Lindsey Shapiro, PhD, science writer —

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Harmony Biosciences’ pitolisant to treat daytime sleepiness in people with Prader-Willi syndrome (PWS). Orphan drug status is meant to incentivize the development of treatments for rare diseases, or those affecting fewer than 200,000 people in…

While food-seeking behaviors normally lessen as people age, they increase over time with Prader-Willi syndrome (PWS), according to a study using the Hyperphagia Questionnaire for Clinical Trials (HQ-CT), a tool commonly used in clinical trials but not in comparing a large PWS patient group to the general population. HQ-CT…

Prader-Willi syndrome (PWS) patients who switched from daily treatment with DCCR (diazoxide choline extended-release tablets) to a placebo experienced worsening excessive hunger, or hyperphagia, and greater body weight gains relative to those who stayed on DCCR. That’s according to top-line data from the new randomized withdrawal period of…

Harmony Biosciences plans to launch a Phase 3 clinical trial by year’s end to investigate whether pitolisant can safely and effectively reduce excessive daytime sleepiness in children, adolescents, and adults with Prader-Willi syndrome (PWS), the company said. The trial will be registrational, meaning that, if positive, the findings…

Neuren Pharmaceuticals will test its therapeutic candidate NNZ-2591 in children with Prader-Willi syndrome (PWS) in a new Phase 2 clinical trial, the company announced in a press release. The U.S. Food and Drug Administration (FDA) gave Neuren the go-ahead to conduct the trial following the company’s…

Researchers have determined that the role of the chemical messenger oxytocin in Prader-Willi syndrome could be associated with its function in astrocytes, a type of nerve support cell in the brain. By counting the number of oxytocin-producing cells and receptors for the molecule in the brain, brain regions and cell…

Daily treatment with oral pitolisant reduced excessive daytime sleepiness in children and adults with Prader-Willi syndrome (PWS), according to top-line data from a placebo-controlled Phase 2 clinical trial. While the relatively small proof-of-concept study wasn’t powered to detect statistically significant differences, the observed improvements with pitolisant relative to…

The loss of certain genetic material called non-coding RNA might lead to the disruption of genes important for brain development and function in Prader-Willi syndrome (PWS) , a study found. These alterations could underlie the cognitive and behavioral symptoms of the disease,  according to researchers. The study, “…

A randomized withdrawal period in the ongoing open-label extension trial of DCCR (diazoxide choline extended-release tablets) for people with Prader-Willi syndrome (PWS) has been initiated to generate more data about the investigational treatment’s effectiveness. The study, called C602 (NCT03714373), is evaluating the safety and effectiveness of daily…

The first patient has been randomly assigned to a dosing group in the pivotal Phase 2/3 SCOUT-015 trial, which will evaluate the safety and efficacy of RAD011, an investigational cannabidiol oral treatment, in people with Prader-Willi syndrome (PWS). SCOUT-015 (NCT05098509) is designed to test the effects of…