Prosensa Receives Rare Disease Company Award from EURORDIS

Leiden, The Netherlands – 27 February 2013 – Prosensa, the Dutch bio­phar­ma­ceu­ti­cal com­pa­ny focus­ing on RNA-mod­u­lat­ing ther­a­peu­tics for rare dis­eases with high unmet need, received the ‘Company Award’ at the EURORDIS Black Pearl Gala Dinner in Brussels last night.
The Black Pearl Gala Dinner is host­ed annu­al­ly by EURORDIS, a non-gov­ern­men­tal alliance of patients and patient orga­ni­za­tions active in the field of rare dis­eases, to cel­e­brate Rare Disease Day, which takes place on the last day of February. Awards rec­og­nize out­stand­ing patients’ advo­ca­cy groups, vol­un­teers, sci­en­tists, com­pa­nies, media, and pol­i­cy mak­ers who have con­tributed to reduc­ing the impact of rare dis­eases on people’s lives. Proceeds from the event are used to sup­port ini­tia­tives to empow­er lead­ers of the rare dis­ease com­mu­ni­ty, reduce iso­la­tion of peo­ple affect­ed by rare dis­eases, stim­u­late cut­ting-edge research and increase pub­lic aware­ness of rare dis­eases.
Yann Le Cam, EURORDIS CEO, com­ment­ed: “We are hon­ored to acknowl­edge the com­mit­ment and achieve­ments of this year’s recip­i­ents of the EURORDIS Awards. Each of this year’s awardees con­tributes in their own unique way toward fos­ter­ing the goals of coop­er­a­tion embod­ied in this year’s Rare Disease Day slo­gan: Rare Disorders with­out Borders.” He added: “The EURORDIS Company Award rec­og­nizes Prosensa’s efforts in devel­op­ing inno­v­a­tive med­i­c­i­nal prod­ucts for rare dis­eases, part­ner­ing with major phar­ma­ceu­ti­cal com­pa­nies and aca­d­e­m­ic cen­ters, pro­mot­ing devel­op­ment and access of orphan drugs in Europe, and last – but not least – engag­ing into a dia­logue with patient advo­ca­cy groups.”
Hans Schikan, Prosensa CEO, com­ment­ed: “This award from EURORDIS is impor­tant to the team at Prosensa. It rec­og­nizes the progress we have made to date and reaf­firms our com­mit­ment to devel­op­ing per­son­al­ized med­i­cines for rare dis­eases for the ben­e­fit of patients.” He added: “We have trans­formed from a biotech start-up to a rare dis­eases bio­phar­ma­ceu­ti­cal com­pa­ny, thanks to our part­ner­ships with patient orga­ni­za­tions, acad­e­mia and phar­ma com­pa­nies.”
Prosensa’s cur­rent port­fo­lio includes six com­pounds for the treat­ment of Duchenne mus­cu­lar dys­tro­phy (DMD), all of which have received orphan drug sta­tus in Europe and the US. These com­pounds use an inno­v­a­tive tech­nique called exon-skip­ping to pro­vide a tru­ly per­son­al­ized med­i­cine approach to treat dif­fer­ent pop­u­la­tions of DMD patients. Prosensa’s lead DMD com­pound, dris­apersen, is in Phase III clin­i­cal tri­als in col­lab­o­ra­tion with GlaxoSmithKline.

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