Authored by James Sheppard
The Dutch biotechnology company Prosensa has announced that it has begun the submission process for its Duchenne Muscular Dystrophy (DMD) drug Drisapersen with the FDA. Prosensa has hoped that it will fully file by the year end.
Drisapersen, which was granted entry into the FDA’s fast track program in June 2013, is an exon skipping drug designed to treat patients with the mutation in exon 51. Although the initial phase III analysis of the drug was not promising Prosensa came back with renewed analysis that showed administering the drug early in the disease and lengthening treatment could delay progression of DMD.
Dr. Giles Campion, Prosensa’s Chief Medical Officer said ‘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.’
After a series of positive discussions with the FDA Prosensa was back on track to submit its regulatory plan to the FDA with a final decision on the treatment expected in mid-2015. This combined with a strengthened management team show that Prosensa is on the right track for growth in 2015.