ModifiedUnitedHealthcarePolicy IEXD00126.07

Elevidys (Delandistrogene Moxeparvovec-Rokl) Medical Benefit Drug Policy

Defines UnitedHealthcare Individual Exchange medical benefit drug coverage criteria, limitations, and billing codes for Elevidys for eligible patients with Duchenne muscular dystrophy (DMD). Applies to Individual Exchange plans in all states except MA, NV, and NY.

Policy Summary

PayerUnitedHealthcare

PolicyMedical Benefit Drug Policy

Policy CodePolicy IEXD00126.07

Change TypeCoverage criteria revised (hepatic thresholds; troponin-I monitoring wording)

Effective DateMar 1, 2026

Next Review Date

Key ActionSubmit medical records confirming DMD diagnosis, gene mutation (not deletion in exon 8 or 9), ambulatory status, hepatic and cardiac evaluations, anti-AAVrh74 titer, and attestation of required monitoring; authorization limited to one lifetime treatment and for no longer than 45 days from approval or until age 6, whichever is first.

SourceLink

POLICY UPDATE CHANGES

Coverage criteria revised to clarify hepatic impairment definition and monitoring wording for troponin-I to align with FDA labeling.

Updated FDA section to reflect most current information.

1Lifetime treatments authorized per patient

4-5Covered patient ages (years)

>=1:400Anti-AAVrh74 titer exclusion threshold

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Coverage Summary

UnitedHealthcare Individual Exchange medical benefit covers Elevidys (delandistrogene moxeparvovec-rokl) with criteria for ambulatory patients aged 4 or 5 years. Coverage applies to Individual Exchange plans in all states except MA, NV, and NY. Refer to Policy Number IEXD00126.07 effective March 1, 2026 for full criteria, limitations, and billing (HCPCS J1413).

Medically Necessary / Initial Coverage Criteria

Elevidys is proven and medically necessary for the treatment of Duchenne muscular dystrophy (DMD) in patients who meet ALL of the following criteria:

Diagnosis of Duchenne muscular dystrophy by, or in consultation with, a pediatric neuromuscular specialist with expertise in the diagnosis of DMD
Genetic confirmation
- DMD gene mutation: A mutation in the DMD gene
- Exon exclusion: The mutation is not a deletion in exon 8 or exon 9
Age: Patient is aged 4 or 5 years of ageAge = 4 or 5 years
Only age 4 or 5 eligible under proven coverage
Ambulatory status: 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.)
Hepatic criteria
- No preexisting hepatic impairment: Patient does not have preexisting hepatic impairmentGGT <= 2 x upper limit of normal; total bilirubin <= upper limit of normal unless due to Gilbert's syndrome
  preexisting hepatic impairment defined as GGT > 2 x upper limit of normal or total bilirubin > the upper limit of normal not due to Gilbert's syndrome
- No acute liver disease: Patient does not have acute liver disease (e.g., acute hepatic viral infection)
Liver monitoring attestation: Prescriber attests that liver function (clinical exam, GGT, and total bilirubin) will be monitored weekly for the first 3 months following Elevidys administration and thereafter in accordance with the FDA approved labeling
Cardiac/troponin criteria
- LVEF: Patient does not have a left ventricle ejection fraction (LVEF) < 40%LVEF >= 40% (i.e., patient must NOT have LVEF < 40%)
- Troponin monitoring attestation: Prescriber attests that troponin-I will be monitored weekly for the first month following Elevidys administration and thereafter in accordance with the FDA approved labeling
Anti-AAVrh74 titer: Patient does not have an elevated anti-AAVrh74 total binding antibody titer ≥ 1:400Anti-AAVrh74 total binding antibody titer < 1:400
Corticosteroid regimen: Patient will receive a corticosteroid regimen prior to and following receipt of Elevidys in accordance with the FDA approved Elevidys labeling
Prescriber expertise: Elevidys is prescribed by, or in consultation with, a pediatric neuromuscular specialist with expertise in the treatment of DMD
No exon-skipping therapies: Patient will not receive exon-skipping therapies for DMD (e.g., Amondys, Exondys 51, Viltepso, Vyondys 53) concomitantly or following Elevidys treatment
No prior gene therapy: Patient has not previously received gene therapy for the treatment of DMD
No planned inpatient solely for administration: Provider does not request a planned inpatient admission for the sole purpose of administering Elevidys
Dosing: Elevidys dosing is in accordance with FDA approved labeling
Authorization limits: Authorization will be issued for no more than one treatment per lifetime and for no longer than 45 days from approval or until 6 years of age, whichever is firstAuthorization: max 1 lifetime treatment; duration <= 45 days from approval or until age 6, whichever is first
Limits authorization duration and lifetime treatments

