Efficacy and safety of vamorolone vs placebo and prednisone among boys with duchenne muscular dystrophy

Sept. 6, 2022
A randomized clinical trial.

For steroidal anti-inflammatory drugs, can efficacy be retained while safety concerns are reduced among boys with Duchenne muscular dystrophy (DMD) with the novel partial receptor agonist vamorolone? 

A randomized, double-blind, placebo- and prednisone-controlled trial of vamorolone (2 dose groups) was carried out in 121 patients with DMD. The trial met the primary (time to stand velocity after 24 weeks for vamorolone, 6 mg/kg per day vs placebo) and first 4 sequential secondary motor function end points; vamorolone showed loss of bone morbidities compared with prednisone, with no stunting of growth and no deleterious changes in bone biomarkers. 

This study found that vamorolone, a dissociative steroidal anti-inflammatory, was able to reduce bone morbidities while retaining efficacy. 

Read the full study in JAMA