ModifiedUnitedHealthcarePolicy IEXD0058.09

Exondys 51 (eteplirsen) — Medical Benefit Drug Policy (Individual Exchange)

Medical benefit drug policy governing coverage criteria for Exondys 51 (eteplirsen) in patients with Duchenne muscular dystrophy amenable to exon 51 skipping for UnitedHealthcare Individual Exchange plans (with some state exceptions).

Policy Summary

PayerUnitedHealthcare

PolicyExondys 51 (eteplirsen) — Medical Benefit Drug Policy (Individual Exchange)

Policy CodePolicy IEXD0058.09

Change TypeRevised coverage criteria (prior gene therapy branching)

Effective DateJuly 1, 2025

Next Review DateN/A

Key ActionSubmit prior authorization with medical records confirming DMD mutation amenable to exon 51 skipping and documentation of prior gene therapy status or clinically meaningful decline if previously treated.

SourceLink

POLICY UPDATE CHANGES

Revised coverage criteria for initial therapy; added requirement that the patient either has not previously received gene therapy or, if previously received gene therapy, there must be documentation of clinically meaningful functional decline since gene replacement therapy.

07/01/2025Policy revision date

6-12 moAuth period

J1428HCPCS

≈13%Proportion amenable

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Coverage Criteria for Exondys 51 (eteplirsen)

Initial Therapy

Covered when ALL of the following are met

Initial Therapy - Core requirements: Diagnosis of Duchenne muscular dystrophy by, or in consultation with, a neurologist with expertise in the diagnosis of DMD; and submission of medical records (e.g., chart notes, laboratory values) confirming the mutation of the DMD gene is amenable to exon 51 skipping; prescribed by, or in consultation with, a neurologist with expertise in the treatment of DMD; dosing in accordance with FDA-approved labeling; initial authorization for no more than 12 months; Exondys 51 is not used concomitantly with other exon skipping therapies for DMD (e.g., Amondys, Viltepso, Vyondys 53).

Initial Therapy - Ambulatory/function requirement: Either: (a) submission of medical records confirming the patient has a 6-Minute Walk Test (6MWT) ≥ 300 meters while walking independently prior to beginning Exondys 51 therapy; OR (b) both: submission of medical records confirming the patient is ambulatory without needing an assistive device AND one of the following: NSAA score > 17 OR time to rise (TTR) < 7 seconds.6MWT ≥ 300 m OR NSAA >17 OR TTR <7s

Documentation via medical records (chart notes, laboratory values) required.

Initial Therapy - Prior gene therapy: Either: (a) patient has not previously received gene therapy (e.g., Elevidys [delandistrogene moxparvovec-rokl]) for the treatment of DMD; OR (b) both: patient has previously received gene therapy for DMD AND submission of medical records documenting a clinically meaningful functional decline resulting from loss of muscle strength/motor ability (e.g., loss of a motor milestone) since receiving gene replacement therapy.

Applied to initial therapy approvals; medical records documenting decline required if prior gene therapy received.

Continuation Therapy

Covered for continuation when ALL of the following are met

Continuation Therapy - Requirements: Submission of medical records (e.g., chart notes) confirming the patient is ambulatory without needing an assistive device; Exondys 51 is not used concomitantly with other exon skipping therapies for DMD (e.g., Amondys, Viltepso, Vyondys 53); prescribed by, or in consultation with, a neurologist with expertise in the treatment of DMD; dosing in accordance with FDA-approved labeling; reauthorization will be for no more than 12 months.

Unproven / Not Medically Necessary

Unproven uses: Eteplirsen (Exondys 51) is unproven and not medically necessary for the treatment of other forms of muscular dystrophy (e.g., Becker muscular dystrophy) or for DMD that is not amenable to exon 51 skipping. FDA approval alone is not a basis for coverage; continued approval is contingent on verification of clinical benefit in confirmatory trials.

Initial Therapy — Prior Gene Therapy Condition

Revised coverage criteria for initial therapy include the following branching requirement:

Prior gene therapy branch: Either the patient has not previously received gene therapy (e.g., Elevidys [delandistrogene moxparvovec-rokl]) for the treatment of DMD; OR both: the patient has previously received gene therapy for DMD AND submission of medical records (e.g., chart notes, laboratory values) documenting a clinically meaningful functional decline resulting from loss of muscle strength/motor ability (e.g., loss of a motor milestone) since receiving gene replacement therapy.

