The investigational drug vamorolone (formerly VBP15), under development by ReveraGen BioPharma, has received U.S. Food and Drug Administration (FDA) Fast Track designation for the treatment of Duchenne muscular dystrophy (DMD). This designation can speed the review of efficacy and safety data for vamorolone in boys with DMD, potentially leading to more rapid regulatory approval.

Vamorolone, a “dissociative steroid,” is an anti-inflammatory compound that researchers hope will convey the same benefits of traditional glucocorticoids, such as prednisone and deflazacort (brand name Emflaza), without the unwanted side effects — including stunted growth, insulin resistance and weight gain — of those drugs. In clinical trials, glucocorticoid steroids have been shown to benefit DMD patients, but many find the side effects to be burdensome or intolerable.

VISION-DMD, a phase 2 clinical trial, currently is under way to test the safety and effectiveness of vamorolone in DMD. In the study, which follows the successful completion of a phase 1 trial to test the drug’s safety and tolerability in healthy volunteers, study investigators also will assess whether the drug has positive effects on muscle strength and timed function tests. (To learn more or to inquire about participation, contact Andrea Smith at smithal7@upmc.edu.)

Two MDA Venture Philanthropy (MVP) grants totaling $1.9 million to ReveraGen supported preclinical development and validation of the drug, and another MVP grant for $1,015,200 supported the phase 1 clinical trial to evaluate safety and tolerability in healthy adult volunteers. MVP is the drug development arm that operates within MDA’s Translational Research Program.

Additionally, through its basic research program, MDA is supporting development of biomarkers for glucocorticoids including vamorolone with a grant to Yetrib Hathout for $300,000, and research by Eric Hoffman to understand the effects of glucocorticoids including vamorolone on muscle repair with a grant for $321,659.