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Agamree (VAMOROLONE)
Agamree works by inhibiting the activity of pro-inflammatory enzymes, reducing muscle damage and inflammation in Duchenne muscular dystrophy.
Agamree (vamorolone) is a small molecule developed by Catalyst Pharms, approved by the FDA in 2023 for the treatment of Duchenne muscular dystrophy (DMD). It is a novel anti-inflammatory agent that works by modulating the immune system to reduce muscle damage. Agamree is a patented medication with no generic manufacturers available. Its short half-life of 2 hours requires frequent dosing. Key safety considerations include its potential effects on the immune system.
At a glance
| Generic name | VAMOROLONE |
|---|---|
| Sponsor | Catalyst Pharms |
| Modality | Small molecule |
| Therapeutic area | Immunology |
| Phase | FDA-approved |
| First approval | 2023 |
Mechanism of action
Vamorolone is a corticosteroid that acts through the glucocorticoid receptor to exert anti-inflammatory and immunosuppressive effects. The precise mechanism by which vamorolone exerts its effect in patients with DMD is unknown.
Approved indications
- Duchenne muscular dystrophy (DMD)
Common side effects
- Cushingoid Features
- Psychiatric disorders
- Vomiting
- Weight increased
- Vitamin D deficiency
- Cough
- Headache
- Diarrhea
- Increased appetite
- Rhinitis
- Abnormal behavior
- Aggression
Drug interactions
- Itraconazole
Key clinical trials
- Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMIT
- Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy
- A Study on Safety and Effectiveness of Long-term Treatment With Vamorolone in Boys With Duchenne Muscular Dystrophy (PHASE4)
- Evaluation of Vamorolone CYP3A4 Induction on Midazolam (a Sensitive CYP 3A4 Substrate) Pharmacokinetics (PHASE1)
- A Study to Assess Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD) (PHASE2)
- A Study to Assess Vamorolone in Becker Muscular Dystrophy (BMD) (PHASE2)
- Evaluation of Vamorolone Mineralocorticoid Receptor Antagonism in Healthy Subjects (PHASE1)
- A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) (PHASE2)
Patents
| Patent | Expiry | Type |
|---|---|---|
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
| FDA Orange Book | Patents + exclusivity |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Agamree CI brief — competitive landscape report
- Agamree updates RSS · CI watch RSS
- Catalyst Pharms portfolio CI