Synonyms: Agamree® | VBP-15 | VBP15
vamorolone is an approved drug (FDA & EMA (2023))
Compound class:
Synthetic organic
Comment: Vamorolone (VBP15) is a novel, oral non-hormonal steroid modulator, that was investigated in Duchenne muscular dystrophy. It is a multi-functional drug that exhibits anti-inflammatory, membrane stabilisation, and mineralocorticoid receptor antagonist activities. Vamorolone was proposed as a potential replacement for the corticosteroids that are currently used in DMD, as it appears to cause fewer of the common corticosteroid adverse effects [4].
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References |
1. Heier CR, Damsker JM, Yu Q, Dillingham BC, Huynh T, Van der Meulen JH, Sali A, Miller BK, Phadke A, Scheffer L et al.. (2013)
VBP15, a novel anti-inflammatory and membrane-stabilizer, improves muscular dystrophy without side effects. EMBO Mol Med, 5 (10): 1569-85. [PMID:24014378] |
2. Keam SJ. (2024)
Vamorolone: First Approval. Drugs, 84 (1): 111-117. [PMID:38103149] |
3. Reeves EK, Hoffman EP, Nagaraju K, Damsker JM, McCall JM. (2013)
VBP15: preclinical characterization of a novel anti-inflammatory delta 9,11 steroid. Bioorg Med Chem, 21 (8): 2241-9. [PMID:23498916] |
4. Smith EC, Conklin LS, Hoffman EP, Clemens PR, Mah JK, Finkel RS, Guglieri M, Tulinius M, Nevo Y, Ryan MM et al.. (2020)
Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study. PLoS Med, 17 (9): e1003222. DOI: 10.1371/journal.pmed.1003222 [PMID:32956407] |