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news.Dutch biotechnology firm Prosensa Holding has started the submission process for a new drug application (NDA) regulatory filing to the US Food and Drug Administration (FDA) for its lead exon-skipping drug candidate, drisapersen, to treat Duchenne muscular dystrophy (DMD).
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Drisapersen secured FDA fast track status, making the drug eligible for a rolling review of an NDA and was also granted breakthrough therapy designation in June 2013.
Prosensa CEO Hans Schikan said the start 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.
The company expects the rolling submission to be completed before the end of 2014 as well as submit a marketing authorization application for conditional approval with the European Medicines Agency (EMA) in early next year.
Prosensa chief medical officer and senior vice-president R&D Dr Giles Campion said to reach this pivotal point, the company has conducted clinical trials in over 300 patients 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," Campion said.
"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."