Prosensa Begins NDA Filing For DMD Drug Lead

Prosensa, a biotech firm focused on RNA-modulating therapeutics for genetic disorders, announced that it has kickstarted the submission process for a New Drug Application (NDA) for its lead drug drisapersen as treatment for Duchenne muscular dystrophy (DMD).

Drisapersen causes exon 51 skipping in the dystrophin gene and is targeted to treat up to around 13 percent of all DMD patients. Antisense oligoneucleotides that skip an exon to correct the fault in RNA processing allows for the production of a new, generally functional dystrophin protein. The drug has been designated orphan status in the U.S., EU, and Japan. Prosensa has also received Breakthrough Therapy designation and Fast Track status from the U.S. Food and Drug Administration (FDA), making it eligible for a rolling review once the company’s NDA is accepted.

Duchenne muscular dystrophy is a rare, severe, debilitating childhood disease that afflicts up to 1 in 3,500 male babies. DMD is caused by mutation in the dystrophin gene and results in the absence or defect of the dystrophin protein. Affected patients suffer from progressive loss of muscle strength as well as the ability to walk. DMD also causes respiratory and cardiac failure especially in the early adulthood of patients.

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 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."

Dr. Giles Campion, Prosensa's CMO and SVP of R&D, shared that more than 300 patients took part in clinical trials of the drug in over 50 sites in 25 countries. He adds, “Just last month we had scientific papers published in the Lancet Neurology and PLOS ONE… This week we are delivering 12 presentations at the World Muscle Society Congress in Berlin.”

The company has also begun a drisapersen re-dosing program for an extension trial in the EU, beginning with Belgium. A similar re-dosing program in the U.S. is also ongoing since last month.