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title

Not Medically Necessary / Unproven & Limitations of Use

Not Medically Necessary / Unproven

Elevidys is unproven and not medically necessary for the treatment of:

Becker muscular dystrophy (BMD)
Ambulatory age exclusions: Duchenne muscular dystrophy (DMD) in ambulatory patients < 4 years of age and ≥ 6 years of ageAge < 4 years OR Age >= 6 years
ambulatory patients outside 4-5 years are unproven
Non-ambulatory DMD: Duchenne muscular dystrophy (DMD) in patients at any age who are non-ambulatory

Limitations of Use / Not Recommended

Provider Actions

Prior Authorization

Prior authorization required with documentation

Submit medical records confirming DMD diagnosis, DMD gene mutation (not a deletion in exon 8 or 9), ambulatory status, hepatic and cardiac evaluations, anti-AAVrh74 total binding antibody titer, and attestation of required monitoring. Authorization is limited to one treatment per lifetime and for no longer than 45 days from approval or until 6 years of age, whichever is first.

HCPCS: J1413
Authorization limits: one lifetime treatment; authorization duration no more than 45 days from approval or until age 6, whichever is first

Documentation Required

Specialist involvement and consultation

Prescribing by, or consultation with, a pediatric neuromuscular specialist with expertise in the diagnosis and treatment of DMD must be documented in the records submitted for coverage.

Applicable Codes

Drug HCPCS codeHCPCSCovered

J1413

Injection, delandistrogene moxeparvovec-rokl, per therapeutic dose

Relevant diagnosis code (for reference)ICD-10

G71.01

Duchenne or Becker muscular dystrophy

Clinical Evidence

0.65EMBARK Phase 3 LS mean difference in NSAA at Week 52 (Elevidys vs placebo); 95% CI: -0.45 to 1.74; p=0.2441

4.3 vs 1.9Study 102 (age 4-5) LS mean change in NSAA at Week 48: Elevidys 4.3 points vs placebo 1.9 points

54.2%Study 103 (ENDEAVOR) cohort 1 mean micro-dystrophin change at Week 12; mean 54.2% (SD 42.6); p<0.0001

Elevidys was evaluated in AAV vector-based micro-dystrophin trials including EMBARK (Phase 3), ENDEAVOR (Study 103), and Study 102 (Phase II/Ib). Evidence of clinical benefit is strongest in the 4–5 year age subgroup; overall trials showed biological micro-dystrophin expression but mixed NSAA functional results. Safety signals include risk of hepatic injury and immune-related effects (immunogenicity); monitoring requirements (weekly liver tests initially and troponin-I monitoring) are specified and align with the FDA-approved labeling.

Background

Elevidys is an adeno-associated virus (AAV) vector-based gene therapy that delivers a micro-dystrophin gene to produce a shortened dystrophin protein. Clinical development includes Phase Ib/II/III studies (Study 102, ENDEAVOR/Study 103, and EMBARK) that assessed biological endpoints (micro-dystrophin expression by western blot at Week 12) and motor function (North Star Ambulatory Assessment, NSAA, up to Week 48/52). Trial results demonstrated robust micro-dystrophin expression in treated cohorts but mixed functional outcomes; subgroup analyses show the strongest NSAA improvements in the 4–5 year age group. Key safety concerns include hepatic injury (preexisting liver impairment is a contraindication) and immunogenicity, and monitoring requirements for liver function and troponin-I are specified in the policy and FDA labeling.

Revision History

2026-03-01material_changeLatest

Coverage criteria revised to clarify hepatic impairment definition (preexisting hepatic impairment defined as GGT > 2 x upper limit of normal or total bilirubin > upper limit of normal not due to Gilbert's syndrome) and prescriber attestation wording for troponin-I monitoring changed to 'in accordance with the FDA approved labeling'.

2026-03-01archived_policy

Previous policy version IEXD00126.06 archived.