Santhera Pharmaceuticals and ReveraGen BioPharma, Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted the new drug application (NDA) for vamorolonefor the treatment of Duchenne muscular dystrophy (DMD) for filing. The FDA has set Octoberá26, 2023, as the Prescription Drug User Fee Act (PDUFA) target action date. The PDUFA date is the target date for the FDA to complete its review of the NDA. Furthermore, the FDA stated that it does not currently plan to hold an advisory committee meeting to discuss the application. Subject to approval, Santhera plans to launch vamorolone in the U.S. in Q4-2023. The NDA submission for vamorolone was supported by clinical data from the positive pivotal Phase 2b VISION-DMD study which met the primary endpoint with statistical significance over placebo. The data package also included data from four open-label studies (including extension) in which vamorolone was administered at doses between 2 and 6 mg/kg/day for a total treatment period of up to 30 months. In Europe, a marketing authorization application (MAA), seeking approval for vamorolone in the European Union, has been validated and is under review by the European Medicines Agency (EMA). Vamorolone has been granted Orphan Drug status in the U.S. and in Europe for DMD, and has received Fast Track and Rare Pediatric Disease designations by the U.S. FDA and Promising Innovative Medicine (PIM) status from the
UK MHRA for DMD.