Prosensa Achieves Orphan Drug Status on its Entire Duchenne Muscular Dystrophy Portfolio

Leiden, The Netherlands – 29 January 2013 – Prosensa, the Dutch bio­phar­ma­ceu­ti­cal com­pa­ny focus­ing on RNA-mod­u­lat­ing ther­a­peu­tics for rare dis­eases with high unmet need, announced it has achieved orphan drug des­ig­na­tion in the EU and the US for all of its com­pounds for the treat­ment of Duchenne mus­cu­lar dys­tro­phy (DMD).
The European Medicines Agency (EMA) assigned orphan drug sta­tus to Prosensa’s two pre­clin­i­cal com­pounds PRO052 and PRO055 and the US Food and Drug Administration (FDA) grant­ed orphan drug sta­tus to the company’s four com­pounds PRO045, PRO052, PRO053 and PRO055. This brings the total num­ber of prod­ucts in Prosensa’s DMD port­fo­lio with orphan drug sta­tus in the EU and the USA to six. Orphan drug des­ig­na­tion pro­vides reg­u­la­to­ry sup­port in devel­op­ment activ­i­ties such as pro­to­col assis­tance, reduced fees, tax incen­tives and mar­ket exclu­siv­i­ty fol­low­ing drug approval.
Commenting on the news, Prosensa CEO Hans Schikan said: “Having orphan drug des­ig­na­tion for each com­pound in our DMD port­fo­lio is of tremen­dous help in the fur­ther devel­op­ment of our per­son­al­ized med­i­cine can­di­dates. We are com­mit­ted to find­ing effec­tive treat­ments for as many DMD patients as pos­si­ble, includ­ing those affect­ed by very rare muta­tions. The knowl­edge and exper­tise we are gain­ing from our col­lab­o­ra­tion with GlaxoSmithKline on our lead com­pound, dris­apersen, in one of the largest clin­i­cal pro­grams ever under­tak­en in this rare and severe­ly debil­i­tat­ing dis­ease, is indis­pens­able in the accel­er­at­ed devel­op­ment of these addi­tion­al com­pounds. Moreover, thanks to these orphan drug des­ig­na­tions, the sup­port and assis­tance of reg­u­la­to­ry bod­ies both in Europe and the USA will be extreme­ly valu­able in our fur­ther devel­op­ment work.”
Prosensa cur­rent­ly has six exon-skip­ping com­pounds in devel­op­ment for treat­ing DMD, of which dris­apersen, being devel­oped in col­lab­o­ra­tion with GlaxoSmithKline, is in a ful­ly enrolled, late stage Phase III clin­i­cal tri­al of 186 patients, in 47 tri­al sites in 20 coun­tries.

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