Exon-skipping therapies for Duchenne muscular dystrophy (eteplirsen, golodirsen, casimersen, viltolarsen)
Customize your policy alerts
Sign up for all Healthfirst policy alerts
Know when Healthfirst releases new policies or updates existing guidance.
Monitor payer policy activity
Defines prior authorization, medical necessity, dosing, monitoring, billing, and coverage criteria for exon-skipping antisense oligonucleotide therapies for patients with Duchenne muscular dystrophy (DMD) within Healthfirst plans.
No material clinical or coverage changes in this revision.
Coverage and Medical Necessity Criteria
Initial therapy - Medical necessity criteria
EXONDYS 51, VYONDYS 53, AMONDYS 45, and VILTEPSO are considered medically necessary when ALL of the following are met:
Provide genetic test report documenting the exon deletion pattern (see Appendices B-D for examples).
Include medication history and dates/doses to demonstrate stability or rationale for not using corticosteroids.
Provide recent laboratory results (e.g., serum creatinine, eGFR) as part of prior authorization record; not required for eteplirsen.
Concurrent use of multiple exon-skipping antisense oligonucleotides is not permitted.
Coverage excludes use that exceeds the FDA-approved dosing specified in the dosing section of this policy. The policy also excludes initiation of other pharmacologic or experimental treatments that affect muscle strength or function within 12 weeks of the first week of exon-skipping therapy (with the exception of corticosteroids). Additionally, members with clinically significant comorbidities, and patients outside the trial populations (safety/efficacy unknown in females and geriatric patients) are subject to these limitations.
Any violation of this policy will be handled in accordance with the plan’s Disciplinary Action policy.
Eteplirsen, golodirsen, casimersen, and viltolarsen are considered investigational and not medically necessary when the specified medical necessity criteria are not met, or for any other indications beyond those defined by the policy. Coverage is contingent on meeting all required criteria such as diagnosis of DMD, genetic confirmation of an exon 45, 51, or 53 mutation amenable to skipping, documented corticosteroid status, and required kidney testing (except for eteplirsen).
Billing, Codes, and Units
| 0260 | IV therapy, general |
| 0636 | Drugs requiring detailed coding |
| 0510 | Clinic |
| G71.01 | Duchenne or Becker muscular dystrophy |
| 60923-0363-02 | Exondys 51 100mg/2ml |
| 60923-0284-10 | Exondys 51 500mg/10ml |
| 60923-0465-02 | Vyondys 53 single use vial |
| 60923-0227-02 | Amondys 45 100 mg/2 mL |
| 73292-0011-01 | Viltepso 250 mg/5 mL |
| J-code billing units | 1 billing unit = 10 mg of drug; examples: Exondys 51 2 ml vial (100 mg) = 10 units; Exondys 51 10 ml vial (500 mg) = 50 units; Vyondys 53 2 ml vial (100 mg) = 10 units; Amondys 45 2 ml vial (100 mg) = 10 units; Viltepso 5 ml vial (250 mg) = 25 units |
Authorization, Documentation, and Billing Requirements
Prior Authorization and Peer Review Required
Prior authorization required; subject to Healthfirst clinical criteria and peer review.
- Peer review required due to expense of medication
- Requests are reviewed against Healthfirst clinical criteria and applicable NCD/LCD
J-code Billing Unit Reporting
Provider must state number of J-code billing units on claim forms. For DMD drugs, 1 billing unit = 10 mg; incorrect or missing billing unit reporting may trigger claim issues or denials.
- 1 billing unit = 10 mg of drug
- Examples: Exondys 51 100 mg vial = 10 units; 500 mg vial = 50 units; Vyondys 53 100 mg vial = 10 units; Amondys 45 100 mg vial = 10 units; Viltepso 250 mg vial = 25 units
Concurrent Therapy Restriction
Patient must not be concurrently treated with another exon skipping therapy for DMD. Concurrent use with other exon skipping agents is not allowed.
- Concurrent treatment with another exon skipping therapy for DMD is a contraindication to coverage
Required Documentation for Prior Authorization
Documentation to support prior authorization must include diagnostic and testing evidence as specified below; requests that fail to meet criteria may be denied.
- Diagnosis of Duchenne muscular dystrophy (DMD)
- Genetic testing confirming a DMD gene mutation amenable to exon 45, 51, or 53 skipping (see Appendices B–D)
- Documentation of stable corticosteroid dose prior to initiation or a documented reason for not receiving corticosteroids
- Baseline kidney function testing prior to initiation (except for eteplirsen)
NSAA Scoring Documentation
Include results from the North Star Ambulatory Assessment (NSAA) when submitting requests; document the activity item scores from Appendix A in the medical record and prior authorization submission.
- Attach NSAA scores (Appendix A) or include them in the clinical documentation
- Document individual activity item scores (e.g., Stand, Walk, Rise from chair, Climb, Jump, Run, Hop, etc.) and total score as applicable
Denial for Unmet Criteria
Requests failing to meet the medical necessity criteria may be denied. Ensure all required documentation and eligibility criteria are addressed on submission to avoid denials.
- Denial may occur if genetic testing does not show an amenable mutation, required labs or NSAA scores are missing, or if concurrent exon skipping therapy is present
- Duration of approval when approved: up to 6 months
Step Therapy
No step therapy requirements specified for these agents.
- No step edits or prior required trials specified in policy
Background and Clinical Context
Duchenne muscular dystrophy (DMD) is an X-linked recessive genetic disorder caused by deficiency of dystrophin, leading to progressive muscle fiber degeneration and weakness. Exon-skipping antisense oligonucleotide therapies (eteplirsen, golodirsen, casimersen, viltolarsen) are designed to exclude specific exons during mRNA processing to restore the dystrophin reading frame and produce a truncated but partially functional dystrophin protein. Prevalence of amenable mutations varies by exon (examples and mutation patterns are provided in the appendices).
Key Definitions and Measures
OpenPayer is powered by Trek Health's payer performance platform. Trek continuously ingests, validates, and normalizes Transparency in Coverage data alongside payer policies and other commercial payer data to create a structured payer intelligence foundation. OpenPayer uses this foundation to deliver personalized search results, dynamically generated policy pages, and tailored policy monitoring based on each user's payers, specialties, billing codes, and areas of interest. The same intelligence powers broader payer performance workflows, including reimbursement benchmarking, contract evaluation, payer negotiations, and financial decision-making.