ModifiedUnitedHealthcarePolicy CSNJ2O25D00580

Exondys 51 (Eteplirsen) — Medical Benefit Drug Policy (NJ)

Policy governing medical benefit drug coverage of Exondys 51 (eteplirsen) for treatment of Duchenne muscular dystrophy in New Jersey members, including initial and continuation authorization criteria and applicable billing codes.

Policy Summary

PayerUnitedHealthcare

PolicyExondys 51 (Eteplirsen) — Medical Benefit Drug Policy (NJ)

Policy CodePolicy CSNJ2O25D00580

Change TypeRevised coverage criteria — added prior gene therapy documentation requirement

Effective DateJul 1, 2025

Next Review Date

Key ActionSubmit prior authorization with medical records documenting exon 51–amenable DMD mutation and, if applicable, clinically meaningful functional decline since prior gene therapy.

SourceLink

POLICY UPDATE CHANGES

Revised coverage criteria for initial therapy; added requirement addressing prior gene therapy (e.g., Elevidys) and documentation of clinically meaningful functional decline after gene replacement therapy.

Revised coverage criteria for initial therapy; added criterion requiring documentation that the patient has previously received gene therapy (e.g., Elevidys) and has had a clinically meaningful functional decline since that therapy.

12 momaximum initial authorization duration

12 momaximum continuation authorization duration

J1428listed drug billing code

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G71.01diagnosis code

1added prior-gene-therapy requirement

Coverage Criteria

Initial Therapy

Covered when ALL of the following are met for initial therapy (unless the alternate prior-gene-therapy branch applies):

Initial Therapy Core Criteria: Submission of medical records confirming the DMD mutation is amenable to exon 51 skipping; diagnosis of Duchenne muscular dystrophy by, or in consultation with, a neurologist with expertise in DMD; submission of medical records providing baseline status prior to starting therapy (e.g., ambulatory without assistive device); patient has not previously received gene therapy (e.g., Elevidys); dosing in accordance with FDA-approved labeling; prescribed by, or in consultation with, a neurologist experienced in DMD; initial authorization for no more than 12 months.

These items are conjunctive; all must be present for coverage.

Initial Therapy After Gene Replacement Therapy

Alternate initial-therapy pathway when patient has previously received gene replacement therapy:

Post–Gene Therapy Initial Criteria: Patient has previously received gene replacement therapy (e.g., Elevidys [delandistrogene moxparvovec-rokl]); submission of medical records documenting a clinically meaningful functional decline since receiving gene replacement therapy (loss of muscle strength/motor ability or loss of a motor milestone); Exondys 51 is not used concomitantly with other exon skipping therapies; dosing in accordance with FDA-approved labeling; prescribed by, or in consultation with, a neurologist experienced in DMD; initial authorization up to 12 months.

All conditions required for coverage in this scenario.

Continuation Therapy

Covered when ALL of the following are met for continuation (reauthorization):

Continuation Therapy: Exondys 51 is not used concomitantly with other exon skipping therapies for DMD; submission of medical records confirming the patient is receiving clinical benefit (e.g., walking without needing an assistive device); prescribed by, or in consultation with, a neurologist experienced in DMD; dosing in accordance with FDA-approved labeling; reauthorization for no more than 12 months.

All listed conditions required for reauthorization.

Unproven / Not Medically Necessary Uses

Unproven Indications: Use of Exondys 51 for other forms of muscular dystrophy (e.g., Becker muscular dystrophy) is considered unproven and not medically necessary.

Explicit not medically necessary stance for non-exon-51 DMD forms and other muscular dystrophies.

Initial Therapy (added requirement)

Revised initial therapy criterion (excerpt):

Initial therapy additional requirement: Patient has previously received gene therapy (e.g., Elevidys [delandistrogene moxparvovec-rokl]) for the treatment of DMD.

Must be documented in medical records; see supporting documentation node

Documentation of decline: 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.

These records are required to support approval for initial eteplirsen therapy after prior gene therapy

Use of Exondys 51 (eteplirsen) for the treatment of other forms of muscular dystrophy, such as Becker muscular dystrophy, is considered unproven and not medically necessary.

No specific exclusions or additional not-medically-necessary items are listed in the cited policy text for this section; reference the overall policy Instructions for Use and applicable federal, state, or contractual plan requirements when determining coverage.

Exondys 51 (eteplirsen) is unproven and not medically necessary for indications other than Duchenne muscular dystrophy caused by mutations amenable to exon 51 skipping.

No additional not-medically-necessary uses are explicitly listed in the provided document excerpts for this section. When applying policy, verify applicable federal, state, or contractual requirements as they may supersede the standard policy language.

Provider Actions and Documentation Requirements

Prior Authorization

Prior Authorization and Duration

Prior authorization is required. Submit medical records demonstrating the DMD gene mutation is amenable to exon 51 skipping, documentation of baseline functional status prior to starting therapy, and that the request is made by or in consultation with a neurologist with expertise in DMD. Initial authorizations will be for no more than 12 months.

