Leiden, The Netherlands, Oct. 10, 2014 (GLOBE NEWSWIRE) — Prosensa Holding N.V. (NASDAQ: RNA), the biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, today announced that it has commenced the submission process for a New Drug Application (NDA) regulatory filing to the United States Food and Drug Administration (FDA) for its lead exon-skipping drug candidate, drisapersen, for treating Duchenne muscular dystrophy (DMD). The drug received “Fast Track status” from the FDA, making it eligible for a rolling review of an NDA and was also granted “Breakthrough Therapy Designation” in June 2013.
Hans Schikan, CEO of Prosensa, said: “The commencement of the NDA submission for drisapersen is the culmination of over 12 years of work focused on making treatment options available for DMD patients globally, to improve the lives of boys with this devastating, debilitating childhood neuromuscular disease. We have worked closely with patient groups, scientists, and clinicians throughout the development of our portfolio of six compounds for treating different DMD patient populations, and we pay tribute to everyone in the DMD community, in particular the boys and their families, who have helped us achieve this moment. We expect the rolling submission to be completed before the end of the year, and we are on track to submitting a marketing authorization application for conditional approval with the EMA in early 2015.”
The FDA Safety and Innovation Act (FDASIA) of 2012 provides for an accelerated approval regulatory pathway which is designed to expedite the development and availability for drugs for serious conditions that fill an unmet medical need, based on whether the drug has an effect on a surrogate or an intermediate clinical endpoint which is reasonably likely to predict clinical benefit.
“To reach this pivotal point in Prosensa’s history, over 300 patients have participated in clinical studies of drisapersen at more than 50 trial sites in 25 countries. Just last month we had scientific papers published in the Lancet Neurology, and PLOS ONE, and commenced a drisapersen re-dosing program in both the United States and Europe. This week we are delivering 12 presentations at the World Muscle Society Congress in Berlin. This substantial global body of work underscores our scientific commitment to, and significant investment in, understanding and developing treatments for DMD.” explained Dr. Giles Campion, Prosensa’s Chief Medical Officer and Senior Vice-President R&D.
