Most recent update: Tuesday, August 14, 2018 - 09:20

Bariatric News - Cookies & privacy policy

You are here

Beloranib and craniopharyngioma

Beloranib gains European OPD for craniopharyngioma

Credit: e-Magine Art/Flickr
Post-treatment hypothalamic dysfunction results in significant obesity in up to 50% of craniopharyngioma patients

The European Commission (EC) has granted Zafegn orphan drug designation for beloranib for the treatment of craniopharyngioma, a rare form of benign brain tumour. Severe and intractable weight gain is a frequent manifestation of craniopharyngioma, which is the most common cause of hypothalamic injury-associated obesity (HIAO).

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 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 authorisation.

HIAO is most commonly caused by damage incurred during removal of a tumour called craniopharyngioma, but it can also result from less common types of hypothalamic injury such as strokes, brain trauma, or radiation therapy to the brain. Craniopharyngioma is a rare form of histologically benign brain tumour that occurs in both children and adults; its location is near the optic nerves, pituitary gland and the hypothalamus and invasion into these tissues results in significant morbidity. Treatment of these tumours commonly involves surgical removal of the tumour, and sometimes radiation treatment, both resulting in disruption or removal of neighbouring structures including the hypothalamus.

Post-treatment hypothalamic dysfunction results in significant obesity in up to 50% of these patients, resulting in a variety of co-morbid conditions and a deteriorated quality of life. Craniopharyngioma-associated obesity occurs in males and females equally and in all races, with the same incidence around the world. The incidence estimates of craniopharyngioma have ranged from 0.13 to 0.17 per 100,000 per year, or approximately 400 to 500 new cases per year in the US and 650 to 850 new cases per year in the EU.

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 methionine aminopeptidase 2, or 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.

"We are very pleased that our application for orphan drug designation of beloranib has been positively reviewed and granted by the EC," said Dr Thomas Hughes, 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 programme."

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 FDA for PWS in January 2013.

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

Want more stories like this? Subscribe to Bariatric News!

Bariatric News
Keep up to date! Get the latest news in your inbox. NOTE: Bariatric News WILL NOT pass on your details to 3rd parties. However, you may receive ‘marketing emails’ sent by us on behalf of 3rd parties.