Document Type

Article

Publication Date

3-3-2026

Keywords

Vamorolone, Duchenne muscular dystrophy, Expanded access protocol study, Corticosteroids, Glucocorticoids

Abstract

Background: Vamorolone is a dissociative glucocorticoid receptor agonist for treating Duchenne muscular dystrophy (DMD). The VBP15-006 study assessed vamorolone safety, tolerability, and pharmacokinetics in 2- to < 4 and 7- to < 18-year-old boys with DMD. Results for 7- to < 18-year-old boys, either corticosteroid (CS)-untreated or switching from prior CS treatment, are reported here. Exploratory objectives included efficacy and pharmacodynamic biomarkers related to safety. Methods: In this phase II, open-label, multiple-dose study, participants received 2 or 6 mg/kg/day vamorolone for 12 weeks, followed by treatment in an expanded access protocol (EAP Canada). Results: 34 participants (12 CS-untreated, 22 CS-treated) aged 7- to < 18 years were enrolled. Most treatment-emergent adverse events were mild. All 34 participants completed VBP15-006 and entered EAP Canada. During EAP Canada, CS-untreated participants maintained stable linear growth, while CS-treated participants exhibited catch-up growth consistent with serum bone biomarkers (median total vamorolone exposure 1.3 and 1.7 years, respectively). Some weight gain occurred, especially in CS-untreated participants receiving 6 mg/kg/day. Dose-dependent adrenal suppression occurred in CS-untreated and CS-treated participants; 2 individuals who switched from deflazacort to vamorolone 2 mg/kg/day had generalized weakness consistent with adrenal insufficiency. Vamorolone showed dose-dependent pharmacokinetics, rapid clearance, and no accumulation. There were no relevant efficacy changes in CS-treated and CS-untreated groups at either dose. Conclusions: Vamorolone demonstrated a consistent safety profile in 7- to < 18-year-old boys with DMD. Switching to 6 mg/kg/day vamorolone appeared to mitigate adrenal insufficiency risk. There was no negative effect on growth, and catch-up growth occurred in previously CS-treated individuals switching to vamorolone. Trial registration: ClinicalTrials.gov: NCT05185622, NCT03863119. First submitted 09-11-2021.

Comments

https://doi.org/10.1007/s00415-026-13711-6

Publisher Attribution

This article is published in Journal of Neurology. This article is licensed under a Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License, which permits any non-commercial use, sharing, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if you modified the licensed material. You do not have permission under this licence to share adapted material derived from this article or parts of it. The images or other third party material in this article are included in the article’s Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by-nc-nd/4.0/.

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