The FDA has granted orphan drug designation to Tesomet as a treatment for Prader-Willi syndrome (PWS). 

According to a release from the manufacturer, Tesomet is a combination of tesofensine and metoprolol.  

The release noted that as many as 34,000 people in the United States and 50,000 people in Europe live with PWS.  

Patients diagnosed with PWS can experience a range of symptoms including insatiable appetite, abnormal growth and body composition, low muscle tone, and other deficits.  

According to the release, due to the issues with appetite, patients can become morbidly obese and have a shorter life expectancy.  

Common causes of mortality in these patients include respiratory disease, cardiac disease, choking, and pulmonary embolism.  

Rudolf Baumgartner, MD, Chief Medical Officer and Heald of Clinical Development at Saniona, said in the release that there are currently no medicines approved to “address the uncontrollable hunger or hyperphagia” associated with the disease.  

Current treatment options can include hormone replacement.  

Tesomet had previously been evaluated in a double-blind, placebo-controlled phase 2a trial. The release noted that adults treated with Tesomet had a statistically significant reduction in hyperphagia and a clinically meaningful reduction in body weight.  

An extension study of adolescent patients showed the treatment was well tolerated at lower doses, which the release said could suggest “dose-dependent effects on weight and hyperphagia.” 

Following that study, the manufacturer is planning a phase 2b study for the first half of 2021. The release noted that an Investigational New Drug application has been opened with the FDA and is assessing sites globally for the trial to be held.  

As part of the preparation for the trial, the manufacturer is reaching out to groups in the PWS advocacy community to get feedback on the treatment process and clinical trial development.  

The release noted that Tesomet is also being evaluated as a treatment for hypothalamic obesity with a clinical trial expected to start this year.  
 

By Adam Hochron