Modifiedsierra health and lifePolicy 2026D0058O

Exondys 51 (eteplirsen) — Medical Benefit Drug Policy (coverage criteria)

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Medical benefit drug policy describing coverage criteria, clinical evidence, and coding for Exondys 51 (eteplirsen) for treatment of Duchenne muscular dystrophy; applies to UnitedHealthcare Commercial and Individual Exchange plans.

Policy Summary

Payersierra health and life

PolicyExondys 51 (eteplirsen) — Medical Benefit Drug Policy

Policy CodePolicy 2026D0058O

Change TypeRevised coverage criteriaadded concomitant use exclusion

Effective DateMay 1, 2026

Next Review Date

Key ActionObtain prior authorization with medical records documenting a DMD mutation amenable to exon 51 skipping and ambulatory function (e.g., 6MWT ≥300 m).

SourceLink

POLICY UPDATE CHANGES

Revised coverage criteria; added criterion requiring Exondys 51 will not be used concomitantly with Duvyzat (givinostat).

Added Application section to indicate this policy applies to UnitedHealthcare Commercial and Individual Exchange benefit plans.

Transferred content to shared policy template that applies to both UnitedHealthcare Commercial and Individual Exchange benefit plans.

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Coverage Criteria for Exondys 51 (eteplirsen)

inv-01: Initial Therapy — Covered when ALL of the following are met

Covered when ALL of the following are met

Initial Authorization Criteria: Diagnosis of Duchenne muscular dystrophy by, or in consultation with, a neurologist with expertise in DMD; and submission of medical records confirming the DMD gene mutation is amenable to exon 51 skipping; and one of the following ambulatory criteria: (1) 6-Minute Walk Test (6MWT) ≥ 300 meters while walking independently; OR (2) patient is ambulatory without need for an assistive device and either NSAA > 17 OR time to rise (TTR) < 7 seconds; and patient has not previously received gene therapy for DMD OR if previously received gene therapy, submission of records documenting a clinically meaningful functional decline since gene replacement therapy; and Exondys 51 will not be used concomitantly with givinostat (Duvyzat) or other exon skipping therapies for DMD; and Exondys 51 is prescribed by, or in consultation with, a neurologist with expertise in the treatment of DMD; and dosing is in accordance with the United States FDA–approved labeling; and initial authorization will be for no more than 12 months.see nodes

Ambulatory eligibility is satisfied by either the 6MWT threshold OR the alternate ambulatory assessments; prior gene therapy is allowed only with documented clinically meaningful decline since gene therapy.

inv-04: Initial therapy / Coverage criteria — Covered when ALL of the following are met

Covered when ALL of the following are met

Indication: Patient has Duchenne muscular dystrophy with a confirmed DMD gene mutation amenable to exon 51 skipping (FDA accelerated approval indication).

Evidence of potential benefit: Treatment with eteplirsen is supported by increased dystrophin in skeletal muscle and non-randomized/open-label trial data (PROMOVI and phase 2) showing attenuation of decline on functional measures compared with external controls; clinical benefit remains uncertain and confirmatory evidence is required per FDA accelerated approval.

PROMOVI: 96-week data; see clinical evidence for details.

Treatment parameters: Eteplirsen administered as 30 mg/kg IV weekly as studied (PROMOVI); candidates in trials were ambulatory and met pulmonary and corticosteroid stability criteria typical of study populations.

Administration ~35–60 minutes per infusion as reported in PROMOVI.

Concomitant therapy exclusion: Eteplirsen will not be used concomitantly with Duvyzat (givinostat).

Coverage criteria revised to add this exclusion.

inv-02: Continuation Therapy — Covered when ALL of the following are met

Covered when ALL of the following are met

Continuation Authorization Criteria: Submission of medical records confirming the patient remains ambulatory without needing an assistive device; Exondys 51 will not be used concomitantly with givinostat (Duvyzat) or other exon skipping therapies for DMD; prescribed by, or in consultation with, a neurologist with expertise in the treatment of DMD; dosing is in accordance with the United States FDA–approved labeling; and reauthorization will be for no more than 12 months.see nodes

Continuation requires ongoing ambulatory status and absence of excluded concomitant therapies.

Concomitant use exclusions: Exondys 51 (eteplirsen) will not be used concomitantly with Duvyzat (givinostat) or with other exon skipping therapies for DMD (for example, Amondys/casimersen, Viltepso/viltolarsen, Vyondys 53/golodirsen). This exclusion was added as a revised coverage criterion in the policy update effective 05/01/2026 and may lead to denial if concomitant use is documented.

