A venture philanthropy success story

  • A venture philanthropy success story

Innovations in steroid chemistry, pharmacodynamic biomarkers, clinical trial design, and business models applied to vamorolone drug development

Clinical Trials

Vamorolone is in clinical testing for Duchenne Muscular Dystrophy (NCT05185622) and Becker Muscular Dystrophy (NCT05166109).
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About vamorolone

Vamorolone is the first therapy for Duchenne muscular dystrophy receiving approval in both EU (EMA) and US (FDA).

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Research

Vamorolone shows efficacy in Duchenne Muscular Dystrophy and safety advantages over prednisone (Guglieri et al. 2022).

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News

Data from international public/private partnership leads to the first therapy for Duchenne muscular dystrophy approved by both EMA and FDA.

Rockville, MD, USA, December 22, 2023
The EMA approved AGAMREE (vamorolone) for Duchenne muscular dystrophy (DMD), ages 4 years and older on 18 December 2023. The US FDA previously approved AGAMREE for DMD (26 October 2023). This makes AGAMREE the first