documentation may prevent review or result in denial.

Diagnosis documentation confirming Duchenne muscular dystrophy (DMD)
Genetic testing results confirming the DMD gene mutation is amenable to exon 45, 51, or 53 skipping
Evidence of stable corticosteroid dosing prior to therapy start, or documentation of medical reason for not being on corticosteroids
Baseline kidney function testing prior to starting therapy (except for eteplirsen)
Any additional clinical notes, prior treatments, and lab results relevant to the request

Documentation Required

Submit Complete Documentation

Documentation requirement. The treating physician or primary care provider must submit all required clinical evidence and information with the prior authorization request. Without complete documentation, EmblemHealth will not be able to properly review the request and the request may be denied.

Submit all supporting clinical records and test results with the prior authorization request
Incomplete documentation may prevent review or result in denial

Background on Duchenne Muscular Dystrophy and Therapies

Duchenne muscular dystrophy (DMD) is an X-linked inherited disorder caused by deficiency of dystrophin, leading to progressive muscle weakness with onset in childhood. Exon-skipping therapies (antisense oligonucleotides such as casimersen, eteplirsen, golodirsen, and viltolarsen) target specific exons in dystrophin pre-mRNA to enable skipping of an affected exon and production of a shortened but partially functional dystrophin protein. These agents are indicated for patients with DMD whose genetic testing demonstrates mutations amenable to skipping of exon 45, 51, or 53.

Key Definitions

Duchenne Muscular Dystrophy (DMD) — definition and key features

DefinitionDuchenne Muscular Dystrophy (DMD) is an inherited disorder resulting from dystrophin deficiency causing progressive muscle weakness and loss of muscle function; it primarily affects males and is the most common and severe form of muscular dystrophy in children.

Typical age of onsetSymptom onset usually occurs between ages 3 and 5.

Genetic basisMutations in the DMD gene impair production of dystrophin, a protein that protects skeletal and cardiac muscle from deterioration.

Clinical consequenceLeads to progressive skeletal and heart muscle deterioration and decline in function.

Exon-skipping therapies — definition and mechanism

Class of therapyAntisense oligonucleotides (exon-skipping therapies)

MechanismThey bind specific exons of dystrophin pre-mRNA to enable exon skipping, producing a shortened but partially functional dystrophin protein.

Drugs coveredAmondys 45 (casimersen), Exondys 51 (eteplirsen), Vyondys 53 (golodirsen), Viltepso (viltolarsen)