Prosensa Extends Drisapersen Re-dosing In Europe For Duchenne Muscular Dystrophy

(RTTNews) - Prosensa Holding N.V. (RNA), the biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, has extended its comprehensive program of re-dosing of drisapersen in patients with Duchenne muscular dystrophy or 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 U.S. last week.

The two sites in the PRO051-02/DMD114673 extension trial, 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 demonstrated a mean change from their baseline six-minute walk test of only -25 meters, including two boys with more severe disease who lost ambulation early on in the study.

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.