Zafgen Granted Orphan Drug Designation in the European Union for Beloranib, a First-in-Class MetAP2 Inhibitor for the Treatment of Craniopharyngioma

Zafgen, Inc.

, a biopharmaceutical company dedicated to significantly improving the health and well-being of patients affected by obesity and complex metabolic disorders, today announced that the European Commission (EC) has granted orphan drug designation for beloranib for the treatment of craniopharyngioma, a rare form of benign brain tumor. Severe and intractable weight gain is a frequent manifestation of craniopharyngioma, which is the most common cause of hypothalamic injury-associated obesity, or HIAO. "We are very pleased that our application for orphan drug designation of beloranib has been positively reviewed and granted by the EC," said Thomas Hughes, Ph.D., Chief Executive Officer of Zafgen. "We believe beloranib represents an important and promising new approach for the treatment of HIAO, and has the potential to meaningfully improve the lives of patients with this debilitating disorder. We remain dedicated to advancing beloranib for the treatment of severe and complicated forms of obesity and look forward to further exploring its potential as a safe and effective treatment option for HIAO as a component of our clinical development program." Zafgen previously received orphan designation by the European Commission for beloranib for the treatment of Prader-Willi syndrome (PWS) in July 2014, and by the U.S Food and Drug Administration for PWS in January 2013. Orphan Drug Designation by the European Commission provides regulatory and financial incentives for companies to develop and market therapies that treat a life-threatening or chronically debilitating condition affecting no more than five in 10,000 persons in the European Union (EU), and where no satisfactory treatment is available. In addition to a 10-year period of marketing exclusivity in the EU after product approval, Orphan Drug Designation provides incentives for companies seeking protocol assistance from the EMA during the product development phase, and direct access to centralized marketing authorization.