Zafgen Says EC Grants Orphan Drug Designation For Beloranib To Treat PWS

(RTTNews) - Zafgen Inc. (ZFGN), a biopharmaceutical company dedicated to significantly improving the health and well-being of patients affected by obesity, announced that the European Commission or EC has granted orphan drug designation for beloranib for the treatment of Prader-Willi syndrome or PWS, a rare disease.

"........We are dedicated to the advancement of beloranib for the treatment of PWS and other severe forms of obesity and we look forward to initiating our Phase 3 clinical program in PWS later this year," said Thomas Hughes, Ph.D., Chief Executive Officer of Zafgen.

In January 2013, the U.S. Food and Drug Administration granted Zafgen orphan designation to treat PWS with beloranib. Initial results from its Phase 2 study of beloranib in patients with PWS demonstrated improvements in hunger-related behaviors and body composition, including reductions in body fat content and preserved lean body mass.

Beloranib is a novel, first-in-class injectable small molecule therapy with a unique mechanism of action that reduces hunger while stimulating the use of stored fat as an energy source. Beloranib is a potent inhibitor of MetAP2, an enzyme that modulates the activity of key cellular processes that control metabolism. MetAP2 inhibitors work, at least in part, by directing MetAP2 binding to cellular stress mediators, and, thus, reducing the tone of signals that drive lipid synthesis by the liver and fat storage throughout the body. In this manner, MetAP2 inhibition increases metabolism of fat as an energy source.

Zafgen holds exclusive worldwide rights (exclusive of South Korea) for development and commercialization of beloranib. Zafgen exclusively licensed beloranib from Chong Kun Dang (CKD) Pharmaceutical Corp. of South Korea.