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.
