Rare Disease Report

Vamorolone (VBP 15) Gets Fast Track Designation for Duchenne Muscular Dystrophy

MARCH 24, 2017
James Radke
The FDA has granted Fast Track Designation for vamorolone (VBP15) for the treatment of patients with Duchenne muscular dystrophy to ReveraGen BioPharma Inc.
The VISION-DMD clinical trial program for vamorolone is currently enrolling boys with DMD into clinical trials in US, Canada, Australia, Sweden, UK, and Israel (open label Phase 2a, Phase 2a extension, and Long-term extension studies). Phase 2b trials are expected to initiate enrollment later this year.

Vamorolone (VBP15)

Vamorolone is an oral, once-daily formulation with multiple mechanisms of action that are thought to target multiple aspects of DMD muscle pathology. The orphan drug has the potential to preserve muscle function and prolong ambulation, without some of the known side effects associated with corticosteroids currently in use. 
In 2015, we talked to the founder and CEO of ReveraGen, Eric Hoffman, Ph.D., about vamorolone and why it may be well suited to treat DMD patients.

ReveraGen – the little engine that could

ReveraGen is a privately held, clinical-stage pharmaceutical company with vamorolone in DMD as the lead program.  The vamorolone programs have been carried out in collaboration with non-profit foundations and governments, and with Actelion Pharmaceuticals, through an initial milestone payment related to an option agreement for future vamorolone sales and distribution.
Last fall, we spoke with Dr. Hoffman about the ReveraGen's collaborations. That interview can be viewed here.

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