The biopharmaceutical company Zafgen has announced the successful completion of the phase 3 trial of its treatment for an unusual genetic obesity disorder, known as Prader-Willi syndrome. The success comes after the trial of the treatment previously resulted in two deaths.
The Boston-based company’s beloranib has shown effectiveness in reducing weight of people suffering from this syndrome, which results in critical obesity due to intolerable hunger.
This syndrome currently doesn’t have any treatment and is found in one out of 12,000-15,000 people, reveals the data of the Prader-Willi Syndrome Association. Beloranib successfully reduced the body weight and controlled the unusually increased hunger behavior, known as hyperphagia. This is the first investigational drug to show such positive outcomes. “These pathologies are the two hallmarks of the disease. They’re widely agreed upon as the most important and completely unmet needs,” said Thomas Hughes, the company’s Chief Executive.
Decrease in body weight by beloranib was 9.45% when given higher dose and 8.2% when given lower dose as compared to the placebo. Reduction in the increased appetite was tested through the questionnaire. Dr. Hughes stated that the positive results will be instrumental in advancing discussions with the Food and Drug Administration (FDA) for allowing the company to continue to develop the drug. He added that the company has developed an advanced benefit/risk relationship after earlier reported tragedies.
The company might now be able to persuade the FDA to lift the ban that had been levied on the treatment after the deaths, with first death reported in October. The data associated with the trial and the company’s strategy to minimize the risk related with the treatment is likely to be submitted to the FDA after the study is completed within the quarter, reported Dr. Hughes. It has been revealed that earlier it was found that people with severe obesity, when treated with beloranib, can cause complications if the person has Type 2 diabetes.