FDA grants PROSENSA orphan drug designation for the treatment of Duchenne muscular dystrophy using Exon skipping

Leiden, The Netherlands, December 6, 2005 – Prosensa BV, a Dutch biopharmaceutical company focused on RNA-based therapeutics targeting post-transcriptional processes, today announced that orphan drug designation has been granted to its lead compound for the treatment of Duchenne muscular dystrophy (DMD) by the Office of Orphan Products Development (OOPD) of the US Food and Drug Administration (FDA).

Orphan drug designation is granted by the FDA to promote the development of products that may offer therapeutic benefits for diseases affecting less than 200,000 people in the USA. Orphan drug designations are based on several criteria that include rarity and seriousness of the condition, the lack of therapies and scientific merit of the proposed medicinal product. Orphan medicinal product designation provides opportunities for fee and tax reductions before and after marketing authorization and seven years of market exclusivity following drug approval.

“Receiving this orphan drug designation from the FDA has been very encouraging and reinforces our approach to develop a treatment for DMD” said G. Platenburg, CEO of Prosensa B.V.

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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