CurrentCignaPolicy IP0135

Muscular Dystrophy - Exondys 51 (eteplirsen)

Defines medical necessity, dosing, and coverage limits for Exondys 51 (eteplirsen) for patients with Duchenne muscular dystrophy who have a DMD gene mutation amenable to exon 51 skipping. Applies to Cigna-administered health benefit plans and providers requesting coverage.

Policy Summary

PayerCigna

PolicyMuscular Dystrophy - Exondys 51 (eteplirsen)

Policy CodePolicy IP0135

Change TypeTitle updateddosing added

Effective Date

Next Review Date

Key ActionObtain prior authorization and document genetic testing confirming a DMD variant amenable to exon 51 skipping; initial approvals are for 6 months.

SourceLink

POLICY UPDATE CHANGES

Updated policy title; previously it was Eteplirsen.

Added dosing to the policy.

30 mg/kg weeklyapproved dosing

6 monthsauthorization period

<14 yrsage limit for initiation

≥180 m6MWD requirement

≈13%proportion amenable

Coverage Criteria for Exondys 51 (eteplirsen)

FDA-Approved Indication: Initial and Continuation Therapy

Exondys 51 is covered when the following FDA‑approved indication criteria are met.

FDA-Approved Indication: Duchenne Muscular Dystrophy. Approve for the duration noted if the patient meets ONE of the following (A or B)

A. Initial Therapy (approve 6 months): All of the following i-v must be met

Initial i: Less than 14 years of age at start of therapy<14 years

Initial ii:

Policy Summary

PayerCigna

PolicyMuscular Dystrophy - Exondys 51 (eteplirsen)

Policy CodePolicy IP0135

Change TypeTitle updateddosing added

Effective Date

Next Review Date

Key ActionObtain prior authorization and document genetic testing confirming a DMD variant amenable to exon 51 skipping; initial approvals are for 6 months.

SourceLink

On This Page

Trek Health ingests and normalizes Transparency in Coverage data and payer policy updates to give provider organizations a clear view of how commercial reimbursement behaves across markets, payers, and services. Our platform transforms raw payer disclosures into structured intelligence that supports contract evaluation, payer negotiations, and service line strategy. By combining market benchmarks with ongoing policy visibility, Trek helps teams identify variability, risk, and opportunity in commercial reimbursement. The result is faster insight, stronger negotiating positions, and more informed financial decisions.

Coding and Measurement Requirements

Considered Medically Necessary when criteria met: HCPCSHCPCSCovered

J1428

Injection, eteplirsen, 10 mg

6-Minute Walk Test requirement — initiation threshold

6-Minute Walk Test (6MWT) thresholdAble to walk a distance of at least 180 meters independently over 6 minutes (6MWD ≥180 m)

Context within initial therapy criteriaRequired for Initial Therapy approval (must meet all initial criteria to approve for 6 months)

Age linkageApplied in patients <14 years at therapy start per initial therapy criteria

Use for continuationContinuation approvals require prior criteria met and demonstration of clinical benefit, including continued ability to walk

Provider Requirements and Authorization

Prior Authorization

Prior Authorization Required

Prior authorization is required. Initial approvals: 6 months. Continuation/renewal approvals: 6 months when continuation criteria are met (see Required Clinical Documentation and Prescriber Requirements).

Initial approval duration: 6 months
Continuation/renewal approval duration: 6 months

Note

Prescriber Requirements

Prescriber must be a neurologist, neuromuscular specialist, or prescriber in consultation with one of these specialists, or a Muscular Dystrophy Association clinic. Medication must be prescribed by, or in consultation with, one of the specified specialists for both initial and continuation approvals.

Prescribing/consultation requirement: neurologist, neuromuscular specialist, or Muscular Dystrophy Association clinic
Specialty requirement applies to initial and continuation therapy

Documentation Required

Required Clinical Documentation

Documentation must include genetic testing confirming a pathogenic or likely pathogenic DMD gene variant that is amenable to exon 51 skipping, documented diagnosis of Duchenne muscular dystrophy, and baseline ambulatory function (6-minute walk distance ≥ 180 meters) for initial requests. For continuation requests, documentation must show the above criteria were met prior to initiation and evidence of clinical benefit (including continued ability to walk).

Genetic testing report documenting a pathogenic or likely pathogenic DMD variant amenable to exon 51 skipping
Documented diagnosis of Duchenne muscular dystrophy
Baseline 6-minute walk distance ≥ 180 meters for initial therapy
For continuation: evidence that initial criteria were met prior to initiation and documentation of beneficial clinical response (including continued ambulation)

Key Definitions

Amenable to exon 51 skipping — definition

DefinitionPatients with DMD gene mutations predicted to be responsive to exclusion (skipping) of exon 51 during mRNA processing; Exondys 51 binds exon 51 of dystrophin pre-mRNA to produce exon 51 skipping.

Proportion of DMD patientsApproximately 13% of patients with Duchenne muscular dystrophy have mutations amenable to exon 51 skipping.

VerificationDiagnosis must be confirmed by a pathogenic or likely pathogenic DMD variant that is amenable to exon 51 skipping (genetic testing required).

Regulatory noteIndication was approved under accelerated approval based on increased dystrophin; clinical benefit has not been established and continued approval may depend on confirmatory trials.

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 induce skipping of exon 51 during mRNA processing, restoring the dystrophin reading frame in patients with DMD gene mutations that are amenable to exon 51 skipping. FDA approval was granted via the accelerated pathway based on increased dystrophin expression; however, definitive clinical benefit has not been firmly established, and continued approval may depend on confirmatory trials.