A new drug to treat Duchenne muscular dystrophy with fewer side effects

2023

Challenge

The anti-inflammatory corticosteroid drugs used as standard treatment for Duchenne muscular dystrophy (DMD) in children act through multiple mechanisms, presenting a risk of significant side effects, including brittle bones, stunted growth, mood changes, and delayed puberty. NIH’s Therapeutics for Rare and Neglected Diseases (TRND) program collaborated with ReveraGen BioPharma to develop VBP15 (vamorolone), a novel corticosteroid that has been modified to retain the beneficial anti-inflammatory and muscle-strengthening effects while decreasing the undesirable side effects that limit the use of traditional corticosteroids. 

Advance

The TRND team conducted rigorous studies to confirm the effectiveness and safety of vamorolone in both cell-based and animal models of DMD. Importantly, these studies showed that this novel compound could maintain many of the positive effects of traditional steroids while avoiding the negative effects. The NIH scientists also designed and executed an improved, more practical process for manufacturing a highly pure version of the drug at the scale necessary to support clinical development and commercialization. 

Impact

TRND’s preclinical studies were vital to jumpstarting development and commercialization of vamorolone, reducing risk to potential investors and helping ReveraGen BioPharma to move the drug forward toward commercialization. As a result of those efforts, vamorolone was approved to treat DMD and brought to market as AGAMREE in the US, Europe, and the UK beginning in 2023. It is now being studied in patients with the related rare disease Becker muscular dystrophy. Moreover, the novel properties of vamorolone suggest that it might work for other inflammatory conditions currently treated with steroids, such as asthma, rheumatoid arthritis, inflammatory bowel disease, and multiple sclerosis.

View All Health Topics