ModifiedUnitedHealthcarePolicy IEXDO0126.05

Elevidys (delandistrogene moxeparvovec-rokl) gene therapy for Duchenne muscular dystrophy

UnitedHealthcare Individual Exchange medical benefit drug policy governing coverage criteria, clinical requirements, and applicable codes for Elevidys (delandistrogene moxeparvovec-rokl) for Individual Exchange plans in all states except MA, NV, and NY.

Policy Summary

PayerUnitedHealthcare

PolicyElevidys (delandistrogene moxeparvovec-rokl) gene therapy for Duchenne muscular dystrophy

Policy CodePolicy IEXDO0126.05

Change TypeCoverage criteria modified; monitoring & eligibility added

Effective DateMay 1, 2025

Next Review Date

Key ActionSubmit medical records confirming diagnosis, DMD gene mutation (not deletion in exon 8 or 9), ambulatory status, and attest to required liver and troponin monitoring; authorizations limited to one lifetime treatment and up to 45 days from approval or until age 6.

POLICY UPDATE CHANGES

Added criterion requiring that the patient does not have preexisting hepatic impairment; acute liver disease, chronic hepatic condition, or elevated GGT.

Added criterion requiring that the patient does not have a left ventricle ejection fraction (LVEF) < 40%.

Added prescriber attestation requirement that troponin-I will be monitored weekly for the first month following administration and thereafter per prescribing information.

Replaced criterion 'the patient has never received Elevidys treatment in their lifetime' with 'the patient has not previously received gene therapy for the treatment of DMD'.

Added language that Elevidys is unproven and not medically necessary for treatment of BMD, DMD in ambulatory patients <4 years and ≥6 years, and DMD in non-ambulatory patients at any age.

Updated Clinical Evidence, FDA, and References sections to reflect current information.

1Therapeutic dose HCPCS code

1Covered indication (ambulatory ages 4-5)

3Unproven / Not Medically Necessary categories

Coverage Summary & Stance

Coverage stance: covered_with_criteria. Subject: Elevidys (delandistrogene moxeparvovec‑rokl) gene therapy for Duchenne muscular dystrophy. Scope: UnitedHealthcare Individual Exchange medical benefit drug policy governing coverage criteria, clinical requirements, and applicable codes for Elevidys for Individual Exchange plans in all states except MA, NV, and NY. Effective date: 2025-05-01; Last review: 2025-05-01.

Proven / Medically Necessary Criteria for Elevidys

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

ALL of the following

Diagnosis and genetic confirmation: Submission of medical records (e.g., chart notes, laboratory values) confirming diagnosis of Duchenne muscular dystrophy by, or in consultation with, a pediatric neuromuscular specialist with expertise in the diagnosis of DMD
Submission of medical records confirming a mutation in the DMD gene
The DMD mutation is not a deletion in exon 8 or exon 9
Age: Patient is aged 4 or 5 years of age
Ambulatory status: Submission of medical records (e.g., chart notes) confirming that the patient is ambulatory without needing an assistive device
policy text truncated but intent: ambulatory status required
Liver-related requirements
- 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
- No preexisting hepatic impairment: Patient does not have preexisting hepatic impairment; acute liver disease (e.g., acute hepatic viral infection), chronic hepatic condition, or elevated GGT
Cardiac function: Patient does not have a left ventricle ejection fraction (LVEF) < 40%>= 40%
Patient must not have LVEF < 40%
Cardiac / troponin monitoring
- Monitoring attestation: Prescriber attests that troponin-I will be monitored weekly for the first month following Elevidys administration and thereafter per recommendations in the prescribing information
Antibody titer: Patient does not have an elevated anti-AAVrh74 total binding antibody titer ≥ 1:400< 1:400
Corticosteroid regimen: Patient will receive a corticosteroid regimen to and following receipt of Elevidys in accordance with the FDA approved Elevidys labeling
Specialist prescriber: Elevidys is prescribed by, or in consultation with, a pediatric neuromuscular specialist with expertise in the treatment of DMD
Concomitant therapies exclusion: Patient will not receive exon-skipping therapies for DMD (e.g., Viltepso [viltolarsen], Vyondys 53 [golodirsen]) concomitantly or following Elevidys treatment
Dosing: Elevidys dosing is in accordance with FDA approved labeling
Prior gene therapy: Patient has not previously received gene therapy for the treatment of DMD
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 first
limits on authorization duration and lifetime administrations

Unproven / Not Medically Necessary

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

ANY of the following

Unproven - ambulatory age groups: Duchenne muscular dystrophy (DMD) in ambulatory patients < 4 years of age and ≥ 6 years of agePatient age not 4 or 5
Unproven - Becker: Becker muscular dystrophy (BMD)
Unproven - non-ambulatory: Duchenne muscular dystrophy (DMD) in patients at any age who are non-ambulatory

Applicable Codes

HCPCS - DrugHCPCSCovered

J1413

Injection, delandistrogene moxeparvovec-rokl, per therapeutic dose

Diagnosis Codes - ReferenceICD-10

G71.01

Duchenne or Becker muscular dystrophy

Prior Authorization, Documentation & Billing Requirements

Prior Authorization

Prior authorization required and limited to one lifetime treatment

Authorization is limited to one Elevidys treatment per patient lifetime and the authorization period will be for no longer than 45 days from approval or until the patient turns 6 years of age, whichever occurs first.

