EC grants orphan drug designation to Zafgen's beloranib to treat craniopharyngioma

Zafgen has secured orphan drug designation from the European Commission (EC) for beloranib inhibitor to treat craniopharyngioma, a rare form of benign brain tumor.

In July last year, the EC granted orphan designation for beloranib to treat Prader-Willi syndrome (PWS).

Beloranib is being studied as a first-in-class obesity therapy that shows a mechanism of action via methionine aminopeptidase 2 (MetAP2 ) inhibition.

Zafgen holds exclusive worldwide rights to develop and commercialize beloranib. The company licensed the inhibitor from CKD Pharma.

Zafgen CEO Thomas Hughes said: "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 completed two Phase 2a clinical trials for evaluating safety, efficacy and pharmacokinetics in more patients studied over a longer period of time.

Three consecutive Phase 1b studies with beloranib showed rapid weight loss averaging about one kilogram (2.2 pounds) per week. Body fat was reduced and cardiovascular risk factors and tolerability was enhanced.

Zafgen is developing beloranib to treat various indications such as severe obesity in two rare diseases, PWS and obesity caused by hypothalamic injury, including craniopharyngioma-related obesity; and severe obesity in the general population.

Published 30 September 2015

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