ReveraGen has announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for vamorolone (VBP15) for the treatment of patients with Duchenne muscular dystrophy.

Action Duchenne supported the Pharmacodynamics BioMarker Panel for the Phase IIa trial and we are delighted to see this potential treatment developed further. Both projects have been supported financially by three UK Duchenne foundations – Joining Jack, Duchenne Children’s Trust and the Duchenne Research Fund.

Eric Hoffman said: “In summary, the support from Action Duchenne has been critical in moving the biomarker programme forward in the vamorolone (VBP15) clinical program in DMD.  With Action Duchenne help, we believe the integration of biomarkers is among the most robust and state-of-the-art of any pediatric drug development programme.”

This designation can speed the review of efficacy and safety data for vamorolone in boys living with Duchenne, potentially leading to more rapid regulatory approval. Vamorolone is under parallel guidance from the FDA and the European Medicines Agency (EMA). By granting this designation, FDA acknowledges that the vamorolone program is directed towards development of a potential treatment for a serious condition, and addresses an unmet medical need.

The VISION-DMD clinical trial program for vamorolone is currently enrolling boys living with Duchenne, into clinical trials in US, Canada, Australia, Sweden, UK, and Israel (open label Phase 2a, Phase 2a extension, and Long-term extension studies). Blinded, placebo- and glucocorticoid-controlled Phase 2b trials are expected to initiate enrollment later this year.

“We are excited about this new development in our discussions with FDA”, said Eric Hoffman, Ph.D., CEO of ReveraGen. “We hope that our innovative clinical program in DMD, with extensive use of pharmacodynamic biomarkers, will lead to a rapid read-out of drug effect.”

About vamorolone

Vamorolone is an oral, once-daily formulation with multiple mechanisms of action that are thought to target multiple aspects of DMD muscle pathology. It is a potent glucocorticoid receptor agonist that shows strong anti-inflammatory activity, but without many of the safety concerns seen with other steroidal immune modulators.

Vamorolone is also a mineralocorticoid receptor antagonist, and has been shown to aid dystrophic heart function in mouse models of DMD.

Finally, vamorolone stabilizes plasma membranes, and may counteract the membrane instability caused by dystrophin deficiency in DMD. The vamorolone DMD clinical program is supported by the National Institutes of Health (NIAMS, NINDS) and the European Community Horizons 2020 program.