JAMA Neurology publishes positive pivotal clinical trial , VISION-DMD, with vamorolone in Duchenne muscular dystrophy

Vamorolone met its primary endpoint by demonstrating statistically significant and clinically relevant improvement in time to stand from floor compared to placebo, the first functional milestone to deteriorate in young children with DMD. Consistent results across multiple secondary endpoints support the results of the primary endpoint. The relative efficacy of vamorolone 6 mg/kg/day was comparable to that seen with prednisone 0.75 mg/kg/day across primary and secondary efficacy endpoints.

Over the 24-week treatment period, no negative impact on biomarkers of bone health and no loss of linear growth were observed with vamorolone. Vamorolone was generally safe and well tolerated. The most commonly reported adverse events versus placebo study were cushingoid features, vomiting and vitamin D deficiency. Adverse events were generally of mild to moderate severity.

"Corticosteroids remain a cornerstone of treatment for many children with Duchenne muscular dystrophy and data published on the VISION-DMD study are an important advancement in the development of additional treatment options for these patients,” said Michela Guglieri, MD, Senior Lecturer and Consultant Neurologist, Newcastle University.

See- "Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy;A Randomized Clinical Trial"-Michela Guglieri, MD; Paula R. Clemens, MD; Seth J. Perlman, MD; et al. JAMA Neurol. Published online August 29, 2022. doi:10.1001/jamaneurol.2022.2480.

A rolling submission to the FDA has been proceeding according to plan with the clinical module submitted on June 28, 2022.