CurrentNorth Carolina MedicaidPolicy N/A

Elevidys Coverage and Prior Approval

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This policy governs North Carolina Medicaid outpatient pharmacy prior approval for one-time Elevidys treatment for eligible beneficiaries with Duchenne muscular dystrophy and establishes clinical, laboratory, specialist-prescribing, safety, age, and FDA-label requirements.

Policy Summary

PayerNorth Carolina Medicaid

PolicyElevidys Coverage and Prior Approval

Policy CodePolicy N/A

Change TypeNew prior approval criteria

Effective DateJune 20, 2025

Next Review DateN/A

Key ActionSubmit prior approval for Elevidys, including for beneficiaries under 21 years of age; approval is limited to one treatment.

SourceLink

POLICY UPDATE CHANGES

New prior approval criteria were established for Elevidys coverage.

One timeApproval duration

>4 and <8Required age

<1:400Antibody titer

30 daysPrior therapy interval

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CurrentNorth Carolina MedicaidPolicy N/A

Elevidys Coverage and Prior Approval

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Policy Summary

PayerNorth Carolina Medicaid

PolicyElevidys Coverage and Prior Approval

Policy CodePolicy N/A

Change TypeNew prior approval criteria

Effective DateJune 20, 2025

Next Review DateN/A

Key ActionSubmit prior approval for Elevidys, including for beneficiaries under 21 years of age; approval is limited to one treatment.

SourceLink

POLICY UPDATE CHANGES

New prior approval criteria were established for Elevidys coverage.

One timeApproval duration

>4 and <8Required age

<1:400Antibody titer

30 daysPrior therapy interval

≥3 monthsMonitoring period

Older age/nonambulatoryInvestigational use

Elevidys Coverage Criteria

Initial and one-time therapy

Covered when ALL of the following requirements are met:

Elevidys coverage criteria

Diagnosis and eligibility: The beneficiary has Duchenne muscular dystrophy; is aged greater than 4 years and less than 8 years old; has a confirmed mutation of the DMD gene; and does not have any deletion in exon 8 and/or exon 9 of the DMD gene.Age >4 and <8 years; confirmed DMD mutation; no exon 8 or exon 9 deletion
Prescriber and functional status: The medication is prescribed by or in consultation with a Pediatric Neuromuscular Specialist, and chart documentation indicates that the beneficiary is ambulatory.Ambulatory
Antibody and concomitant therapy requirements: The baseline anti-AAVrh74 total binding antibody titer, measured by ELISA, is less than 1:400; the beneficiary is not receiving concomitant DMD-directed antisense oligonucleotide therapy; and the beneficiary has not received a DMD-directed antisense oligonucleotide within the past 30 days.Anti-AAVrh74 titer <1:400; no antisense oligonucleotide use within 30 days
Examples of DMD-directed antisense oligonucleotides include golodirsen, casimersen, viltolarsen, and eteplirsen.
Safety and monitoring: The beneficiary does not have an active infection, including a clinically important localized infection; has been on a stable corticosteroid dose before therapy unless corticosteroids are contraindicated or not tolerated; will use a corticosteroid regimen before and after infusion; will have troponin-I levels monitored at baseline and subsequently as clinically indicated; and will have liver function and platelet counts assessed before and after therapy for at least 3 months and as indicated, including at minimum GGT and total bilirubin.Liver function and platelet monitoring for at least 3 months
Refer to the package insert for recommended corticosteroid dosing during therapy.
FDA label and timing: The beneficiary must meet the FDA-approved label for use, and the provider must be reasonably certain that Elevidys can be administered before the beneficiary turns 8 years old.FDA-approved use; administration before age 8
Use outside the studied population is considered investigational.

Coverage is excluded when the beneficiary has any deletion in exon 8 and/or exon 9 of the DMD gene.

Coverage is excluded when the beneficiary has an active infection, including a clinically important localized infection.

Coverage is excluded for beneficiaries receiving concomitant DMD-directed antisense oligonucleotide therapy or who received such therapy within the prior 30 days. Examples include golodirsen, casimersen, viltolarsen, and eteplirsen.

Use in a non-ambulatory beneficiary is considered investigational under this policy and is not medically necessary for coverage purposes pending confirmatory clinical-trial evidence.

Use in beneficiaries older than 8 years is considered investigational because the State is not aware of resulting trial evidence for those ages.