Applied to initial therapy approvals; supporting documentation required if prior gene therapy received.

Use of Exondys 51 (eteplirsen) concomitantly with other exon skipping therapies for Duchenne muscular dystrophy (for example, Amondys, Viltepso, Vyondys 53) is prohibited. Prior authorization and ongoing coverage require documentation that Exondys 51 is not being administered at the same time as any other exon skipping agent.

FDA approval or labeling alone is not a basis for coverage. Coverage determinations for Exondys 51 are governed by the member-specific benefit plan document and any applicable federal or state mandates; when plan terms differ, the member-specific benefit plan controls. Providers should verify member benefits and submit required supporting records with any prior authorization request.

Exondys 51 is not medically necessary for the treatment of other forms of muscular dystrophy, including Becker muscular dystrophy, or for Duchenne muscular dystrophy that is not amenable to exon 51 skipping.

Coding — Procedure and Diagnosis Codes

Applicable HCPCS CodesHCPCS

J1428

Injection, eteplirsen, 10 mg

Diagnosis CodesICD-10

G71.01

Duchenne or Becker muscular dystrophy

6-Minute Walk Test — functional threshold used in criteria

6-Minute Walk Test threshold6MWT ≥ 300 meters while walking independently (no side-by-side assist, cane, walker, wheelchair, etc.) prior to initiation of Exondys 51 therapy.

Alternate ambulatory/function criteriaAmbulatory without assistive device AND (NSAA score > 17 OR time to rise < 7 seconds).

Documentation requiredSubmission of medical records (chart notes, laboratory values) confirming the 6MWT result and ambulatory status prior to therapy.

Prior Authorization, Documentation, and Provider Requirements

Prior Authorization

Prior Authorization Required for Initial Eteplirsen Therapy

Prior authorization is required for initial Exondys 51 (eteplirsen) therapy. Requests must document a diagnosis of Duchenne muscular dystrophy (DMD) and that the DMD gene mutation is amenable to exon 51 skipping. Initial authorization will be for no more than 12 months.

Submit medical records (e.g., chart notes, laboratory values) confirming diagnosis and mutation amenable to exon 51 skipping.
Initial therapy criteria must be met (see clinical criteria): ambulatory status and functional measures such as 6MWT ≥ 300 meters OR ambulatory without assistive device plus NSAA >17 or TTR <7 seconds.
If patient previously received gene therapy (e.g., Elevidys), submit records documenting a clinically meaningful functional decline since gene therapy.

Documentation Required

Member-Specific Benefit Verification

Verify the member-specific benefit plan and any applicable federal or state mandates prior to submission. UnitedHealthcare policy is informational; the member-specific benefit document governs in case of conflict.

Background on Duchenne Muscular Dystrophy and Eteplirsen

Duchenne muscular dystrophy (DMD) is an X-linked disorder caused by mutations in the dystrophin gene that lead to progressive muscle degeneration, loss of ambulation during childhood, and cardiopulmonary complications. The population prevalence is approximately 1 in 3,500 to 5,000 live male births. Eteplirsen is an antisense oligonucleotide designed to induce exon 51 skipping, producing a truncated dystrophin protein and targeting the subset of patients with DMD whose mutations are amenable to exon 51 skipping (approximately 13% of DMD patients).

Definitions and Key Terms

Exon 51 skipping — mechanism and brief definition

MechanismAntisense oligonucleotide approach that binds dystrophin pre-mRNA to exclude exon 51 during mRNA processing, enabling production of an internally truncated dystrophin protein.

Target population fractionApproximately 13% of DMD patients have deletions amenable to exon 51 skipping.

Intended effectRestore reading frame to allow truncated dystrophin production rather than full-length dystrophin.

Exondys 51 indication — approved indication and regulatory status

Approved indicationTreatment of Duchenne muscular dystrophy in patients with a confirmed DMD gene mutation amenable to exon 51 skipping.

Regulatory status

Policy Revision History

07/01/2025coverage_criteria_revisionLatest

Revised coverage criteria for initial therapy to add an explicit branching requirement regarding prior gene therapy: either the patient has not previously received gene therapy (e.g., Elevidys) or, if previously received gene therapy, submission of medical records must document a clinically meaningful functional decline since gene replacement therapy.