ModifiedUnitedHealthcarePolicy CSPA2O2SDOOS8L

Exondys 51 (Eteplirsen) (for Pennsylvania Only)

Medical Benefit Drug Policy for Exondys 51 (eteplirsen) applicable only to Pennsylvania members, defining initial and continuation authorization criteria, unproven indications, applicable HCPCS and ICD-10 codes, and policy revision history.

Policy Summary

PayerUnitedHealthcare

PolicyExondys 51 (Eteplirsen) (for Pennsylvania Only)

Policy CodePolicy CSPA2O2SDOOS8L

Change TypeCoverage criteria revised

Effective DateAug 1, 2025

Next Review Date

Key ActionSubmit medical records confirming exon 51-amenable DMD mutation, baseline standardized motor function assessment, and diagnosis by or in consultation with a DMD-experienced neurologist; initial auth limited to 12 months.

POLICY UPDATE CHANGES

Revised coverage criteria for initial therapy to add requirement: one of the following — either patient has not previously received gene therapy for DMD OR both that patient has previously received gene therapy and documentation of clinically meaningful functional decline since gene replacement therapy.

PAApplicable State

J1428Key HCPCS Code

G71.01Relevant Diagnosis Code

With criteriaCoverage Stance

Coverage Summary

Background: Eteplirsen (Exondys 51) is an antisense oligonucleotide indicated under accelerated approval for Duchenne muscular dystrophy (DMD) patients with confirmed DMD gene mutations amenable to exon 51 skipping. This policy applies only to Pennsylvania members. Coverage stance: covered with criteria for patients who meet the policy's initial and continuation requirements. Authorization durations: initial authorization will be for 12 months, and reauthorization will be for no more than 12 months when continuation criteria are met.

Initial Therapy Criteria

Initial Therapy

Exondys 51 (eteplirsen) may be covered for the treatment of Duchenne muscular dystrophy (DMD) in patients who meet ALL of the following criteria:

ALL of the following

Submission of medical records (e.g., chart notes, laboratory values) confirming the mutation of the DMD gene is amenable to exon 51 skipping
Diagnosis of Duchenne muscular dystrophy by, or in consultation with, a neurologist with expertise in the diagnosis of DMD
Submission of medical records documenting a baseline evaluation, including a standardized assessment of motor function by a neurologist with experience in treating Duchenne muscular dystrophy, prior to beginning Exondys 51 therapy
ONE of the following
- Patient has not previously received gene therapy (e.g., Elevidys [delandistrogene moxparvovec-rokl]) for the treatment of DMD
- Both of the following
  - Patient has previously received gene therapy (e.g., Elevidys [delandistrogene moxparvovec-rokl]) for the treatment of DMD
  - 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 (e.g., Elevidys)
Exondys 51 is not used concomitantly with other exon skipping therapies for DMD (e.g., Amondys [casimersen]; Viltepso [viltolarsen]; Vyondys 53 [golodirsen])
Dosing is in accordance with the United States Food and Drug Administration approved labeling
Prescribed by, or in consultation with, a neurologist with expertise in the treatment of DMD
Initial authorization will be for 12 months
Authorization duration

Continuation / Reauthorization Criteria

Continuation Therapy

Reauthorization/continuation covered when ALL of the following are met:

ALL of the following

Exondys 51 is not used concomitantly with other exon skipping therapies for DMD (e.g., Amondys [casimersen], Viltepso [viltolarsen], Vyondys 53 [golodirsen])
Documentation of continued clinical benefit based on prescriber's assessment, including an evaluation with a standardized assessment of motor function ability by a neurologist with experience in treating Duchenne muscular dystrophy
Prescribed by, or in consultation with, a neurologist with expertise in the treatment of DMD
Dosing is in accordance with the United States Food and Drug Administration approved labeling
Reauthorization will be for no more than 12 months
Authorization duration

Prior Authorization

Reauthorization requirements

For reauthorization, submit documentation demonstrating continued clinical benefit based on the prescriber's assessment, including an evaluation with a standardized motor function assessment performed by a neurologist experienced in treating Duchenne muscular dystrophy. Reauthorization may be granted for up to 12 months. Dosing should follow the FDA‑approved labeling.

Affected code(s): J1428
Reauthorization duration: up to 12 months
Dosing per FDA label

Unproven / Not Medically Necessary

Exondys 51 is unproven and not medically necessary for the treatment of other forms of muscular dystrophy (e.g., Becker muscular dystrophy)

Denial Risk

Concomitant exon-skipping therapies prohibited

Claims may be denied if Exondys 51 is used concomitantly with other exon‑skipping therapies for DMD. Do not bill Exondys 51 when it is being used at the same time as other exon‑skipping agents.

Amondys (casimersen)
Viltepso (viltolarsen)
Vyondys 53 (golodirsen)

Provider Actions / Prior Authorization

Prior Authorization

Prior authorization required with documentation

Prior authorization is required with submission of medical records confirming a DMD mutation amenable to exon 51 skipping, a baseline standardized assessment of motor function performed by a neurologist experienced in DMD, and a diagnosis made by or in consultation with a DMD‑experienced neurologist. Initial authorization is limited to 12 months.

Affected code(s): J1428
Initial authorization: limited to 12 months

Documentation Required

Gene therapy history documentation

Document whether the patient previously received gene therapy (e.g., Elevidys). If the patient has received gene replacement therapy, include medical records showing a clinically meaningful functional decline (for example, loss of muscle strength or a motor milestone) since receiving gene therapy to satisfy the alternative pathway for initiation.

Other actionable billing notes: Dosing must follow the United States Food and Drug Administration approved labeling. Exondys 51 must be prescribed by, or in consultation with, a neurologist experienced in treating DMD, and baseline and ongoing standardized motor function assessments must be submitted as documentation. Claims may be denied if Exondys 51 is used concomitantly with other exon-skipping therapies for DMD (e.g., Amondys, Viltepso, Vyondys 53).

Clinical Evidence

-68.9 mPROMOVI 6MWT change at 96 weeks (attenuation vs external controls: PROMOVI -68.9 m; external controls -133.8 m)

0.93%Dystrophin protein average (phase 2/extension) at 180 weeks (0.93% of healthy subjects); increases in dystrophin-positive fibers reported

AcceleratedFDA approval based on increased dystrophin in skeletal muscle; continued approval contingent on verification of clinical benefit

Evidence context: Clinical evidence includes small randomized trials and open-label extension studies showing increases in dystrophin production and dystrophin-positive fibers; PROMOVI and other studies report increases in dystrophin and attenuation of decline on motor and pulmonary measures in some analyses. However, the FDA prescribing information and the ICER review indicate that the clinical benefit remains uncertain; continued approval is contingent on verification of clinical benefit in confirmatory trials.

Background & Definitions

Eteplirsen is an antisense oligonucleotide designed to skip exon 51 of dystrophin pre-mRNA to allow production of an internally truncated dystrophin protein. Approximately 13% of DMD patients have mutations amenable to exon 51 skipping. The indication for Exondys 51 was granted under accelerated approval based on increased dystrophin in skeletal muscle; continued approval may be contingent upon verification of clinical benefit.

Term	Definition
Exon 51 skipping amenable mutation
A confirmed mutation of the DMD gene that is amenable to exon 51 skipping, as documented in medical records/laboratory testing (patients with out-of-frame deletions amenable to exon 51 skipping; ~13% of DMD patients).
Clinically meaningful functional decline
Loss of muscle strength/motor ability such as loss of a motor milestone since receiving gene replacement therapy (used to document decline after prior gene therapy).