Prosensa Holding N.V. (RNA) announced that it has initiated the process of rolling submission of a new drug application (NDA) to the FDA for its lead candidate, drisapersen, for the treatment of Duchenne muscular dystrophy (DMD).
Drisapersen has Breakthrough Therapy Designation in the U.S. Moreover, the fast track status for drisapersen has made Prosensa eligible for making a rolling NDA submission for the candidate.
According to Prosensa, DMD is a severely debilitating childhood neuromuscular disease affecting up to 1 infant in 3,500 live male births. This disease can also affect the respiratory and cardiac muscles, causing most patients to die in their early adulthood. No therapy has yet been approved in this indication.
Currently, Prosensa has six DMD candidates in its pipeline, drisapersen being the most advanced one. Notably, all these candidates have orphan drug status in the U.S. and the EU.
We note that Prosensa had previously completed a randomized, double-blind, placebo-controlled phase III study (results announced in Sep 2013) on drisapersen for DMD. However, the candidate had failed to meet the primary endpoint in this study. The company started re-dosing patients in Sep 2014.
Meanwhile, Prosensa is making the rolling submission of the NDA for drisapersen based on existing data according to a regulatory path outlined by the FDA under an accelerated approval process. The submission should be completed by year end. The company also intends to submit a marketing authorization application to the European Medicines Agency for conditional approval of drisapersen in early 2015.
We believe the initiation of the NDA submission for drisapersen is an important milestone for Prosensa. The company’s shares gained 2.1% following the announcement.
Sarepta Therapeutics, Inc. (SRPT) is also developing eteplirsen for the treatment of DMD. The company intends to file for its approval in the U.S. by year-end.
Prosensa currently carries a Zacks Rank #1 (Strong Buy). Other well-placed stocks in the biotech sector include Medivation, Inc. (MDVN) and Ligand Pharmaceuticals Inc. (LGND). Both carry a Zacks Rank #1 (Strong Buy).
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