ModifiedColorado Rocky Mountain Health PlansPolicy CS2026D0058Q

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

Medical benefit drug policy governing coverage and authorization criteria for Exondys 51 (eteplirsen) for treatment of Duchenne muscular dystrophy for Colorado Rocky Mountain Health Plans members.

Policy Summary

PayerColorado Rocky Mountain Health Plans

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

Policy CodePolicy CS2026D0058Q

Change TypeRevised coverage criteriaadded concomitant-use exclusion

Effective DateMay 1, 2026

Next Review DateN/A

Key ActionSubmit prior authorization with medical records confirming DMD diagnosis amenable to exon 51 skipping and required baseline functional measures.

SourceLink

POLICY UPDATE CHANGES

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

Updated References section to reflect the most current information.

Archived previous policy version CS2026D0058P.

30 mg/kg weeklytypical treatment dose

≤12 monthsinitial auth duration

J1428HCPCS code

1new concomitant-use exclusion

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Coverage and Clinical Criteria

Initial Therapy

Covered when ALL of the following are met

Initial Therapy Requirements: 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 dosing in accordance with FDA‑approved labeling; and initial authorization limited to no more than 12 months; and Exondys 51 will not be used concomitantly with Duvyzat (givinostat) or other exon skipping therapies (e.g., Amondys, Viltepso, Vyondys 53); and prescribed by, or in consultation with, a neurologist experienced in DMD.

See alternative ambulatory criteria below.

Baseline ambulatory/functional requirement: One of the following must be provided: (1) 6‑Minute Walk Test (6MWT) ≥ 300 meters while walking independently prior to initiation; OR (2) ambulatory without assistive device AND (NSAA > 17 OR time to rise < 7 seconds).6MWT ≥ 300 m OR NSAA >17 OR TTR <7s

Documentation via medical records required.

Gene therapy status: Either the patient has not previously received gene therapy (e.g., Elevidys) for DMD; OR if previously received gene therapy, submission of records documenting a clinically meaningful functional decline (loss of muscle strength/motor ability or loss of milestone) since gene replacement therapy.

Supports coverage in select previously gene‑treated patients with documented decline.

Continuation Therapy (Reauthorization)

Covered when ALL of the following are met

Continuation Requirements: Submission of medical records confirming the patient remains ambulatory without needing an assistive device; Exondys 51 will not be used concomitantly with Duvyzat (givinostat) or other exon skipping therapies (e.g., Amondys, Viltepso, Vyondys 53); prescribed by, or in consultation with, a neurologist experienced in DMD; dosing in accordance with FDA‑approved labeling; and reauthorization limited to no more than 12 months.

Ambulatory documentation required at each reauthorization.

Unproven / Not Medically Necessary

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

Coverage Rationale

Policy revised coverage rationale includes the following requirement:

Concomitant use restriction: Exondys 51 will not be used concomitantly with Duvyzat (givinostat)

Added in 05/01/2026 revision

FDA indication note: Indicated for DMD patients with a confirmed DMD mutation amenable to exon 51 skipping (accelerated approval based on increased dystrophin)

Informational; continued approval contingent on confirmatory trials

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

Per the May 1, 2026 policy revision, the coverage criteria explicitly exclude the concomitant use of Exondys 51 with Duvyzat (givinostat); requests for combined use will not meet the policy’s coverage requirements.

Treatment of muscular dystrophies that are not amenable to exon 51 skipping (for example, Becker muscular dystrophy) is not medically necessary and therefore excluded from coverage under this policy.

FDA approval for Exondys 51 is provided for informational purposes only and does not by itself establish coverage. The agent’s approval is under an accelerated pathway based on increased dystrophin; continued FDA approval may be contingent on confirmatory trials. The policy was revised 05/01/2026 to add an explicit restriction that Exondys 51 not be used concomitantly with Duvyzat (givinostat).

Billing and Diagnosis Codes

Applicable procedure/HCPCS codesHCPCS

J1428

Injection, eteplirsen, 10 mg.

Diagnosis codesICD-10

G71.01

Duchenne or Becker muscular dystrophy

6-Minute Walk Test (6MWT) — baseline threshold used in criteria

Baseline 6MWT threshold6-Minute Walk Test (6MWT) ≥ 300 meters while walking independently prior to initiation of Exondys 51.

Alternate ambulatory documentationIf 6MWT not provided: ambulatory without assistive device AND NSAA > 17 OR time to rise (TTR) < 7 seconds.

Measurement context6MWT must be performed without side‑by‑side assist, cane, walker, wheelchair, or similar assistance.

Use at initiationRequired as part of initial authorization documentation for Exondys 51 coverage pathway requiring functional baseline.

Prior Authorization, Documentation, and Operational Steps

Prior Authorization

Prior Authorization Required

Prior authorization is required. Submit medical records confirming diagnosis of Duchenne muscular dystrophy (DMD) with a DMD gene mutation amenable to exon 51 skipping, required baseline and ongoing functional status documentation, and a treatment plan. Initial approvals are limited to a maximum of 12 months; reauthorization approvals are limited to a maximum of 12 months and require documentation of continued ambulatory status and clinical benefit as specified below. Verify plan-specific federal, state, and contractual benefit requirements prior to rendering a coverage decision.

Affected product: Exondys 51 (eteplirsen)
Initial authorization: up to 12 months
Reauthorization: up to 12 months
Prescriber: Prescribed by or in consultation with a neurologist experienced in DMD
Plan verification: Check federal, state, and contractual benefit terms before applying this policy

Documentation Required

Key Terms and Clinical Definitions

Amenable to exon 51 skipping — definition and required genetic confirmation

DefinitionA confirmed mutation of the DMD gene that allows eteplirsen to bind exon 51 of dystrophin pre‑mRNA and induce exon skipping to produce an internally truncated dystrophin protein.

Required confirmationSubmission of medical records (e.g., chart notes, laboratory values) confirming the DMD gene mutation is amenable to exon 51 skipping is required for coverage.

Prevalence noteApproximately 13% of DMD patients have mutations amenable to exon 51 skipping.

6-Minute Walk Test (6MWT) — definition and policy threshold

DefinitionA standardized functional ambulation measure assessing distance walked in six minutes while walking independently.

Policy threshold for coverage

Clinical Background

Duchenne muscular dystrophy (DMD) is an X-linked, progressive neuromuscular disorder caused by mutations in the dystrophin gene that lead to loss of functional dystrophin, progressive weakness, loss of ambulation, and life‑threatening cardiopulmonary complications. Eteplirsen is an antisense oligonucleotide intended to induce skipping of exon 51 during dystrophin pre‑mRNA processing and is indicated for patients with a confirmed DMD mutation amenable to exon 51 skipping (approximately 13% of patients). The U.S. Food and Drug Administration granted accelerated approval based on increases in dystrophin; clinical benefit is being evaluated in confirmatory trials.

Policy Revision History

2026-05-01revisionLatest

Revised coverage criteria to add an exclusion: Exondys 51 (eteplirsen) must not be used concomitantly with Duvyzat (givinostat).