Currentsierra health and lifePolicy 2025D0058N

Exondys 51 (eteplirsen) for Duchenne muscular dystrophy

Defines medical benefit coverage criteria, initial and continuation authorization requirements, applicable HCPCS/ICD-10 codes, and limitations for Exondys 51 (eteplirsen) treatment of Duchenne muscular dystrophy in members whose mutation is amenable to exon 51 skipping.

Policy Summary

Payersierra health and life

PolicyExondys 51 (eteplirsen) for Duchenne muscular dystrophy

Policy CodePolicy 2025D0058N

Change TypeCoverage criteria revised; Benefit Considerations updated

Effective DateJul 1, 2025

Next Review Date

Key ActionSubmit medical records confirming diagnosis, genotype amenable to exon 51 skipping, required functional measures, and prior gene therapy status (with documentation if applicable); initial authorization limited to 12 months.

POLICY UPDATE CHANGES

Revised coverage criteria for initial therapy to add requirement that either the patient has not previously received gene therapy or, if they have, submission of records documenting a clinically meaningful functional decline since gene therapy.

Updated Benefit Considerations section to reflect the most current information.

1Indication covered (DMD amenable to exon 51 skipping)

12 monthsInitial authorization duration (max)

12 monthsReauthorization duration (max)

Coverage Summary

Defines medical benefit coverage criteria, initial and continuation authorization requirements, applicable HCPCS/ICD-10 codes, and limitations for Exondys 51 (eteplirsen) treatment of Duchenne muscular dystrophy in members whose mutation is amenable to exon 51 skipping. Coverage stance: Covered with criteria. Effective date: 2025-07-01; Last review: 2025-07-01.

Initial Therapy Criteria

Continuation Therapy Criteria

Continuation Therapy

Covered when ALL of the following are met:

ALL of the following

Submission of medical records confirming that the patient is ambulatory without needing an assistive device (e.g., without side-by-side assist, cane, walker, wheelchair, etc.)
Exondys 51 is not used concomitantly with other exon skipping therapies for DMD (e.g., Amondys [casimersen], Viltepso [viltolarsen], Vyondys 53 [golodirsen])
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 months12 months (max)

Unproven / Not Medically Necessary

ALL of the following

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

Applicable Codes

Applicable HCPCS (drug)HCPCSCovered

J1428

Injection, eteplirsen, 10 mg

Diagnosis codes (reference)ICD-10Covered

G71.01

Duchenne or Becker muscular dystrophy

Provider Actions / Prior Authorization

Prior Authorization

Prior authorization required for initial therapy

For initial Exondys 51 (eteplirsen) therapy, submit medical records confirming: diagnosis of Duchenne muscular dystrophy; a DMD gene mutation amenable to exon 51 skipping; required functional measures (6-Minute Walk Test ≥ 300 m or documentation of ambulation plus NSAA > 17 or TTR < 7 seconds); and prior gene therapy status (either no prior gene therapy or, if prior gene therapy, records documenting a clinically meaningful functional decline since gene therapy). Initial authorization will be for no more than 12 months.

Affected HCPCS code: J1428
Initial authorization limited to 12 months

Prior Authorization

Reauthorization requirements

For reauthorization of Exondys 51, submit medical records confirming the patient remains ambulatory without an assistive device and that dosing follows the FDA-approved labeling. Reauthorization will be for no more than 12 months.

Clinical Evidence

-68.9 mPROMOVI 96-week change in 6MWT (mean decrease from 374.6 m to 256.2 m)

67.3 mPhase 2 ambulation benefit vs placebo (Mendell)

0.93%Average dystrophin after 180 weeks (of normal)

Eteplirsen was approved under the FDA’s accelerated approval pathway based on observed increases in dystrophin in skeletal muscle; continued approval may depend on verification of clinical benefit in confirmatory trials. Clinical studies demonstrated increases in dystrophin (for example average dystrophin ~0.93% of normal after long-term treatment and increases in dystrophin-positive fibers), but definitive clinical benefit remains uncertain per prescribing information and reviews. Approximately 13% of DMD patients have mutations amenable to exon 51 skipping. Key trial endpoints referenced in the evidence include dystrophin production, the 6-Minute Walk Test (6MWT) distance, North Star Ambulatory Assessment (NSAA) score, time to rise (TTR), loss of ambulation (LOA), and pulmonary function measures such as percent predicted forced vital capacity (FVC%p).

Definitions & Background

6MWT: 6-Minute Walk Test, reported as 6-minute walk distance (meters).

NSAA: North Star Ambulatory Assessment, a scale assessing motor function in ambulant DMD patients.

TTR: Time to rise from the floor (seconds).

Eteplirsen is an antisense oligonucleotide approved under accelerated approval to treat DMD in patients with a DMD gene mutation amenable to exon 51 skipping. Approximately 13% of DMD patients have deletions amenable to exon 51 skipping.

Key Functional Thresholds

6-Minute Walk Test (6MWT)>= 300 meters

North Star Ambulatory Assessment (NSAA)> 17

Time to Rise (TTR)< 7 seconds

Revision History

2025-07-01material_changeLatest

Revised coverage criteria for initial therapy to add a branching requirement: either the patient has not previously received gene therapy for DMD (e.g., Elevidys) or, if they have, submission of medical records documenting a clinically meaningful functional decline since receiving gene replacement therapy is required.

2025-07-01non-material_updateLatest

Supporting information revised: Benefit Considerations section updated to reflect the most current information; previous policy version 2025D0058M archived.

2025-07-01