Prosensa begins NDA sub­mis­sion to the FDA for exon-skip­ping drug dris­apersen to treat Duchenne mus­cu­lar dys­tro­phy

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.

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