Prosensa extends the re-dos­ing of dris­apersen in Europe in patients with Duchenne mus­cu­lar dys­tro­phy

Leiden, The Netherlands, Sept. 26, 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 extend­ed its com­pre­hen­sive pro­gram of re-dos­ing of dris­apersen in patients with Duchenne mus­cu­lar dys­tro­phy (DMD) into Europe, with the PRO051-02/DMD114673 sites re-open­ing, begin­ning with Belgium. This fol­lows quick­ly on the re-dos­ing pro­gram that began in the United States (US) last week.
The two sites in the PRO051-02/DMD114673 exten­sion study, the longest run­ning study with a dis­ease mod­i­fy­ing ther­a­py in DMD, had been treat­ing boys with dris­apersen for almost 4 years pri­or to dos­ing being sus­pend­ed in September 2013. Efficacy data from 10 boys ana­lyzed in this study at 177 weeks showed a mean change from their base­line six-minute walk test (6MWT) of only -25 meters, includ­ing two boys with more severe dis­ease who lost ambu­la­tion ear­ly on in the study.
The re-dos­ing pro­gram in Belgium is being led by Dr. Nathalie Goemans, Head of the Neuromuscular Reference Center for Children at the University Hospitals Leuven (UHL) and in Sweden is led by Dr. Mar Tulinius, Professor and Chief Physician at the University of Gothenburg, both key inves­ti­ga­tors in var­i­ous dris­apersen stud­ies.
Dr. Goemans wel­comed the recom­mence­ment of dos­ing, com­ment­ing, “When nat­ur­al his­to­ry of the dis­ease sug­gests that DMD boys lose between 40-60 meters in their 6MWT per year, the results from this long-run­ning study are very encour­ag­ing in terms of walk­ing abil­i­ty and oth­er mea­sures of mus­cle func­tion. Boys treat­ed in our clin­ic are eager to get back onto dris­apersen treat­ment since dos­ing was stopped last year.”
Prosensa is urgent­ly pro­gress­ing prepa­ra­tions to roll out sim­i­lar pro­grams of re-dos­ing in oth­er European coun­tries and glob­al­ly for all pre­vi­ous­ly treat­ed dris­apersen patients, via expand­ed access, com­pas­sion­ate use or named patient pro­grams, where fea­si­ble.
Dr. Giles Campion, Prosensa’s Chief Medical Officer said “Today’s news that Dr. Goemans has been able to resume dos­ing of boys with DMD is anoth­er step for­ward towards our goal of mak­ing dris­apersen avail­able to as many boys as pos­si­ble in a time­ly man­ner. We are pleased that we have been able to accom­plish this soon after re-dos­ing began in the US, and we antic­i­pate Dr. Tulinius’ site to resume dos­ing immi­nent­ly.”
In June, Prosensa announced that the US Food and Drug Administration (FDA) out­lined a reg­u­la­to­ry path for­ward for dris­apersen, under an accel­er­at­ed approval path­way, based upon exist­ing data. “Prosensa remains on track to pur­su­ing reg­u­la­to­ry fil­ings for dris­apersen, ini­tial­ly in the US and Europe, with an FDA sub­mis­sion planned before the end of the year and an EMA sub­mis­sion short­ly there­after” re-con­firmed Hans Schikan, Prosensa’s CEO.

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