CurrentUnitedHealthcarePolicy CSPA2025D0058L

Exondys 51 (eteplirsen) (for Pennsylvania Only)

Medical Benefit Drug Policy governing coverage of Exondys 51 (eteplirsen) for treatment of Duchenne muscular dystrophy in Pennsylvania members of UnitedHealthcare Community Plan.

Policy Summary

PayerUnitedHealthcare

PolicyExondys 51 (eteplirsen) (for Pennsylvania Only)

Policy CodePolicy CSPA2025D0058L

Change TypeAdded initial-therapy requirement related to prior gene therapy status

Effective DateAug 1, 2025

Next Review Date

Key ActionObtain prior authorization and submit medical records confirming an exon 51–amenable DMD mutation and baseline standardized motor-function assessment.

SourceLink

POLICY UPDATE CHANGES

Added initial-therapy requirement that the patient either has not previously received gene therapy (e.g., Elevidys) or, if they have, must have documentation of clinically meaningful functional decline since receiving gene replacement therapy.

12 monthsinitial authorization duration

≤12 mocontinuation reauthorization period

J1428HCPCS code

G71.01Diagnosis code

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Coverage Criteria for Exondys 51 (eteplirsen)

Initial Therapy

Covered when ALL of the following are met:

Initial therapy criteria: Diagnosis of Duchenne muscular dystrophy by, or in consultation with, a neurologist with expertise in the diagnosis of DMD; and submission of medical records confirming the DMD gene mutation is amenable to exon 51 skipping; and submission of medical records documenting a baseline evaluation including a standardized assessment of motor function by a neurologist experienced in DMD; dosing in accordance with FDA-approved labeling; initial authorization for 12 months; and Exondys 51 is not used concomitantly with other exon-skipping therapies; and prescribed by, or in consultation with, a neurologist with expertise in the treatment of DMD.

Prior gene therapy status: One of the following must be met regarding prior gene therapy: (1) patient has not previously received gene therapy (e.g., Elevidys [delandistrogene moxparvovec-rokl]); OR (2) both of the following: patient previously received gene therapy (e.g., Elevidys) AND submission of medical records documents 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.

This criterion was added per policy revision.

Continuation Therapy

Covered when ALL of the following are met:

Continuation therapy criteria: Documentation of continued clinical benefit based on the prescriber's assessment, including evaluation with a standardized assessment of motor function by a neurologist experienced in DMD; dosing in accordance with FDA-approved labeling; reauthorization for no more than 12 months; Exondys 51 is not used concomitantly with other exon-skipping therapies; and prescribed by, or in consultation with, a neurologist with expertise in the treatment of DMD.

Unproven / Not Medically Necessary

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

Exondys 51 (eteplirsen) must not be used in patients whose DMD mutation is not amenable to exon 51 skipping. The policy also prohibits concomitant use with other exon‑skipping therapies for Duchenne muscular dystrophy — for example, Amondys (casimersen), Viltepso (viltolarsen), and Vyondys 53 (golodirsen). Use with any of these agents at the same time is excluded from coverage.

Providers should ensure that prior authorization documentation clearly shows the patient’s mutation is exon 51–amenable and that Exondys 51 will be used as a single exon‑skipping therapy (i.e., not in combination with other exon‑skipping agents). Failure to document mutation amenability or attempts at concomitant exon‑skipping therapy may result in denial of coverage.

Use of Exondys 51 for other forms of muscular dystrophy, such as Becker muscular dystrophy, is considered unproven and not medically necessary and is not covered under this policy.

Relevant Procedure and Diagnosis Codes

Relevant HCPCS CodesHCPCS

J1428

Injection, eteplirsen, 10 mg

Relevant Diagnosis CodesICD-10

G71.01

Duchenne or Becker muscular dystrophy

Provider Actions and Requirements

Prior Authorization

Prior Authorization Required

Prior authorization required. Submission of medical records confirming the DMD gene mutation is amenable to exon 51 skipping, and documentation of baseline standardized motor-function assessment by a neurologist with expertise in DMD, is required prior to initiation. Initial authorization will be for 12 months.

Codes/conditions: Exondys 51 (eteplirsen) for exon 51–amenable DMD
Required docs: chart notes, laboratory values, baseline standardized motor assessment

Denial Risk

Documentation and Denial Risk

Failure to provide required documentation (e.g., records confirming exon 51–amenable DMD mutation, baseline standardized motor assessment, or evidence of clinically meaningful decline when applicable) may result in denial of the request. Providers should submit all requested medical records and supporting documentation with the authorization request.

Background

Duchenne muscular dystrophy (DMD) is an X‑linked, progressive muscle‑wasting disorder caused by mutations in the dystrophin gene that lead to absence or deficiency of functional dystrophin, resulting in progressive loss of ambulation and respiratory and cardiac complications. Eteplirsen (Exondys 51) is an antisense oligonucleotide designed to skip exon 51 in eligible dystrophin gene mutations, permitting production of an internally truncated dystrophin protein in patients with mutations amenable to exon 51 skipping.

Definitions

Exon 51–amenable mutation — definition and clinical meaning

DefinitionA dystrophin gene mutation (typically out-of-frame deletions) for which exon 51 skipping can restore the reading frame and potentially allow production of an internally truncated dystrophin protein.

Clinical implicationPatients with exon 51–amenable mutations (≈13% of DMD patients) may produce truncated dystrophin after treatment with eteplirsen, which binds exon 51 of dystrophin pre-mRNA and causes exclusion of exon 51 during mRNA processing.

Typical mutation typeUsually out‑of‑frame deletions in the DMD gene that disrupt the reading frame and are correctable by skipping exon 51.

Intended biological effectExon 51 skipping is intended to restore the open reading frame to permit production of an internally truncated, partially functional dystrophin protein.

Regulatory contextExondys 51 (eteplirsen) is FDA‑approved for DMD patients with a confirmed mutation amenable to exon 51 skipping (accelerated approval based on increased dystrophin).