Agamree FDA Approval History
Last updated by Judith Stewart, BPharm on Oct 31, 2023.
FDA Approved: Yes (First approved October 26, 2023)
Brand name: Agamree
Generic name: vamorolone
Dosage form: Oral Suspension
Company: Catalyst Pharmaceuticals, Inc.
Treatment for: Duchenne Muscular Dystrophy
Agamree (vamorolone) is a corticosteroid indicated for the treatment of Duchenne muscular dystrophy in patients 2 years of age and older.
- Duchenne muscular dystrophy (DMD) is a rare and life-threatening neuromuscular disorder characterized by progressive muscle dysfunction, ultimately leading to loss of ambulation, respiratory failure, and fatality.
- Agamree contains vamorolone, a novel corticosteroid that acts through the glucocorticoid receptor to exert antiinflammatory and immunosuppressive effects. Vamorolone is thought to have a better-tolerated side effect profile than current standard-of-care corticosteroids.
- FDA approval of Agamree was based on the data from the pivotal Phase 2b VISION-DMD study. Compared with current standard-of-care corticosteroids, Agamree exhibited comparable efficacy, with data suggesting a reduction in adverse events, notably related to bone health, growth trajectory, and behavior.
- Agamree suspension is administered orally, once daily preferably with a meal.
- Warnings and precautions associated with Agamree include alterations in endocrine function, immunosuppression and increased risk of infection, alterations in cardiovascular/renal function, gastrointestinal perforation, behavioral and mood disturbances, decreases in bone mineral density, and ophthalmic effects including cataracts, infections, and glaucoma.
- Common adverse reactions include cushingoid features, psychiatric disorders, vomiting, increased weight, and vitamin D deficiency.
Development timeline for Agamree
Further information
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