ArticleRheumatology (Oxford, England)2025
Vamorolone: a novel metabolism resistant steroid that suppresses joint destruction in chronic polyarthritis with reduced systemic side effects.
Article in Rheumatology (Oxford, England), 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers.
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Who cites it
7 citing papers in PubMed.
- Prednisone, not vamorolone, suppresses novel serum bone and cartilage biomarkers associated with growth failure in children with Duchenne muscular dystrophy.Scientific reports · 2026Trial
- Vamorolone for Duchenne Muscular Dystrophy: A Cross-Trial Efficacy Comparison With Classic Corticosteroids From the FOR-DMD Trial.Neurology · 2026Trial
- Trial
- Results of a phase II open-label, multiple-dose study of vamorolone (VBP15-006) in 7- to < 18-year-old boys with duchenne muscular dystrophy.Journal of neurology · 2026Trial
- 20(R)-Rg3 derived from ginseng fibrous roots ameliorates DSS-induced colitis via selective modulation of glucocorticoid receptor signaling.Naunyn-Schmiedeberg's archives of pharmacology · 2026Article
- Regimen-dependent glucocorticoid effects improve muscle performance without altering CNS physiology in mdx mice.The Journal of physiology · 2026Article
- Post-marketing safety surveillance and signal characterization of the novel dissociative steroid Vamorolone in Duchenne muscular dystrophy: a comparative disproportionality analysis based on FAERS data.Frontiers in pharmacology · 2026Article
Corrections and comments
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Authors and funding
7 authors.
Funding
Abstract
objectivesVamorolone, a dissociated steroidal compound with reduced side effects, offers a promising alternative to traditional glucocorticoids for inflammatory diseases. Unlike conventional glucocorticoids, vamorolone lacks the hydroxyl or ketone groups required for metabolism by 11β-hydroxysteroid dehydrogenase type 1 (11β-HSD1), a key enzyme that modulates glucocorticoid activity. This study investigates vamorolone's resistance to 11β-HSD1 metabolism and assesses its therapeutic efficacy in the murine tumour necros factor-alpha-overexpressing (TNFtg) model of polyarthritis.
methods11β-HSD1 metabolism and action were examined in Hs68 and primary leucocyte culture. Vamorolone 20 mg/kg/day, prednisolone (standard of care) or vehicle were administered by gavage to TNFtg or TNFtg 11β-HSD1 knock-out (TNFtg11BKOKO) animals. Body weight and disease severity were scored daily, and markers of inflammation, joint destruction and side effects assessed at day 56 of age.
resultsVamorolone was entirely resistant to 11β-HSD1 metabolism in vitro. Vamorolone demonstrated comparable anti-inflammatory actions in TNFtg mice, with a comparable reduction in joint inflammation, serum interleukin-6 (IL-6) and synovitis relative to prednisolone. However, vamorolone-treated mice did not experience typical glucocorticoid side effects, including adrenal atrophy, body weight reduction, muscle wasting or inhibition of anabolic bone metabolism. These benefits persisted in 11β-HSD1 knockout mice, indicating that the efficacy of vamorolone is largely independent of 11β-HSD1 metabolism.
conclusionThe findings suggest that at the effective anti-inflammatory dose examined in this study, vamorolone possesses a reduced profile of deleterious systemic effects relative to prednisolone. Whilst highlighting its potential for broader clinical application in inflammatory conditions, it remains unclear whether these side effects would remain mild at markedly higher doses.
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