Use outside the FDA-approved label or outside the studied population is considered investigational rather than medically necessary under this policy.

Coding and Clinical Parameters

Therapeutic Class Codemixed

Z16	GENE THERAPY AGENTS-PROTEIN DEFICIENCY

Beneficiary age

Age requirementGreater than 4 years and less than 8 years old

Lower age boundaryThe beneficiary must be older than 4 years

Upper age boundaryThe beneficiary must be younger than 8 years; administration must reasonably occur before the beneficiary turns 8

Baseline antibody requirement

Maximum titerLess than 1:400

Antibody typeAnti-AAVrh74 total binding antibody titer

Testing method

Prior Approval and Documentation Requirements

Prior Authorization

Prior Approval Required

Prior approval is required for Elevidys, including for beneficiaries under 21 years of age. Approval is limited to one treatment.

Submit the request through the NC Medicaid prior approval process.
One-time approval only.

Documentation Required

Diagnosis and Clinical Eligibility

Document all clinical eligibility requirements for Elevidys, including the diagnosis, age, specialist involvement, confirmed DMD gene mutation, exon deletion status, and ambulatory status.

Duchenne muscular dystrophy diagnosis.
Beneficiary is aged >4 years and <8 years.
Prescribed by or in consultation with a Pediatric Neuromuscular Specialist.

Policy Background

Elevidys is a gene therapy agent for protein deficiency used in beneficiaries with Duchenne muscular dystrophy. North Carolina Medicaid limits coverage to a narrowly defined population: beneficiaries with a confirmed DMD gene mutation who meet the specified age, ambulatory-status, antibody-testing, specialist-prescribing, corticosteroid, safety-monitoring, and FDA-label requirements. The policy provides for one-time approval and considers use in older-than-studied ages and non-ambulatory beneficiaries investigational.

Definitions

Early and Periodic Screening, Diagnostic, and Treatment (EPSDT)

DefinitionEarly and Periodic Screening, Diagnostic, and Treatment (EPSDT) is a federal Medicaid requirement for beneficiaries under 21 years of age

Coverage relevanceEPSDT requires coverage of medically necessary services, products, or procedures needed to correct or ameliorate a defect, illness, or condition identified through screening

Policy limitationsClinical policy limits may be exceeded or may not apply when provider documentation demonstrates that the requested service meets all EPSDT medical-necessity criteria

ExclusionsEPSDT does not require coverage of services that are unsafe, ineffective, or experimental/investigational

DMD-directed antisense oligonucleotide

Definition

Initial One-Time Therapy

Initial one-time therapy

Elevidys is approved for one-time therapy when all policy criteria are met.

Initial Elevidys therapy: The beneficiary must have Duchenne muscular dystrophy; be aged greater than 4 years and less than 8 years old; have a confirmed DMD gene mutation without deletion in exon 8 and/or exon 9; be ambulatory; have a baseline anti-AAVrh74 total binding antibody titer below 1:400 as measured by ELISA; receive prescribing by or consultation with a Pediatric Neuromuscular Specialist; meet the restrictions on concomitant and recent DMD-directed antisense oligonucleotide therapy; have no active infection; use corticosteroid therapy as required; complete required troponin-I, liver function, and platelet monitoring; and meet the FDA-approved label for use.Age >4 and <8 years; anti-AAVrh74 titer <1:400; no antisense oligonucleotide use within 30 days

The provider must attest that administration can reasonably occur before the beneficiary turns 8 years old. Approval is limited to one treatment.

Quantity and Duration Limits

Elevidys approval

MedicationElevidys

Approval durationOne-time approval for one treatment course

Prior approvalPrior approval is required, including for beneficiaries under 21 years of age

Policy Summary

PayerNorth Carolina Medicaid

PolicyElevidys Coverage and Prior Approval

Policy CodePolicy N/A

Change TypeNew prior approval criteria

Effective DateJune 20, 2025

Next Review DateN/A

Key ActionSubmit prior approval for Elevidys, including for beneficiaries under 21 years of age; approval is limited to one treatment.

