CurrentMass General Brigham Health PlanPolicy N/A

Agamree (vamorolone) prior authorization for Duchenne muscular dystrophy

Prior authorization policy for Agamree (vamorolone) for treatment of Duchenne muscular dystrophy in patients aged ≥2 years; commercial/exchange pharmacy-benefit specialty drug requiring specialty pharmacy fill.

Policy Summary

PayerMass General Brigham Health Plan

PolicyAgamree (vamorolone) prior authorization for Duchenne muscular dystrophy

Policy CodePolicy N/A

Change TypeRemoval of deflazacort step (material change)

Effective DateJul 1, 2025

Next Review DateN/A

Key ActionObtain prior authorization and ensure medication is filled at a contracted specialty pharmacy.

SourceLink

POLICY UPDATE CHANGES

Deflazacort step was removed from the policy.

≥2 yrsminimum patient age for coverage

6 moinitial authorization duration

12 moreauthorization duration

7.5 mL/daydaily quantity limit

specialty onlyspecialty fill requirement

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Coverage Criteria for Agamree (vamorolone)

Initial Authorization

Authorization may be granted when ALL the following criteria are met:

Initial criteria: 1) Diagnosis of Duchenne muscular dystrophy (DMD) confirmed by genetic testing (laboratory confirmation required); 2) Member is 2 years of age or older; 3) One of: (a) clinically significant adverse reaction to prednisone or prednisolone (examples: clinically significant weight gain, Cushingoid appearance, psychiatric/behavioral issues persisting beyond the first 6 weeks of therapy), OR (b) prednisone or prednisolone is clinically inappropriate for the member; 4) Requested medication is prescribed by or in consultation with a specialist experienced in treating DMD.

Document laboratory confirmation of DMD mutation and details supporting adverse reaction or clinical inappropriateness; specialist involvement must be stated.

Continuation of Therapy

Requests for reauthorization will be approved when the following criteria are met:

Reauthorization criteria: 1) Member continues to meet the initial authorization criteria; 2) Documentation of clinical response to vamorolone, demonstrated by motor function tests (e.g., 6 minute walk test [6MWT], time to stand, time to walk/run) or pulmonary function tests.

Initial approvals are granted for 6 months; reauthorizations are granted for 12 months. Provide objective test results to demonstrate response to therapy.

Authorization review exclusions: For members who are new to the plan within the last 90 days and are currently receiving treatment with the requested medication, the authorization review may be performed but exclusions apply when the product was obtained as samples or through a manufacturer's patient assistance program. Providers should document the source of the drug (e.g., pharmacy claim, sample, or assistance program) to support appropriate handling at review.

Product, Quantity Limits, and Coding

Covered pharmacy product and quantity limitNDC | mixedCovered

Agamree oral suspension

Agamree (vamorolone) oral suspension, quantity limit 7.5 mL per day

Coding and Limits

Daily quantity limitAgamree (vamorolone) oral suspension — 7.5 mL per day

Initial approval durationInitial approvals: 6 months

Reauthorization durationReauthorization approvals: 12 months

Provider Actions, Documentation, and Denial Triggers

Prior Authorization

Prior Authorization Required

Prior authorization is required for members under the commercial/exchange pharmacy benefit. The drug is designated specialty and must be filled at a contracted specialty pharmacy.

Applies to Commercial/Exchange pharmacy benefit
Contact (Pharmacy Benefit): Phone: 800-711-4555; Fax: 844-403-1029
Specialty drug — must be dispensed at contracted specialty pharmacy

Step Therapy

Step Therapy Note

Policy previously included a step involving deflazacort; that step was removed during the 06/11/2025 review effective 07/01/2025. No step-therapy requirement remains in this policy.

06/11/2025 P&T review removed deflazacort step; effective 07/01/2025

Definitions

DEFINITIONS

DefinitionDuchenne muscular dystrophy (DMD) confirmed by genetic testing demonstrating a mutation in the DMD gene; laboratory confirmation required

Minimum age for coverageMember must be 2 years of age or older

Prescriber requirementPrescribed by or in consultation with a specialist experienced in treating DMD

Background

Vamorolone (Agamree) is a corticosteroid indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 2 years of age and older. It is presented as an alternative to traditional glucocorticoids (such as prednisone, prednisolone, or deflazacort) for patients who have clinically significant adverse reactions to, or for whom standard corticosteroids are clinically inappropriate. Agamree is provided as an oral suspension and is managed as a specialty pharmacy product under the commercial/exchange pharmacy benefit.

Coverage and authorization decisions for Agamree follow defined criteria including genetic confirmation of DMD, age minimum, documentation of prior adverse reaction or inappropriateness of standard corticosteroids when applicable, specialist involvement, and monitoring of clinical response for reauthorization. Initial approvals are typically limited to 6 months with reauthorizations granted for 12 months, and the policy specifies a daily quantity limit of 7.5 mL per day.