Operational notes for authorization: prior authorization is required and requests must document that the member will not be receiving any excluded concomitant therapies. Failure to document absence of these concomitant agents is listed among the triggers for denial; verify medication history and include explicit statements in submitted records that Duvyzat or other exon‑skipping agents are not being prescribed alongside Exondys 51.

Indication limitation: Exondys 51 is indicated only for patients with Duchenne muscular dystrophy who have a confirmed DMD gene mutation amenable to exon 51 skipping, per the FDA accelerated approval. Documentation confirming the specific mutation and that it is amenable to exon 51 skipping is required for coverage.

Clinical scope: the clinical evidence and trial experience for eteplirsen are limited to patients with exon 51 amenable mutations; the drug has not been studied in DMD that is not amenable to exon 51 skipping, and continued approval depends on verification of clinical benefit in confirmatory trials.

Not medically necessary — other forms: Exondys 51 is unproven and not medically necessary for the treatment of other forms of muscular dystrophy (for example, Becker muscular dystrophy). The published clinical trials and regulatory indication do not support use outside DMD patients with exon 51 amenable mutations.

Not studied outside indication: eteplirsen has not been studied in other muscular dystrophies and the evidence does not support extension of use to non‑DMD muscular dystrophies; requests for coverage in these diagnoses should be considered not medically necessary.

Use not supported for mutations not amenable to exon 51 skipping: Use of eteplirsen for DMD mutations that are not amenable to exon 51 skipping is not supported by clinical data and is not medically necessary. Trials and the FDA label are limited to exon 51–amenable mutations; eteplirsen has not been studied in patients with other mutation classes.

Authorization implication: prior authorization requests must include genetic testing results confirming the mutation is amenable to exon 51 skipping. Absence of such documentation or documentation of a non‑amenable mutation should result in denial per policy criteria.

inv-03: Unproven Indications — 1 top-level node

Unproven/Not Medically Necessary: Use of Exondys 51 (eteplirsen) for other forms of muscular dystrophy (e.g., Becker muscular dystrophy) is unproven and not medically necessary.

Initial Therapy Requirements

inv-23: Initial Therapy — initial therapy coverage requirements

Covered when ALL of the following are met

Initial Authorization Criteria: Confirmed diagnosis of Duchenne muscular dystrophy and submission of medical records confirming a DMD gene mutation amenable to exon 51 skipping; documentation of ambulatory function defined as 6MWT ≥ 300 meters while walking independently OR ambulatory without assistive device plus NSAA >17 or time to rise <7 seconds; prescribing by, or in consultation with, a neurologist experienced in DMD; dosing per FDA labeling; not concurrently on excluded therapies (e.g., givinostat or other exon skipping agents); initial authorization up to 12 months; if prior gene therapy received, documentation of clinically meaningful functional decline is required.

inv-24: Initial therapy regimen — as studied in PROMOVI

Dose and Administration (as studied in PROMOVI): 30 mg/kg IV weekly administered over approximately 35–60 minutes as used in PROMOVI; trial population were ambulatory males meeting typical inclusion criteria (e.g., pulmonary function and stable corticosteroid use).

See PROMOVI 96‑week study results for efficacy and safety outcomes.

Continuation / Reauthorization Criteria

inv-25: Continuation Therapy — continuation therapy requirements

Covered when ALL of the following are met

Continuation: Patient remains ambulatory without needing an assistive device; no concomitant use of givinostat or other exon skipping therapies; prescribed by, or in consultation with, a neurologist experienced in DMD; dosing per FDA labeling; reauthorization up to 12 months.

inv-26: Continuation therapy — continued use consistent with initial coverage criteria and tolerability

Continued use may be considered when ongoing treatment demonstrates continued use consistent with initial coverage criteria and tolerability

Ongoing benefit and tolerability: Continued treatment may be supported by stabilization or slowed decline in functional measures consistent with trial outcomes (e.g., attenuation of 6MWT decline observed in PROMOVI) and absence of significant adverse effects.

PROMOVI showed attenuation of decline over 96 weeks; document functional measures and tolerability in medical records.

Billing and Diagnosis Codes

HCPCS CodesHCPCSCovered

J1428

Injection, eteplirsen, 10 mg

Diagnosis CodesICD-10

G71.01

Duchenne or Becker muscular dystrophy

inv-11: 6-Minute Walk Test ambulatory requirement — value threshold

6-Minute Walk Test threshold>= 300 meters while walking independently (without side-by-side assist, cane, walker, wheelchair, etc.)