SourceLink

On This Page

Prior DMD-directed antisense oligonucleotide therapy

Concomitant therapyThe beneficiary must not be receiving DMD-directed antisense oligonucleotide therapy

Required treatment-free intervalNo DMD-directed antisense oligonucleotide use within the past 30 days

ExamplesGolodirsen, casimersen, viltolarsen, and eteplirsen

Post-therapy monitoring

Required assessmentsLiver function and platelet counts must be assessed before and after therapy

Monitoring durationMonitoring is required for at least 3 months following therapy

Minimum liver testsLiver monitoring must include, at minimum, GGT and total bilirubin

Confirmed mutation of the DMD gene.

No deletion in exon 8 and/or exon 9 of the DMD gene.

Chart documentation confirms the beneficiary is ambulatory.

Documentation Required

Baseline Antibody Testing

Submit baseline anti-AAVrh74 total binding antibody testing measured by ELISA. The result must be less than 1:400.

Document the assay method: ELISA.
Required result: anti-AAVrh74 total binding antibody titer <1:400.

Documentation Required

Antisense Oligonucleotide Therapy History

Document that the beneficiary is not receiving concomitant DMD-directed antisense oligonucleotide therapy and has not received one within the prior 30 days.

Applicable therapies include golodirsen, casimersen, viltolarsen, and eteplirsen.
No DMD-directed antisense oligonucleotide therapy within the past 30 days.

Documentation Required

Safety and Corticosteroid Documentation

Document the absence of active infection and the required corticosteroid history and treatment plan.

No active infection, including clinically important localized infection.
Beneficiary has been on a stable corticosteroid dose before therapy, unless contraindicated or intolerant.
Corticosteroid regimen will be used before and after infusion; follow the package insert for recommended dosing.

Documentation Required

Monitoring and Administration Attestation

Attest to the required pre- and post-therapy monitoring and administration plan, including cardiac, liver function, and platelet assessments.

Troponin-I will be monitored at baseline and subsequently as clinically indicated.
Liver function and platelet count will be assessed before and after therapy for at least 3 months and as clinically indicated.
Post-therapy liver assessment must include, at minimum, GGT and total bilirubin.
Provider attests that Elevidys can reasonably be administered before the beneficiary turns 8 years old.

Prior Authorization

EPSDT Does Not Waive Prior Approval

Being under 21 years of age does not eliminate the prior approval requirement when the service, product, or procedure requires prior approval.

EPSDT does not substitute for or waive NC Medicaid prior approval.
Submit prior approval documentation for beneficiaries under 21.

Denial Risk

Clinical Eligibility Not Met

Coverage may be denied when core clinical eligibility requirements are not met or cannot be documented.

DMD diagnosis is absent or unconfirmed.
Beneficiary is not aged >4 years and <8 years.
Confirmed DMD gene mutation is absent.
Deletion in exon 8 and/or exon 9 is present.
Required clinical eligibility documentation is incomplete.

Denial Risk

Specialist, Ambulatory, or Concomitant Therapy Requirement Not Met

Coverage may be denied when required specialist involvement, ambulatory status, or antisense oligonucleotide therapy restrictions are not satisfied.

No prescription by or consultation with a Pediatric Neuromuscular Specialist.
Beneficiary is non-ambulatory.
Beneficiary is receiving concomitant DMD-directed antisense oligonucleotide therapy.
Beneficiary received DMD-directed antisense oligonucleotide therapy within the prior 30 days.

Denial Risk

Safety or Monitoring Requirements Not Met

Coverage may be denied when safety or monitoring requirements are not met or cannot be documented.

Active infection is present.
Required corticosteroid use is contraindicated, not tolerated, or cannot be administered as required.
Baseline or follow-up troponin-I monitoring is not planned.
Required liver function and platelet assessments are not planned or documented.

Denial Risk

Investigational Use

Use in beneficiaries older than 8 years or in non-ambulatory beneficiaries is considered investigational under this policy because the State considers these populations insufficiently studied or subject to unresolved confirmatory evidence.

The provider must attest that Elevidys can reasonably be administered before the beneficiary turns 8 years old.
Use in older ages is considered investigational.
Use in non-ambulatory beneficiaries is considered investigational until confirmatory trials establish clinical benefit.
The ENVISION trial (NCT05881408) is expected to be completed in January 2027.

A DMD-directed antisense oligonucleotide is a therapy directed at Duchenne muscular dystrophy

ExamplesGolodirsen and casimersen

Additional examplesViltolarsen and eteplirsen