AGAMREE (vamorolone)

AGAMREE (vamorolone)

Self-Administration – oral

Diagnosis considered for coverage:

• DMD: Indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 2 years of age and older

Coverage Criteria:

For diagnosis of Duchenne muscular dystrophy (DMD):

• Diagnosis of Duchenne muscular dystrophy (DMD), AND
• Patient is 2 years of age or older, AND
• Patient has received genetic testing for a mutation of the dystrophin gene, AND
• One of the following:
  • Patient has a confirmed mutation of the dystrophin gene, OR
  • Muscle biopsy confirmed an absence of dystrophin protein, AND
• Patient has had a trial and failure or intolerance to prednisone or prednisolone given at a dose of 0.75 mg/kg/day or 10 mg/kg/weekend, AND
• Prescribed by or in consultation with a neurologist who has experience treating children, AND
• One of the following:
  • For patients less than or equal to 50kg, dose will not exceed 6mg/kg of body weight once daily, OR
  • For patients greater than 50kg, dose will not exceed 300mg/day

Reauthorization Criteria:

For diagnosis of Duchenne muscular dystrophy (DMD):

• Patient has experienced a benefit from therapy (e.g., improvement in preservation of muscle strength), AND
• One of the following:
  • For patients less than or equal to 50kg, dose will not exceed 6mg/kg of body weight once daily, OR
  • For patients greater than 50kg, dose will not exceed 300mg/day, AND
• Patient has had a trial and failure or intolerance to prednisone or prednisolone given at a dose of 0.75 mg/kg/day or 10 mg/kg/weekend

Coverage Duration:

• Initial: 1 year
• Reauthorization: 1 year

Dosing:

For diagnosis of Duchenne muscular dystrophy (DMD):

• Recommended dose: 6 mg/kg taken orally once daily
• Maximum dose: 300 mg daily for patients weighing more than 50 kg
• Some patients may respond to a dose of 2 mg/kg daily. Doses may be titrated down to 2 mg/kg/day as needed, based on individual tolerability

Authorization is not covered for the following:

The use of this drug for indications not listed in this policy does not meet the coverage criteria established by the Western Health Advantage (WHA) Pharmacy and Therapeutics (P&T) Committee.

Additional Information:

• Approximately 70% of individuals with DMD have a single-exon or multi-exon deletion or duplication in the dystrophin gene, dystrophin gene deletion, and duplication testing is usually the first confirmatory test. If genetic testing does not confirm a clinical diagnosis of DMD, then a muscle biopsy sample should be tested for the presence of dystrophin protein by immunohistochemistry of tissue cryosections or by western blot of a muscle protein extract.
• Prednisone 0.75 mg/kg/d should be considered the optimal prednisone dose in DMD. Over 12 months, prednisone 10 mg/kg/weekend is equally effective, although long term outcomes of this alternative regimens are unknown.

Policy Updates:

• 9/1/2024 (policy effective date) – New policy approved by WHA P&T Committee. (P&T, 8/20/2024) (P&T meeting date) 

References:

1. Agamree Prescribing Information. Catalyst Pharmaceuticals, Inc. Coral Gables, FL. October 2023
2. Birnkrant DJ, Bushby K, Bann CM, et al. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management. The Lancet Neurology. 2018;17(3):251-267.
3. Gloss D, Moxley RT 3rd, Ashwal S, Oskoui M. Practice guideline update summary: Corticosteroid treatment of Duchenne muscular dystrophy: Report of the Guideline Development Subcommittee of the American Academy of Neurology. Neurology. 2016;86(5):465-72.