Vamorolone
- Generic Name
- Vamorolone
- Brand Names
- Agamree
- Drug Type
- Small Molecule
- Chemical Formula
- C22H28O4
- CAS Number
- 13209-41-1
- Unique Ingredient Identifier
- 8XP29XMB43
- Background
Vamorolone is under investigation in clinical trial NCT03439670 (A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)).
- Indication
Vamorolone is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients ≥2 years of age.
- Associated Conditions
- Duchenne Muscular Dystrophy (DMD)
- Associated Therapies
- -
Breakthrough therapies have given Duchenne muscular dystrophy families like mine hope
Recent FDA approvals of three new therapies for Duchenne muscular dystrophy have increased hope but also confusion among families. The lack of long-term data on gene therapy, especially in older and nonambulatory patients, and the potential for future treatment ineligibility due to antibody creation, adds to the complexity of decision-making. Families need better guidance from neurologists on therapy priorities and polypharmacy, as well as support to make informed decisions without judgment.
Catalyst Pharmaceuticals Reports FDA Approval of AGAMREE® (vamorolone) for Duchenne Muscular Dystrophy Granted to Santhera Pharmaceuticals
Catalyst Pharmaceuticals announced FDA approval for AGAMREE® (vamorolone) to treat Duchenne Muscular Dystrophy (DMD) in patients aged two and older. AGAMREE, a novel corticosteroid with a favorable side effect profile, is expected to launch in Q1 2024. Catalyst holds exclusive North American rights and plans a patient assistance program to ensure access. AGAMREE showed comparable efficacy to standard treatments with fewer adverse effects in trials.
US FDA approves Santhera's drug for rare muscular dystrophy
Catalyst Pharmaceuticals announced FDA approval for Santhera Pharmaceuticals' DMD treatment, Agamree, for patients aged two and older. The drug, similar to corticosteroids but with fewer side effects, is set for U.S. launch in Q1 2024. Catalyst plans a financial assistance program to enhance drug accessibility.
Drug Development Updates
Stay informed with timely notifications on clinical trials, regulatory changes, and research advancements related to this medication.
© Copyright 2025. All Rights Reserved by MedPath