EMEA grants PROSENSA orphan drug designation for the treatment of Duchenne Muscular Dystrophy using Exon skipping

Leiden, The Netherlands, January 24, 2006 – Prosensa BV, a Dutch biopharmaceutical company focused on RNA-based therapeutics targeting post-transcriptional processes, today announced that orphan drug designation has been awarded to its lead compound for the treatment of Duchenne Muscular Dystrophy (DMD) by the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMEA). Recently, Prosensa received also orphan drug designation from the FDA (USA).

Orphan drug designation is granted by the European Commission to promote the development of products that may offer therapeutic benefits for diseases affecting less than 5 per 10,000 people in the EU. Orphan drug designations are based on several criteria that include rarity and seriousness of the condition, the lack of adequate therapies and scientific merit of the proposed medicinal product. Orphan medicinal product designation provides opportunities for fee reductions, free scientific advice and ten years of market exclusivity following drug approval in Europe.

“Receiving the orphan drug designation from the EMEA and the FDA over the past months has been very encouraging and confirms the validity of our scientific approach for the treatment of DMD. Indeed, the recognition by these major regulatory organisations is exhilarating and strengthens our commitment. The orphan drug designation provides opportunities to stimulate the development of our programme.” said G. Platenburg, CEO of Prosensa BV.

Prosensa’s compounds can correct genetic diseases at the RNA level and are based on the use of short nucleotide chains able to modulate posttranscriptional processes. In isolated cells of DMD patients and a mouse model of DMD, this approach has been demonstrated to restore the production of a functional dystrophin protein. Preparation for a first clinical trial is currently in progress.

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