CurrentCignaPolicy IP0624

Agamree

Defines medical necessity criteria, documentation requirements, and coverage limitations for Agamree (vamorolone oral suspension) for treatment of Duchenne Muscular Dystrophy (DMD) in patients 6GE; 2 years of age under Cigna plans.

Policy Summary

PayerCigna

PolicyAgamree

Policy CodePolicy IP0624

Change TypeNew and revised (07/15/2024; 10/15/2024; 04/15/2025)

Effective DateApr 15, 2025

Next Review Date

Key ActionApprove for 1 year when medical necessity criteria are met; ensure documentation of genetic confirmation, prior prednisone/prednisolone use and adverse effects, clinical response for continuation, and specialist prescribing/consultation.

POLICY UPDATE CHANGES

New policy created (Summary of Changes = New policy). New, Date = 07/15/2024.

Removed requirement for muscle biopsy showing absence or marked decrease in dystrophin protein for diagnosis confirmation; added requirement that patient has tried prednisone or prednisolone.

Updated adverse effect criterion to specify 'severe behavioral adverse event' requiring dose reduction per prescriber.

Annual Revision dated 04/15/2025 applied; no criteria changes.

1Covered Indication (DMD)

≥2Minimum age for coverage

1 yrApproval duration

Coverage Summary

Coverage stance: covered_with_criteria. Subject: Agamree (vamorolone) for Duchenne Muscular Dystrophy. Scope summary: Defines medical necessity criteria, documentation requirements, and coverage limitations for Agamree (vamorolone oral suspension) for treatment of Duchenne Muscular Dystrophy (DMD) in patients ≥ 2 years of age under Cigna plans. Minimum age threshold: Age ≥ 2 years (policy indicates Agamree is indicated for treatment of DMD in patients ≥ 2 years of age and criteria require patient be ≥ 2 years). Approval duration: Approve for 1 year when medical necessity criteria are met (prior authorization/approval duration text and Medical Necessity section approve for 1 year). Policy reference identifiers: Policy number: IP0624 (Coverage Policy Number).

Key thresholds

Age≥ 2 years

Prior prednisone/prednisolone duration≥ 6 months (documentation required)

Undesirable weight gain threshold≥ 10% body weight increase over a 6-month period (documentation required)

Approval duration1 year

Covered IndicationDuchenne Muscular Dystrophy (DMD)

Initial and Continuation Therapy Criteria

Provider Actions and Authorization

Prior Authorization

Prior authorization / approval duration

Approve for 1 year when medical necessity criteria are met; documentation required to support genetic confirmation, prior prednisone/prednisolone use and adverse effects, clinical response for continuation, and specialist prescribing/consultation.

Approval duration: 1 year
Documentation required for genetic confirmation (confirmed pathogenic dystrophin variant)
Documentation required for prior prednisone/prednisolone use and duration (≥ 6 months when applicable)
Documentation required for intolerable adverse effects or severe behavioral adverse events
Documentation required for clinical response for renewals
Documentation required that medication is prescribed by or in consultation with a DMD/neuromuscular specialist

Applicable Codes and Policy Identifiers

Policy identifiersmixed

IP0624

Coverage Policy Number (Cigna)

Background and Clinical Evidence

Background: Agamree (vamorolone) is a corticosteroid indicated for the treatment of Duchenne Muscular Dystrophy (DMD) in patients ≥ 2 years of age. DMD is an X-linked, progressive muscle-weakening disease due to mutations in the dystrophin gene that results in absent or minimal dystrophin protein and progressive loss of skeletal, respiratory, and cardiac function. Current DMD care considerations identify glucocorticoids and physical therapy as mainstays of treatment, and AAN corticosteroid guidance (2016, reaffirmed 2022) supports use of prednisone to improve strength and pulmonary function; other corticosteroids (e.g., deflazacort/Emflaza) have evidence for motor and pulmonary outcomes. Note: Documentation required may include chart notes, prescription claims records, prescription receipts, and/or other information to support criteria.

Evidence: - FDA Prescribing Information: Agamree oral suspension; June 2024 (prescribing information supports indication and product labeling). - Guidelines: AAN corticosteroid guideline (2016, reaffirmed 2022) and DMD Care Considerations (2018) describe the role of glucocorticoids and supportive care in DMD and provide evidence summaries for prednisone and deflazacort.

Revision History

07/15/2024policy_created

New policy created (Summary of Changes = New policy). Marked material change.

10/15/2024early_annual_revision

Early Annual Revision updated prior therapy and adverse effect criteria: removed muscle biopsy requirement for diagnosis confirmation; added requirement that the patient has tried prednisone or prednisolone (≥ 6 months) and specified significant intolerable adverse effects. Added documentation required statements. Marked material change.

unspecifiedwording_update

Wording of intolerable adverse effects clarified to specify 'severe behavioral adverse event' requiring prednisone/prednisolone dose reduction. Marked material change.

Documentation Required

Required documentation

Document genetic testing showing a confirmed pathogenic dystrophin variant, prior prednisone/prednisolone treatment and duration, specific adverse effects or severe behavioral events (as applicable), and treatment response for renewals.

Genetic testing showing a confirmed pathogenic dystrophin variant
Prior prednisone or prednisolone treatment and duration (e.g., ≥ 6 months when applicable)
Specific intolerable adverse effects: Cushingoid appearance; central (truncal) obesity; undesirable weight gain (≥ 10% body weight increase over 6 months); diabetes and/or hypertension difficult to manage
Severe behavioral adverse event on prednisone/prednisolone that has or would require dose reduction
Documentation of clinical response or continued benefit from Agamree for renewals (examples: motor function, muscle strength, pulmonary function)