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

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

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January 29, 2013

For immediate release

Leiden, the Netherlands, January 29, 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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About DMD

Duchenne muscular dystrophy (DMD) is a severely debilitating childhood neuromuscular disease that affects 1 in 3,500 live male births. This rare disease is caused by mutations in the dystrophin gene, resulting in the absence or defect of the dystrophin protein. As a result, patients suffer from progressive loss of muscle strength, often rendering them wheelchair-bound before the age of 12. Respiratory and cardiac muscle can also be affected by the disease and most patients die in early adulthood due to respiratory and cardiac failure.

About exon skipping

The dystrophin gene is the largest gene in the body, consisting of 79 exons. Exons are small sequences of genetic code which, via an intermediate step involving RNA, lead to the assembly of sections of protein. In DMD, when certain exons are mutated/deleted, the RNA cannot read past the fault. This prevents the remainder of the exons from being read, resulting in a non-functional dystrophin protein and the severe symptoms of DMD. RNA-based therapeutics, specifically antisense oligonucleotides inducing exon skipping, are currently in development for DMD. These antisense oligonucleotides skip an exon next to a defective exon and thereby correct the reading frame, enabling the production of a novel, functional dystrophin protein. Prosensa's exon skipping technology originated in Leiden University Medical Center.

About Prosensa

Prosensa is an innovative Dutch biopharmaceutical company focused on the discovery, development and commercialization of RNA-modulating therapeutics correcting gene expression in diseases with significant unmet need, in particular neuromuscular disorders. Prosensa's current focus is on developing treatments for Duchenne muscular dystrophy (DMD), myotonic dystrophy and Huntington's disease. In 2009, Prosensa entered into a strategic alliance for part of its DMD exon skipping program with GlaxoSmithKline. Prosensa's lead compound, drisapersen (GSK2402968/ PRO051), being developed by GSK, is in Phase III clinical trials. Prosensa is a privately held biopharmaceutical company, backed by a consortium of Abingworth, Gimv, Idinvest Partners, Life Sciences Partners, MedSciences Capital and New Enterprise Associates. For more information, please visit

Prosensa won the 2012 Emerging Star Award at the European Mediscience Awards and was listed as a Fierce 15 Biotech Company

Press contacts for this press release

    Luc Dochez Chief Business Officer Prosensa

    languages: English, Dutch, French

    Timezone: Europe/Amsterdam

    Phone: +31 (0) 71 33 22 085

    Phone: +31 (0) 71 33 22 100

    E-mail [email protected]

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