The FDA is expected to complete the NDA review in October this year and stated that it currently has no plans to hold an advisory committee meeting to discuss the application.
Subject to approval, Santhera plans to launch the investigational drug candidate vamorolone in the US in the fourth quarter of this year.
ReveraGen BioPharma president and CEO Eric Hoffman said: “This is an exciting time for the Duchenne community as the data generated across the clinical trial programme indicate that vamorolone has the potential to address relevant aspects in patient care that could also enhance treatment outcomes.
“If approved, vamorolone will emerge as an additional treatment option in current standards of care in DMD, with the potential to address unmet medical needs and treat a majority of Duchenne patients starting at an early age.”
The NDA submission for the drug candidate was supported by data obtained from the positive pivotal Phase IIb VISION-DMD clinical trial which met the primary endpoint with statistical significance over placebo.
Results from four open-label studies of vamorolone were also included in the data package.
Vamorolone secured Orphan Drug status in the US and in Europe for DMD.
The US FDA also granted Fast Track and Rare Pediatric Disease designations for the drug which received Promising Innovative Medicine (PIM) status from the UK MHRA for DMD.