ModifiedCignaPolicy IP0571

Muscular Dystrophy - Gene Therapy - Elevidys

Defines Cigna's coverage stance and clinical information for Elevidys gene therapy for Duchenne muscular dystrophy (DMD) as used in benefit plans administered by Cigna Companies.

Policy Summary

PayerCigna

PolicyMuscular Dystrophy - Gene Therapy - Elevidys

Policy CodePolicy IP0571

Change TypeSafety-driven non-approval; investigational language added

Effective Date

Next Review Date

Key ActionSubmit prior authorization and include HCPCS J1413 on claims, but expect denials as approval is not supported given current safety and efficacy concerns.

SourceLink

POLICY UPDATE CHANGES

Policy states approval is not supported for Elevidys due to serious safety concerns leading to patient deaths.

Notes suspension/pauses of Elevidys shipments to non-ambulatory patients and temporary pause/resume developments in 2025.

The current Elevidys efficacy information is insufficient to determine if the medication demonstrates any clinically meaningful benefits.

Updated experimental, investigational or unproven statement with the addition of 'regardless of U.S. Food and Drug Administration (FDA) approval status. Criteria will be updated as new published data are available.'

Coding entry for HCPCS J1413 for delandistrogene moxeparvovec-rokl per therapeutic dose is listed.

2reported patient deaths linked to acute liver failure

≥4 yrsFDA indication minimum age

<1:400required antibody binding titer

1.33e14 vg/kgrecommended Elevidys dose

Coverage Determination for Elevidys

Not Covered / Experimental

Covered when ALL of the following are met:

Primary coverage stance: Elevidys is considered experimental, investigational, or unproven due to insufficient data establishing safety, efficacy, and improved health outcomes; approval is not supported due to serious safety concerns leading to patient deaths.

Policy will be updated as new published data are available.

Investigational / Insufficient Efficacy

Summary statements from the revision history that affect coverage determinations:

Investigational/Efficacy statement: The current Elevidys efficacy information is insufficient to determine if the medication demonstrates any clinically meaningful benefits.

In the absence of additional clinical trials, there is not enough information to support approval.

Regulatory status clarification: The policy clarifies that Elevidys is considered experimental, investigational, or unproven regardless of U.S. Food and Drug Administration (FDA) approval status; criteria will be updated as new published data are available.

Conditions Not Covered were updated to specifically reference Duchenne Muscular Dystrophy (DMD) under this investigational determination.

Elevidys (delandistrogene moxeparvovec-rokl) is considered experimental, investigational, or unproven for any condition. This investigational designation applies regardless of U.S. Food and Drug Administration (FDA) approval status, and criteria will be updated as new published data become available. The policy states that, due to serious safety concerns leading to patient deaths, approval is not supported for Elevidys.

Specific to Duchenne Muscular Dystrophy (DMD), Elevidys is listed under Conditions Not Covered when the investigational/insufficient-efficacy criteria are not met. Shipment of Elevidys to non-ambulatory patients has been suspended following reported patient deaths due to acute liver failure, and the policy explicitly updates DMD as a condition not covered under these investigational determinations.

The policy rationale states that approval is not supported because of serious safety concerns, including reported patient deaths attributed to acute liver failure, and because available evidence is insufficient to demonstrate clinically meaningful benefits. In the absence of additional clinical trials and published data, the policy finds insufficient evidence of safety and efficacy to support coverage.

The investigational/unproven designation for Elevidys applies regardless of FDA approval status until additional published data are available. The policy will be updated as new evidence emerges; until then, Elevidys remains considered experimental or unproven for any condition.

Coding and Billing Codes

Considered Medically Necessary when criteria met: HCPCS CodesHCPCSCovered

J1413

Injection, delandistrogene moxeparvovec-rokl, per therapeutic dose

Covered HCPCS CodesHCPCSCovered

J1413

Injection, delandistrogene moxeparvovec-rokl, per therapeutic dose

inv-09: anti-AAVrh74 total antibody binding titer — required pre-administration threshold

Required pre-administration antibody titerAnti‑AAVrh74 total antibody binding titer < 1:400

RationalePre-infusion antibody screening required because pre-existing immunity to AAVrh74 can affect safety and efficacy

Source guidancePer Elevidys prescribing information: anti‑AAVrh74 total antibody binding titer should be < 1:400 for administration

inv-10: minimum age for indication — FDA minimum age detail

Minimum FDA-approved agePatients at least 4 years of age (≥ 4 years)

Populations included in label

Provider Requirements and Warnings

Denial Risk

Prior Authorization and Coverage Stance

Prior authorization/coverage is not supported for Elevidys because the policy determined the therapy to be investigational/unproven and there are serious safety concerns, including reported patient deaths related to liver failure.

