CurrentCareSourcePolicy N/A

UTILIZATION MANAGEMENT MEDICAL POLICY

CareSource utilization management policy evaluating clinical evidence and coverage recommendation for Elevidys (delandistrogene moxeparvovec) for treatment of Duchenne muscular dystrophy (DMD). States coverage stance and lists conditions not recommended for approval, with dosing, safety, and trial evidence summarized.

Policy Summary

PayerCareSource

PolicyUTILIZATION MANAGEMENT MEDICAL POLICY

Policy CodePolicy N/A

Change TypeAnnual Revision (No criteria changes)

Effective Date

Next Review Date

Key ActionClaims for Elevidys should be denied/not approved because the policy states approval is not recommended due to unclear clinical benefit; there are no Recommended Authorization Criteria provided.

SourceLink

POLICY UPDATE CHANGES

Annual Revision, Review Date = 08/14/2024 with 'No criteria changes.'

0Recommended Approvals (indications)

1Drug policies evaluated (Elevidys)

2Key safety checks (exon 8/9 deletion, anti-AAVrh74 titer)

Coverage Summary

CareSource utilization management policy evaluating Elevidys (delandistrogene moxeparvovec) for treatment of Duchenne muscular dystrophy (DMD). Policy stance: Not covered — approval not recommended due to unclear clinical benefit. Last review: 2024-08-14. Status: CURRENT.

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Quick thresholds & operational checks

Approved Elevidys dose1.33 x 10^14 vg/kg (or 10 mL/kg for patients <70 kg); fixed 9.31 x 10^15 vg total for patients ≥70 kg

Anti-AAVrh74 total antibody titer threshold< 1:400 before administration

Recommended Approvals (indications)0

Drug policies evaluated1 (Elevidys)

Key safety checksExon 8/9 deletion contraindication + anti-AAVrh74 titer check

Steroid prophylaxis guidanceStart corticosteroids 1 day before infusion and continue ≥60 days post-infusion unless earlier taper indicated

Medical-Necessity Criteria

Policy Statement / Conditions Not Recommended for Approval

Due to the lack of clinical efficacy data, approval is not recommended for Elevidys.

ALL of the following

Elevidys (delandistrogene moxeparvovec) is not recommended for approval for Duchenne Muscular Dystrophy (DMD) due to unclear clinical benefit.
Coverage is not recommended for any circumstances not listed in the Recommended Authorization Criteria (none provided).

Not Recommended for Coverage

The policy's rationale for not covering Elevidys centers on limited and unconvincing efficacy data and trial limitations. The EMBARK Phase III confirmatory trial did not meet the NSAA primary endpoint (between-group LSM difference 0.65 points; P = not significant). The Phase II study was affected by a dosing issue in which 60% of patients in Part I received a dose lower than the approved 1.33 x 10^14 vg/kg, limiting interpretability. Overall, the policy concludes that the clinical benefit is unclear and therefore approval is not recommended.

Provider Actions / Operational Notes

Denial Risk

No approval recommended

Claims for Elevidys should be denied/not approved because the policy states approval is not recommended due to unclear clinical benefit; there are no Recommended Authorization Criteria provided.

Documentation Required

Contraindication screening

Before consideration, providers must ensure patients do not have any deletion in exon 8 and/or exon 9 of the DMD gene (Elevidys contraindication) and check anti-AAVrh74 total antibody binding titer < 1:400 per the prescribing information.

Documentation Required

Steroid prophylaxis

If administered, corticosteroids should be started one day prior to Elevidys infusion and continued for a minimum of 60 days after the infusion, unless earlier tapering is clinically indicated (operational dosing recommendation).

Background & Evidence

Elevidys is an AAV vector–based gene therapy indicated for treatment of individuals ≥ 4 years of age with DMD, including ambulatory and non-ambulatory patients (the latter under accelerated approval based on micro-dystrophin expression). Safety concerns noted in the policy include risks of acute serious liver injury, immune-mediated myositis, myocarditis, and issues related to preexisting immunity to AAVrh74. The prescribing/dosing guidance specifies the recommended dose as 1.33 x 10^14 vg/kg (or 10 mL/kg for patients < 70 kg) or fixed 9.31 x 10^15 vg total for patients ≥ 70 kg, that re-administration is not recommended, and that corticosteroid prophylaxis should begin one day prior to infusion and continue for a minimum of 60 days post-infusion. Elevidys is contraindicated in patients with any deletion in exon 8 and/or exon 9 of the DMD gene, and the anti-AAVrh74 total antibody binding titer should be < 1:400 for administration.

Finding	Summary
EMBARK Phase III NSAA result	Between-group difference LSM 0.65 points (95% CI -0.45, 1.74); P = not significant
Phase II dosing issue	Retrospective analysis identified that 60% of patients in Part I received a dose lower than the approved 1.33 x 10^14 vg/kg

Elevidys: delandistrogene moxeparvovec-rokl intravenous infusion gene therapy for DMD.

NSAA: North Star Ambulatory Assessment, a functional motor score used as a primary endpoint in DMD trials.