Zafgen Obesity Drug Positive Results Could be the key to Restart Beloranib FDA Review

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A study conducted by Zafgen, a biopharmaceutical company focusing on obesity and complex metabolic disorders, on the safety and efficacy of the beloranib drug yielded positive results. This could pave the way for a reconsideration on the company's regulatory approval application with the FDA.

Beloranib is a drug developed by Zafgen for patients with Prader-Willi syndrome (PWS), a rare genetic condition linked to overeating and obesity.

The approval application of the drug was put on hold by the FDA in 2015 after two patients died in a trial involving beloranib, according to a report from Bloomberg.The patients suffered respiratory failure due to blood clots in the lungs, although it was not confirmed whether they really died because of the drug, says one medical officer.

"This clear efficacy outcome is a crucial first step in moving discussions forward with the Food and Drug Administration regarding continued development of beloranib," stated Thomas Hughes, Ph.D., Chief Executive Officer of Zafgen.

"While we take the previously reported adverse events very seriously, we now have the robust data to provide greater perspective on the benefit/risk relationship of beloranib in this high-risk patient population. We thank our investigators, and the patients and their families for participating in the bestPWS ZAF-311 clinical trial," he added.

In their six-month randomized trial period, beloranib demonstrated a statistically significant reduction in both body weight and hyperphagia-related behaviors; the first drug to have that kind of positive results when it comes to PWS.

According to a statement from Zafgen, PWS is the most common genetic cause of obesity, which can pose great danger to the lives of the patients. Some of the related health risks that contributes to death due to obesity include slowed metabolism, psychiatric conditions including aggression, anxiety, and psychosis, higher risk for cardiopulmonary and metabolic co-morbidities.

Apart from the weight loss effect of beloranib, there is also an improvement in the hyperphagia-related behaviors among PWS patients. These behaviors are hunger-related and could have a great impact in the lives of the patients.

"Prader-Willi syndrome significantly impacts the quality of life of affected individuals and their families, as it drives patients to engage in excessive overeating, or hyperphagia, and may also lead to morbid obesity, which can be life-threatening if not controlled," said Merlin G. Butler, professor of Psychiatry, Behavioral Sciences and Pediatrics and director of the Division of Research and Genetics, Department of Psychiatry, Behavioral Sciences and Pediatrics at the Kansas University Medical Center.

According to Butler, there is no available treatment for PWS so far and the positive results from the trial are meaningful and may warrant further evaluation of the drug as a potential treatment.