Initial authorization duration: up to 12 months
Prescriber requirement: neurologist with DMD expertise

Documentation Required

Prior Therapy and Decline Documentation

For patients who previously received gene replacement therapy (e.g., Elevidys [delandistrogene moxparvovec-rokl]), prior authorization must include medical records documenting a clinically meaningful functional decline (for example, loss of muscle strength, loss of a motor milestone) that occurred after the gene therapy.

Examples of supporting records: chart notes, clinic visit summaries, relevant lab or functional assessment results

Coding

HCPCS CodesHCPCSCovered

J1428

Injection; eteplirsen, 10 mg

Diagnosis CodesICD-10

G71.01

Duchenne or Becker muscular dystrophy

Definitions

Exon 51–amenable mutation

DefinitionA DMD gene deletion or other mutation that can be addressed by skipping exon 51 to restore the reading frame and allow production of a truncated (internally shortened) dystrophin protein.

Population affectedApproximately 13% of DMD patients have out-of-frame deletion mutations amenable to exon 51 skipping.

MechanismExon 51 skipping uses antisense oligonucleotides to bind dystrophin pre-mRNA and exclude exon 51 during mRNA processing, enabling production of internally truncated dystrophin.

Clinical benefit / clinically meaningful functional decline

DefinitionProvider-documented functional measures indicating clinical benefit or meaningful functional decline, such as ambulatory status (e.g., walking without an assistive device).

Examples of meaningful decline

Background

Duchenne muscular dystrophy (DMD) is an X-linked disorder caused by mutations in the dystrophin gene that lead to progressive muscle degeneration and loss of ambulation during childhood. Eteplirsen (Exondys 51) is an antisense oligonucleotide designed to induce skipping of exon 51 and produce a truncated, partially functional dystrophin protein; approximately 13% of patients with DMD have mutations amenable to exon 51 skipping.

Revision History

2025-07-01policy_revisionLatest

Revised coverage criteria for initial therapy to add a pathway for patients previously treated with gene replacement therapy (e.g., Elevidys) and require submission of medical records documenting a clinically meaningful functional decline since gene therapy; initial authorizations limited to 12 months.

Policy Summary

PayerUnitedHealthcare

PolicyExondys 51 (Eteplirsen) — Medical Benefit Drug Policy (NJ)

Policy CodePolicy CSNJ2O25D00580

Change TypeRevised coverage criteria — added prior gene therapy documentation requirement

Effective DateJul 1, 2025

Next Review Date

Key ActionSubmit prior authorization with medical records documenting exon 51–amenable DMD mutation and, if applicable, clinically meaningful functional decline since prior gene therapy.

SourceLink

On This Page

Denial Risk

Documentation Requirement for Prior Gene Therapy

Failure to submit medical records documenting a clinically meaningful decline after prior gene replacement therapy (e.g., Elevidys) may result in denial or an incomplete authorization decision.

Required: documentation of decline since receiving gene therapy

Billing Rule

Concomitant Therapy Restriction

Exondys 51 must not be used concomitantly with other exon skipping therapies for DMD (for example, Amondys, Viltepso, Vyondys 53).

Examples of excluded concomitant therapies: Amondys (casimersen), Viltepso (viltolarsen), Vyondys 53 (golodirsen)

Documentation Required

Required Clinical Documentation and Deficiency Risk

Submit complete medical records to support all requested elements: confirmation of an exon 51–amenable DMD mutation, baseline functional status prior to therapy, documentation of prior gene therapy if applicable, and evidence of clinically meaningful decline when required. Failure to provide these records may result in denial or delayed processing.

Acceptable documents: chart notes, clinic visit summaries, motor milestone assessments, laboratory values

Loss of muscle strength or motor ability, including loss of a motor milestone compared with baseline or since prior gene therapy.

Documentation sourcesMedical records such as chart notes and laboratory values that demonstrate change from baseline or since gene replacement therapy.

Exondys 51 (eteplirsen)

Generic and brand nameEteplirsen (brand name: Exondys 51)

Drug classAntisense oligonucleotide targeting exon 51 of dystrophin pre-mRNA to induce exon skipping and produce truncated dystrophin.

ReferenceSee Exondys 51 package insert (Sarepta Therapeutics, December 2024) for prescribing information and FDA-approved labeling.

Elevidys / gene replacement therapy

Example therapyElevidys (delandistrogene moxparvovec-rokl)

Context in policyReferenced as an example of prior gene replacement therapy that, if previously received, requires documentation of clinically meaningful functional decline to support subsequent eteplirsen initiation.

Required documentationMedical records documenting functional decline (loss of muscle strength/motor ability or loss of a motor milestone) since receiving Elevidys are required per the revised initial therapy criteria.