CurrentCignaPolicy IP0135

Muscular Dystrophy - Exondys 51 (eteplirsen)

Defines Cigna coverage criteria for Exondys 51 (eteplirsen) intravenous infusion for patients with Duchenne muscular dystrophy who have a DMD gene mutation amenable to exon 51 skipping; applies to benefit plans administered by Cigna Companies where this Coverage Policy governs utilization review and coverage determinations.

Policy Summary

PayerCigna

PolicyMuscular Dystrophy - Exondys 51 (eteplirsen)

Policy CodePolicy IP0135

Change TypeTitle reviseddosing added

Effective DateAug 15, 2024

Next Review DateMar 26, 2026

Key ActionObtain prior authorization and submit J1428 with documentation of genetic confirmation, 6-minute walk test (≥180 m), and specialist prescription.

SourceLink

POLICY UPDATE CHANGES

Updated policy title; previously it was Eteplirsen. Added dosing to the policy.

Effective date updated to 8/15/2024 and review date noted as 6/6/2024 for the update.

6 monthsApproval duration

30 mg/kg weeklyDosing (IV)

≥180 m6MWT minimum

<14 yrsMax age at start

~13%Amenable population

Coverage Criteria for Exondys 51 (eteplirsen)

Initial Therapy

Approve for 6 months if the patient meets ALL of the following (A.i–v):

Initial Therapy (A)

i: Less than 14 years of age at start of therapyage <14 years
ii: Documented diagnosis of Duchenne muscular dystrophy confirmed by a pathogenic or likely pathogenic variant in the DMD gene that is amenable to exon 51 skippinggenetic confirmation
Genetic testing required
iii:

Policy Summary

PayerCigna

PolicyMuscular Dystrophy - Exondys 51 (eteplirsen)

Policy CodePolicy IP0135

Change TypeTitle reviseddosing added

Effective DateAug 15, 2024

Next Review DateMar 26, 2026

Key ActionObtain prior authorization and submit J1428 with documentation of genetic confirmation, 6-minute walk test (≥180 m), and specialist prescription.

SourceLink

On This Page

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Continuation Therapy / Currently Receiving Exondys 51

Approve for 6 months if the patient currently receiving Exondys 51 meets ALL of the following (B.i–iii):

Continuation Therapy (B)

i: The above criteria were met prior to initiation of Exondys 51prior eligibility met
Documentation of baseline criteria required
ii: Patient has experienced a beneficial clinical response, including the continued ability to walkclinical benefit demonstrated
Continued ambulation required as example of benefit
iii: Medication continues to be prescribed by, or in consultation with, a neurologist, neuromuscular specialist, or by a Muscular Dystrophy Association clinicspecialist prescriber

Coding and Clinical Thresholds

Covered HCPCS CodesHCPCSCovered

J1428

Injection, eteplirsen, 10 mg

inv-07: 6-Minute Walk Test — minimum threshold for initiation

6-minute walk test thresholdAble to walk a distance of at least 180 meters independently over 6 minutes (>= 180 meters)

Context for useRequired for initial approval (part of Initial Therapy criteria A.iii)

Documentation required6-minute walk test distance must be provided in prior authorization documentation

inv-08: Age at therapy initiation — maximum age threshold for initial approval

Maximum age for initiationLess than 14 years of age at start of therapy (age < 14 years)

ApplicabilityRequired for initial approval (part of Initial Therapy criteria A.i)

Approval duration when criteria metInitial approval duration: 6 months

Provider Actions, Prior Authorization, and Billing

Prior Authorization

Prior Authorization Required

Prior authorization required. Exondys 51 (eteplirsen) is billed with HCPCS code J1428 and will be covered only when the policy medical necessity criteria are met.

Prior authorization required
Bill Exondys 51 using HCPCS J1428 (Injection, eteplirsen, 10 mg)

Documentation Required

Required Clinical Documentation

Genetic testing confirming a pathogenic or likely pathogenic DMD variant that is amenable to exon 51 skipping must be provided. For initial therapy, documentation must also include age at therapy start (<14 years), a 6-minute walk test demonstrating at least 180 meters, and that the prescribing clinician is a neurologist, neuromuscular specialist, or a Muscular Dystrophy Association clinic. For patients currently receiving Exondys 51, documentation must show that the above criteria were met prior to initiation and that the patient has experienced a beneficial clinical response (including continued ambulation). Receipt of sample product does not satisfy documentation requirements.

Genetic test confirming pathogenic or likely pathogenic DMD variant amenable to exon 51 skipping
Initial therapy documentation: age at start (<14 years), 6-minute walk test ≥180 meters, prescribing clinician specialty
Continuation documentation: prior criteria met before initiation and evidence of beneficial clinical response (continued ability to walk)
Receipt of sample product does not meet documentation requirements

Denial Risk

Concurrent Exon-skipping Agents Not Covered

Concurrent use of Exondys 51 with other exon-skipping agents (for example, Amondys 45, Viltepso, Vyondys 53) is not covered. There is no clinical evidence to support concurrent exon-skipping therapy; claims for concurrent administration will be denied as experimental/investigational.

Concurrent use with other exon-skipping DMD agents is not covered (e.g., Amondys 45, Viltepso, Vyondys 53)
Claims for concurrent exon-skipping agents will be denied

Billing Rule

Coding and Billing Compliance

Claims submitted without an appropriate covered diagnosis or without the required supporting clinical documentation will be denied or adjusted. Use the most appropriate and current codes as of the date of service. Ensure J1428 is accompanied by documentation demonstrating that the medical necessity criteria are met.

Claims without covered diagnosis/procedure codes or lacking required documentation will be denied/adjusted
Use current, appropriate codes as of the date of service
Ensure J1428 is accompanied by medical necessity documentation

Background

Duchenne muscular dystrophy (DMD) is an X‑linked recessive disorder caused by mutations in the DMD gene that lead to loss of dystrophin and progressive muscle degeneration. Exondys 51 (eteplirsen) is an antisense oligonucleotide designed to skip exon 51 during pre‑mRNA processing to restore the dystrophin reading frame in patients with mutations amenable to exon 51 skipping; however, clinical benefit has not been definitively established and continued approval has been linked to confirmatory studies. This background supports the policy's focus on genetic confirmation of an exon 51‑amenable DMD variant when considering therapy.

OpenPayer

Muscular Dystrophy - Exondys 51 (eteplirsen)

Coverage Criteria for Exondys 51 (eteplirsen)

Coding and Clinical Thresholds

Provider Actions, Prior Authorization, and Billing

Background

Key Definitions