Belinostat gets EU Orphan Drug Designation for the treatment of peripheral T-cell lymphoma

To NASDAQ OMX Copenhagen A/S Announcement no. 18-12 / Copenhagen, October 15, 2012

On October 15, 2012, the European Commission granted Topotarget A/S an Orphan Drug Designation for belinostat, its novel histone deacetylase (HDAC) inhibitor, for the treatment of peripheral T-cell lymphoma (PTCL). PTCL is a rare form of non-Hodgkin’s lymphoma.

“Obtaining Orphan Drug Designation for belinostat in PTCL in the European Union is an important milestone on the path to filing a registration dossier of belinostat in this region. This is positive news for Topotarget and potentially likewise for the rare disease patients with PTCL in need of new treatment options”, said Anders Fink Vadsholt, CEO of Topotarget.

Orphan Drug Designation The EU regulation on orphan medicinal products is intended to encourage the development of drugs that may provide a significant benefit to patients suffering from rare (affecting fewer than five out of 10,000 people) and life-threatening or chronic debilitating conditions for which there is no effective therapies available. The orphan drug designation offers important incentives such as free protocol assistance (to optimize drug development) at the European Medicines Agency, fee reductions for various regulatory activities and following drug approval, and a grant of 10 years’ market exclusivity in the EU.

Belinostat was in September 2009 granted Orphan Drug Designation in the US by the U.S. Food and Drug Administration (FDA) for the treatment of PTCL.

Belinostat has clinical effect in the treatment of PTCL Topotarget has recently announced that the primary efficacy endpoint for the belinostat pivotal BELIEF trial for patients with PTCL has been met in accordance with the Special Protocol Assessment with the FDA which requires the BELIEF trial to reach an objective response rate of at least 20%.

BELIEF is a pivotal, open-label, multi-center, single-arm efficacy and safety trial of i.v. belinostat in patients with relapsed or refractory PTCL. Data from the trial are being further analyzed and are expected to be communicated during Q4 2012. The trial was initiated in December 2008 and recruitment was completed with 129 patients in September 2011.

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