Santhera Pharmaceuticals (SIX: SANN) and ReveraGen BioPharma, Inc announce that the U.S. Food and Drug Administration (FDA) has accepted the new drug application (NDA) for vamorolone for the treatment of Duchenne muscular dystrophy (DMD) for filing. The FDA has set October 26, 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.
“We are delighted that the FDA has accepted Santhera’s vamorolone NDA for filing. We believe this product addresses a clear unmet medical need and for Santhera, this represents the achievement of an important milestone with key significance for our future success,” said Dario Eklund, CEO of Santhera. “We look forward to working closely with U.S. regulators to advance vamorolone towards approval.”
“This is an exciting time for the Duchenne community as the data generated across the clinical trial program indicate that vamorolone has the potential to address relevant aspects in patient care that could also enhance treatment outcomes,” said Eric Hoffman, PhD, President and CEO of ReveraGen BioPharma. “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 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.
 Guglieri M et al (2022). JAMA Neurol. Published online August 29, 2022. doi:10.1001/jamaneurol.2022.2480. Link.
 Mah JK et al (2022). JAMA Netw Open. 2022;5(1):e2144178. doi:10.1001/jamanetworkopen.2021.44178. Link.
 Guglieri, et al (2022) JAMA. doi:10.1001/jama.2022.4315
 Heier CR at al (2019). Life Science Alliance DOI: 10.26508
 Liu X, et al (2020). Proc Natl Acad Sci USA 117:24285-24293