CurrentCentenePolicy CP.PHAR.659

Vamorolone (Agamree) Clinical Policy

Coverage and medical necessity criteria for vamorolone (Agamree) for treatment of Duchenne muscular dystrophy (DMD) in members, including initial and continuation approval requirements and dosing limits.

Policy Summary

PayerCentene

PolicyVamorolone (Agamree) Clinical Policy

Policy CodePolicy CP.PHAR.659

Change TypeNo material changes

Effective Date03.01.24

Next Review Date

Key ActionSubmit documentation (e.g., chart notes, labs) confirming diagnosis, current weight, prior prednisone trial, and dosing within specified limits for prior authorization.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

2 yearsminimum age for FDA-approved indication

6 mg/kg/daystandard maximum DMD dosing

6 monthsinitial approval duration

12 monthscontinued therapy approval duration

Coverage Criteria for Vamorolone (Agamree)

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Initial approval criteria: 1) Diagnosis of Duchenne muscular dystrophy confirmed by genetic testing showing dystrophin deletion or duplication mutation OR if genetic testing is negative, positive muscle biopsy showing absence of dystrophin; 2) Prescribed by or in consultation with a neurologist; 3) Age ≥ 2 years; 4) Documentation of member's current weight in kg; 5) Failure of a ≥ 6 month trial of prednisone unless contraindicated or clinically significant adverse effects are experienced; 6) Dose does not exceed one of the following dosing limits: a) 6 mg/kg per day (up to a maximum of 300 mg per day); b) if mild to moderate hepatic impairment (Child-Pugh A or B): 2 mg/kg per day (up to a maximum of 100 mg per day); c) if co-administered with a strong CYP3A4 inhibitor (e.g., itraconazole): 4 mg/kg per day (up to a maximum of 200 mg per day).see dosing thresholds

Approval duration: 6 months

Continuation Therapy

Covered when ALL of the following are met:

Continuation criteria: 1) Member is currently receiving medication via a Centene benefit or has previously met initial approval criteria (including continuity of care considerations where applicable); 2) Member is responding positively to therapy; 3) Documentation of member's current weight in kg; 4) If the request is for a dose increase, the new dose does not exceed one of the following dosing limits: a) 6 mg/kg per day (up to a maximum of 300 mg per day); b) if co-administered with a strong CYP3A4 inhibitor (e.g., itraconazole): 4 mg/kg per day (up to a maximum of 200 mg per day); c) if mild to moderate hepatic impairment (Child-Pugh A or B): 2 mg/kg per day (up to a maximum of 100 mg per day).see dosing thresholds

Approval duration: Medicaid/HIM 12 months; Commercial 12 months or duration of request, whichever is less

Requests for uses that are not FDA‑approved and that are not explicitly addressed by this policy will not be authorized unless the provider supplies sufficient documentation demonstrating efficacy and safety in accordance with applicable off‑label use policies (see CPC.PA.09 for commercial, HIM.PA.154 for health insurance marketplace, and CP.PMN.53 for Medicaid) or the member’s evidence of coverage documents.

Coverage decisions and the administration of benefits for this policy are subject to the terms, conditions, exclusions, and limitations of the member’s coverage documents (for example, evidence of coverage, certificate of coverage, policy, contract of insurance) and to state and federal requirements. Where regulatory or contractual requirements conflict with this clinical policy, those requirements govern and may affect coverage determinations.

Provider Requirements and Authorization Actions

Prior Authorization

Continuation requirements

Continuation (renewal) requests: Member must meet one of the following: a) member is currently receiving the medication via the Centene benefit or has previously met initial approval criteria; or b) member is currently receiving the medication and is enrolled in a state/product with continuity of care regulations (refer to state-specific addendums). Member must be responding positively to therapy. If a request is for a dose increase, the new dose must not exceed the dosing limits specified in the policy (6 mg/kg/day up to 300 mg/day; if mild to moderate hepatic impairment 2 mg/kg/day up to 100 mg/day; if co-administered with strong CYP3A4 inhibitors 4 mg/kg/day up to 200 mg/day). Approval duration: Medicaid/HIM 12 months; Commercial 12 months or duration of request, whichever is less.

Documentation of member's current weight in kg is required for dosing and approval
If requesting dose increase, submit rationale and documentation demonstrating continued response and safety

Documentation Required

Documentation and coverage terms

Provider must submit clinical documentation (such as office chart notes, laboratory results, and any other relevant clinical information) supporting that the member has met all applicable approval criteria. This includes documentation of diagnosis, prior therapy trials (e.g., ≥6 months of prednisone use unless contraindicated), current weight in kilograms, and evidence of clinical response for continuation requests. Coverage is subject to the terms, conditions, exclusions, and limitations of the member's coverage documents (e.g., evidence of coverage, certificate of coverage, policy, contract of insurance) and applicable state and federal requirements. Non‑FDA indications are not authorized under this policy unless sufficient off‑label documentation of efficacy and safety is provided per applicable off‑label use policies referenced in this document.

Submit office chart notes, labs, and other clinical documentation with the PA request
Coverage decisions are governed by the member's EOC/certificate and state/federal rules
Off-label use requires sufficient documentation per applicable off-label use policies

Background

Vamorolone (Agamree) is a corticosteroid approved for the treatment of Duchenne muscular dystrophy (DMD) in patients aged 2 years and older. Clinical development compared vamorolone with prednisone for motor outcomes and suggested potential advantages for bone safety, though available data are limited and clinical judgement should guide use.

Key Definitions and Contraindications

DMD Dosing Regimens

Typical DMD dosing6 mg/kg/day (up to a maximum of 300 mg/day)

Lower effective dose for some patientsSome patients may respond to 2 mg/kg/day; doses may be titrated down based on tolerability

Hepatic impairment adjustmentIf mild–moderate hepatic impairment (Child‑Pugh A–B): 2 mg/kg/day (up to 100 mg/day)

CYP3A4 inhibitor adjustmentIf co‑administered with strong CYP3A4 inhibitors: 4 mg/kg/day (up to 200 mg/day)

Contraindications

ContraindicationHypersensitivity to vamorolone or any of the inactive ingredients in Agamree

Boxed warningsNone reported

Clinical Policy Definition

PurposeGuide to medical necessity developed from professional standards, literature, guidelines, and expert opinion

ScopeIntended to support coverage and medical necessity decisions for vamorolone (Agamree) in DMD; not a substitute for provider clinical judgment

LimitationsDoes not constitute medical advice or dictate how providers must practice medicine; providers remain responsible for treatment decisions