Zafgen Joins NORD to Support Rare Disease Day(R)

BOSTON, Feb. 27, 2015 (GLOBE NEWSWIRE) -- 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 it joins the National Organization for Rare Disorders (NORD) and others around the world in observing Rare Disease Day on February 28, 2015. On this annual day of observance, patients and families share their stories to help raise awareness of rare diseases as a continued global public health concern.

The theme for this year's Rare Disease Day, "Living with a Rare Disease", speaks to the ongoing challenges that individuals and families face including delay in diagnosis, difficulty finding a medical expert, lack of access to treatment and inability to take advantage of ancillary services.

A rare disease is defined as a condition that affects less than 200,000 patients. According to the latest figures from the National Institute of Health (NIH), rare diseases affect approximately 30 million people in America alone, two-thirds of which are children. There are more than 7,000 known rare diseases, with fewer than 400 FDA-approved treatments.

"We are honored to show our support of NORD, its members and affiliates as we continue to raise awareness of rare diseases, and the importance of seeking better care and treatment options for people living with them every day," said Thomas Hughes, PhD, Chief Executive Officer of Zafgen. "Prader-Willi Syndrome and Hypothalamic Injury Associated Obesity are among the most severe and rare forms of obesity, with no effective treatment options to date. The impact of these chronic and debilitating conditions affects the health and quality of life of patients and their loved ones, and we are dedicated to addressing this unmet need."

For more information about the Rare Disease Day activities in the U.S., go to For more information about global activities, go to For additional information about rare diseases, visit NORD's website,

About Prader-Willi Syndrome

Prader-Willi Syndrome (PWS), the most common known genetic cause of life-threatening obesity, causes constant hunger that drives PWS patients to gain more weight on fewer calories than the average person. As a result, many of those affected become morbidly obese before the age of five. There is currently no cure for this disease. Although the cause is complex, it results from a deletion or loss of function of a cluster of genes on the 15th chromosome. PWS typically causes low muscle mass and function, short stature, incomplete sexual development, and a chronic feeling of hunger that, coupled with a metabolism that utilizes drastically fewer calories than normal, can lead to excessive eating and life-threatening obesity. PWS occurs in males and females equally and in all races, with the same incidence around the world. Prevalence estimates have ranged from 1:8,000 to 1:50,000 with the most likely figure being approximately 1:40,000. To the best of our knowledge, prevalence is about 5,000-7,000 people in the United States needing treatment. You can learn more through the Prader-Willi Syndrome Association website at

About Hypothalamic Injury-Associated Obesity (HIAO)

HIAO is most commonly caused by damage incurred during removal of a central nervous system tumor 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 benign brain tumor that occurs most commonly during childhood and infiltrates near the optic nerve, pituitary gland and the hypothalamus. Treatment of these tumors commonly involves surgical removal of the tumor mass, followed by radiation treatment, which results in disruption or removal of neighboring structures including the hypothalamus. Post-treatment hypothalamic dysfunction results in hyperphagia and 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 have ranged from 0.13 to 0.17 per 100,000 per year, or approximately 400 to 500 new cases per year in the United States and 650 to 850 new cases per year in the European Union.

About Zafgen

Zafgen (Nasdaq:ZFGN) is a biopharmaceutical company dedicated to significantly improving the health and well-being of patients affected by obesity. Beloranib, Zafgen's lead product candidate, is a novel, first-in-class, twice-weekly subcutaneous injection being developed for the treatment of multiple indications, including severe obesity in Prader-Willi Syndrome, hypothalamic injury-associated obesity, including craniopharyngioma-associated obesity, and severe obesity in the general population. Zafgen was founded in 2005 to explore novel approaches to obesity therapeutics, including agents known to inhibit MetAP2 that had been found to drive unprecedented weight loss and metabolic improvements in mice. The company is located in Boston, MA.

CONTACT: Media Relations Shauna Elkin (212) 850-5613 Shauna.Elkin@fticonsulting.comSource:Zafgen, Inc.