Coverage stance: Approval is not supported due to safety concerns and insufficient efficacy data.
Investigational/unproven designation may apply regardless of FDA status.

Prior Authorization

Prior Authorization Likely Required

Providers should expect that prior authorization will likely be required and that requests may be denied based on the investigational/unproven determination and documented safety risks.

Clinical Background

Duchenne muscular dystrophy (DMD) is an X-linked progressive disorder caused by mutations in the DMD gene that result in absent or deficient dystrophin, progressive muscle weakness, loss of ambulation, and progressive respiratory and cardiac decline. Elevidys is a gene therapy developed for DMD, but safety concerns—including reported deaths from acute liver failure—have prompted shipment suspensions for certain patient populations and driven the policy’s current investigational stance.

Definitions and Key Drug Information

inv-20: Duchenne muscular dystrophy (DMD) — key definition/value entries for DMD

DefinitionDuchenne muscular dystrophy (DMD): a rare, progressive X-linked disease caused by mutations in the DMD (dystrophin) gene leading to absent or deficient dystrophin and progressive muscle degeneration.

Clinical courseProgressive muscle weakness with loss of ambulation, respiratory impairment, and cardiac decline; first symptoms often by age 2 and diagnosis commonly by ages 3–5.

EpidemiologyIncidence approximately 1 in 5,000 live male births in the U.S.

Treatment goalsManage symptoms, slow disease progression, and delay disability; corticosteroids are a mainstay of therapy.

inv-21: Elevidys dosing — dosing and administration key values (1.33 x 10^14 vg/kg, fixed dose for ≥70 kg, volume guidance)

Policy Summary

PayerCigna

PolicyMuscular Dystrophy - Gene Therapy - Elevidys

Policy CodePolicy IP0571

Change TypeSafety-driven non-approval; investigational language added

Effective Date

Next Review Date

Key ActionSubmit prior authorization and include HCPCS J1413 on claims, but expect denials as approval is not supported given current safety and efficacy concerns.

SourceLink

On This Page

Includes both ambulatory and non-ambulatory patients with confirmed DMD gene mutation

Regulatory noteDMD indication in non-ambulatory patients approved under accelerated approval requiring confirmatory trials

Prior authorization is likely required for Elevidys requests.
Denials may occur due to investigational status and safety concerns.

Denial Risk

Investigational/Unproven Status and Denial Risk

Investigational or unproven policy status may trigger claim denial even if the product has FDA approval; review will consider available published data and policy criteria.

Investigational/unproven designation may result in denial regardless of FDA approval.
Criteria will be updated as new published data are available.

Documentation Required

Coding and Billing Documentation

Use the most appropriate codes when billing and ensure documentation supports the date of service and the specific service rendered. Claims lacking covered codes per the policy will be denied as not covered.

When billing, providers must use the most appropriate codes as of the date of service.
Claims submitted without covered code(s) under the policy will be denied.

Billing Rule

Coding Details

Include HCPCS code J1413 on claims for Elevidys (delandistrogene moxeparvovec-rokl), reported per therapeutic dose, and ensure documentation matches the billed code and service date.

HCPCS: J1413 — Injection, delandistrogene moxeparvovec-rokl, per therapeutic dose

Step Therapy

Step Therapy / Prior Authorization Specifics

No step therapy or specific step/prior authorization program details are provided in the source material; providers should follow general prior authorization processes and monitor for updates.

Step therapy specifics not provided in policy source — none available to reference.
Providers should check payer-specific prior authorization systems for any operational requirements.

Recommended therapeutic dose (weight-based)

1.33 x 10^14 vg/kg of body weight (recommended)

Volume guidance for patients < 70 kgAlternatively administered as 10 mL/kg for patients weighing < 70 kg

Fixed total dose for patients ≥ 70 kgFixed total dose of 9.31 x 10^15 vg for patients ≥ 70 kg

Readministration and periprocedural immunosuppressionReadministration not recommended; begin corticosteroids 1 day prior and continue for minimum 60 days post‑infusion to reduce immune risk

inv-22: Elevidys product name key value — product identification

Proprietary product nameElevidys (delandistrogene moxeparvovec-rokl) intravenous infusion

HCPCS billing codeJ1413 — Injection, delandistrogene moxeparvovec-rokl, per therapeutic dose

Administration routeIntravenous infusion