Agamree®

Drug - Agamree® (vamorolone) [Catalyst Pharmaceuticals Inc.]

January 2025

Therapeutic area - Duchene Muscular Dystrophy Treatments

Initial approval criteria

Patient must be at least 2 years of age or must meet the age limit in the FDA-approved label AND
Must be prescribed by a provider specializing in neurology AND
Prescriber’s specialty must be provided at time of request AND
Patient must have documentation of a confirmed diagnosis of Duchenne muscular dystrophy (DMD) AND
Patient retains meaningful voluntary motor function (for example, patient is able to speak, manipulate objects using upper extremities, ambulate, and so forth) AND
Patient will not be taking concurrent deflazocort; AND
Prescriber attests that multidisciplinary rehabilitation assessments are conducted every 6-12 months AND
Patient has tried and failed prednisone, having experienced one of the following unacceptable adverse reactions directly attributable to previous therapy with prednisone:
- Patient has manifested significant behavioral changes negatively impacting function at school, day care, etc. OR
- Patient has experienced significant weight gain (for example, crossing two percentiles reaching 98th percentile for age and sex, or both)

Patient retains meaningful voluntary motor function (for example, patient is able to speak, manipulate objects using upper extremities, ambulate, and so forth) AND
Prescriber attests that multidisciplinary rehabilitation assessments are conducted every 6-12 months AND
Patient has received benefit from therapy, which may include one or more of the following:
- Stability or slowing of decline in motor function
- Stability or slowing of decline in respiratory function
- Stability or slowing of decline in sequelae related to diminished strength of stabilizing musculature (for example, scoliosis, etc.)

Daily quantity limit must not exceed those established in the FDA-approved label.
Dosing frequency must reflect those established in the FDA-approved label.
Patient’s most up-to-date weight (in kilograms [kg]) AND requested quantity (in mL) must be submitted at time of request.
If patient is switched from oral corticosteroid treatment (e.g., prednisone or deflazacort), a dosage of prednisone of 0.75 mg/kg/day (or corticosteroid equivalent) corresponds to Agamree 6 mg/kg/day

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