Affected code: J1413
Authorization: no more than one treatment per lifetime
Authorization duration: no longer than 45 days from approval or until 6 years of age, whichever is first

Documentation Required

Submit medical records and specialist documentation

Submit medical records confirming diagnosis and specialist involvement, genetic confirmation of a DMD mutation (excluding deletions in exon 8 or exon 9), and documentation that the patient is ambulatory without an assistive device. The diagnosis must be made by, or in consultation with, a pediatric neuromuscular specialist with expertise in DMD.

Clinical Evidence & Trial Metrics

Background: Elevidys clinical development included multiple phase trials (e.g., SRP‑9001 studies and EMBARK) with mixed functional outcomes across studies. Study 102 met its biological endpoint for micro‑dystrophin expression but did not show a statistically significant NSAA change at Week 48 overall, with exploratory age subgroup analyses indicating differing results by age. EMBARK (Study 301) did not demonstrate a statistically significant difference in Week 52 NSAA change versus placebo. Evidence note: benefit appears to be age‑dependent in some studies, with the 4–5 year subgroup in Study 102 showing improved NSAA scores while older subgroups did not; other analyses (Study 102 Part 2 crossover) showed a statistically significant NSAA advantage versus external controls in a treated crossover cohort.

Background & Definitions

Elevidys is a recombinant rAAVrh74‑based gene therapy that delivers a micro‑dystrophin transgene to produce a shortened dystrophin protein. Duchenne muscular dystrophy (DMD) is an X‑linked, progressive neuromuscular disorder with typical onset between ages 3 and 5, causing progressive muscle weakness and life‑threatening cardiac and respiratory complications. Clinical development for Elevidys has included multiple studies (e.g., Study 102, Study 103/ENDEAVOR, and the EMBARK Phase 3 trial) evaluating micro‑dystrophin expression and functional outcomes such as NSAA over follow‑up periods.

Term	Definition
Elevidys	Brand name for delandistrogene moxeparvovec-rokl, an rAAVrh74-based micro-dystrophin gene therapy for DMD.
Ambulatory	Patient is able to walk without needing an assistive device (per policy requirement).

Key Clinical Thresholds

Coverage Thresholds

Anti-AAVrh74 total binding antibody titer< 1:400 (patient must not have a titer ≥ 1:400)

Left ventricle ejection fraction (LVEF)>= 40% (patient must not have LVEF < 40%)

Patient age for coverage4 or 5 years of age (ambulatory)

Policy Revision History

2025-05-01policy_modifiedLatest

Added criterion requiring that the patient does not have preexisting hepatic impairment (acute liver disease, chronic hepatic condition, or elevated GGT) and added prescriber attestation that liver function (clinical exam, GGT, total bilirubin) will be monitored weekly for the first 3 months and thereafter per FDA labeling.

2025-05-01policy_modifiedLatest

Added criterion requiring that the patient does not have a left ventricle ejection fraction (LVEF) < 40%.

Policy Summary

PayerUnitedHealthcare

PolicyElevidys (delandistrogene moxeparvovec-rokl) gene therapy for Duchenne muscular dystrophy

Policy CodePolicy IEXDO0126.05

Change TypeCoverage criteria modified; monitoring & eligibility added

Effective DateMay 1, 2025

Next Review Date

On This Page

Chart notes confirming diagnosis of Duchenne muscular dystrophy by or in consultation with a pediatric neuromuscular specialist
Medical records confirming a mutation in the DMD gene (mutation must NOT be a deletion in exon 8 or exon 9)
Documentation (e.g., chart notes) that the patient is ambulatory without needing an assistive device

Documentation Required

Laboratory and monitoring attestations required

Prescriber attestations and laboratory/monitoring requirements are required: weekly liver function monitoring (clinical exam, GGT, total bilirubin) for the first 3 months post-administration and then per FDA labeling; weekly troponin-I monitoring for the first month post-administration and then per prescribing information. The prescriber must confirm the patient does not have preexisting hepatic impairment (including acute hepatic viral infection, chronic hepatic condition, or elevated GGT) and that the anti-AAVrh74 total binding antibody titer is not elevated (i.e., < 1:400).

Liver monitoring: clinical exam, GGT, and total bilirubin weekly for first 3 months, then per FDA labeling
Troponin-I monitoring: weekly for first month, then per prescribing information
Hepatic status: patient must not have preexisting hepatic impairment (e.g., acute hepatic viral infection), chronic hepatic condition, or elevated GGT
Anti-AAVrh74 total binding antibody titer must be < 1:400 (not elevated ≥ 1:400)

Billing Rule

HCPCS billing

Bill Elevidys using HCPCS code J1413 and report per therapeutic dose.

HCPCS code: J1413 (Injection, delandistrogene moxeparvovec-rokl, per therapeutic dose)
Report Elevidys using J1413 per therapeutic dose

Added prescriber attestation requirement that troponin‑I will be monitored weekly for the first month following administration and thereafter per prescribing information.

2025-05-01policy_revisedLatest

Replaced the criterion 'the patient has never received Elevidys treatment in their lifetime' with 'the patient has not previously received gene therapy for the treatment of DMD'.

2025-05-01policy_modifiedLatest

Added language that Elevidys is unproven and not medically necessary for Becker muscular dystrophy (BMD); for DMD in ambulatory patients <4 years and ≥6 years of age; and for DMD in non‑ambulatory patients at any age.

2025-05-01documentation_updatedLatest

Updated Clinical Evidence, FDA, and References sections to reflect current information.