Alternative ambulatory criteriaAmbulatory without assistive device plus NSAA score >17

Alternative ambulatory criteria (time)Ambulatory without assistive device plus time to rise (TTR) <7 seconds

Use for initial authorizationDocumentation of 6MWT >=300 m OR the listed alternative ambulatory measures required prior to initiating Exondys 51

Provider Requirements and Prior Authorization

Prior Authorization

Prior Authorization Required

Prior authorization is required before initiation of Exondys 51. Submit medical records confirming diagnosis of Duchenne muscular dystrophy (DMD) and that the patient’s DMD gene mutation is amenable to exon 51 skipping. Include documentation of baseline ambulatory function (see examples below). Initial authorization is for up to 12 months; reauthorization requests must include updated documentation of continued ambulatory status and adherence to FDA‑approved dosing.

Affected plans: UnitedHealthcare Commercial and Individual Exchange benefit plans — verify member‑specific benefits and applicable federal/state mandates before submission.
Required documentation examples: chart notes, genetic test results confirming exon 51 amenability, 6‑Minute Walk Test (6MWT) results, North Star Ambulatory Assessment (NSAA) score, time to rise (TTR) measurements, notes on use (or non‑use) of assistive devices.
Triggers for denial: missing documentation of exon 51‑amenable mutation; lack of documentation that patient is ambulatory (per criteria); concurrent use of disallowed therapies (see concomitant therapy restrictions).
Prior authorization duration: initial and reauthorization approvals limited to no more than 12 months.

Administration and Site of Care Considerations

Billing Rule

Infusion center — IV infusion per label; weekly over ~35–60 minutes; site‑of‑care considerations

Administer Exondys 51 as an IV infusion per the FDA‑aligned regimen used in trials: weekly intravenous infusion administered over approximately 35–60 minutes; site‑of‑care rules and benefit limitations may apply per plan policies.

30 mg/kg weekly IV infusion administered over ~35–60 minutes as reported in PROMOVI ([[chunk 19]])
Site of care considerations and Provider Administered Drugs policy may govern benefit limitations ([[chunk 6]])

Billing Rule

Infusion center — administer IV over 35–60 minutes in appropriate infusion setting

Eteplirsen is administered intravenously over approximately 35–60 minutes in a setting appropriate for IV infusion; follow plan rules for acceptable infusion settings.

Step Therapy and Alternative Treatments

Requirement	Details
Concomitant therapy exclusion	Exondys 51 will not be used concomitantly with Duvyzat (givinostat) or other exon skipping therapies for DMD (e.g., Amondys/casimersen, Viltepso/viltolarsen, Vyondys 53/golodirsen).
Coverage impact	Policy prohibits concomitant use of Exondys 51 with the specified alternative or investigational DMD therapies; use with these agents may trigger denial of coverage.

Statement	Details
Step therapy requirement	No specific step therapy (prior failure of another DMD therapy) is required by this policy for initiation of Exondys 51.
Evidence context	Clinical evidence for eteplirsen is limited; ICER found data insufficient and benefits uncertain, and FDA granted accelerated approval based on dystrophin increase with continued approval contingent on confirmatory trials.

Definitions and Eligibility

inv-21: Definition — Exon 51 skipping molecular approach

DefinitionExon 51 skipping is a molecular approach that excludes exon 51 during mRNA processing to permit production of an internally truncated dystrophin protein in patients with deletions amenable to that change.

MechanismAntisense oligonucleotide binding to exon 51 of dystrophin pre-mRNA to cause exon exclusion during splicing

Clinical intentAllows production of internally truncated, partially functional dystrophin in eligible DMD patients

inv-22: Definition — Patients with confirmed DMD mutation amenable to exon 51 skipping per FDA label

Eligibility per FDA labelPatients with Duchenne muscular dystrophy who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping

Regulatory note

Clinical Background

Background: Duchenne muscular dystrophy (DMD) is an X‑linked progressive muscle degenerative disease caused by mutations in the dystrophin gene that lead to progressive loss of ambulation and cardiopulmonary complications. Eteplirsen is an antisense oligonucleotide designed to induce exon 51 skipping of dystrophin pre‑mRNA to allow production of an internally truncated dystrophin protein in patients with deletions amenable to that change.

Epidemiology and mechanism: approximately 13% of DMD patients have mutations amenable to exon 51 skipping. The FDA granted Exondys 51 accelerated approval for treatment of patients with confirmed DMD gene mutations amenable to exon 51 skipping based on increases in dystrophin observed in treated skeletal muscle; continued approval may be contingent on verification of clinical benefit in confirmatory trials.

Clinical trial context: the primary clinical data derive from non‑randomized and open‑label studies (including PROMOVI) in which eteplirsen was administered as 30 mg/kg IV weekly; trial results showed increased dystrophin but clinical benefit on functional endpoints remains uncertain, with PROMOVI reporting attenuation of decline versus external controls over 96 weeks.