New Drug Application Is Submitted for Vamorolone for Treating Duchenne Muscular Dystrophy 

The submission of a new drug application (NDA) to the Food and Drug Administration (FDA) has been completed, seeking priority review for vamorolone (Santhera Pharmaceuticals, Prattein, Switzerland; and ReveraGen BioPharma, Rockville, MD) for the treatment of Duchenne muscular dystrophy (DMD). 
 
At the core of the NDA submission are positive data from the pivotal phase 2b VISION-DMD study (NCT02760277) which comprised a 24-week period to demonstrate efficacy and safety of vamorolone (2 mg/kg/day and 6 mg/kg/day) vs prednisone (0.75 mg/kg/day) and placebo, followed by a 24-week period for evaluating the maintenance of efficacy and collecting additional safety and tolerability data. 

In the study, vamorolone met the primary endpoint Time to Stand (TTSTAND) velocity vs placebo (P=0.002) at 24 weeks of treatment and showed a good safety and tolerability profile. 

“Completion of the vamorolone NDA submission is a major step towards our goal of bringing this investigational therapy to patients living with DMD,” said Dario Eklund, chief executive officer, Santhera. “We look forward to working closely with US regulators to advance vamorolone towards approval.”

The most commonly reported adverse events versus placebo from the study were cushingoid features, vomiting, and vitamin D deficiency. Adverse events were generally of mild to moderate severity. 

Vamorolone is an investigational drug candidate with a mode of action based on binding to the same receptor as corticosteroids but modifying its downstream activity. It is considered a dissociative anti-inflammatory drug.