News

The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to CSTI-500, Consynance Therapeutics’ oral treatment candidate for children and adolescents with Prader-Willi syndrome (PWS). That status is given to experimental therapies with the potential to provide clinically meaningful benefits to patients, primarily those younger than…

Soleno Therapeutics is seeking approval of diazoxide choline controlled-release (DCCR) tablets in the U.S. for Prader-Willi syndrome (PWS) patients, 4 and older, with excessive hunger. The filing of the new drug application with the U.S. Food and Drug Administration (FDA) follows positive data from an open-label extension…

About every 10th person with Prader-Willi syndrome (PWS) experiences epilepsy or fever-induced seizures, and more likely so if the disease is caused by a specific type of mutation, a review study has found. Also, this type of mutation — deletions in a segment of chromosome 15 and the most common…

Throughout May, supporters will celebrate Prader-Willi Syndrome (PWS) Awareness Month with a variety of educational, advocacy, and fundraising initiatives. The goal of the month-long event is to raise awareness of the rare genetic disease and to support efforts that will accelerate PWS research and treatment development. As it has…

Harmony Biosciences has started a pivotal Phase 3 clinical trial testing pitolisant for excessive daytime sleepiness (EDS) and behavioral problems in people with Prader-Willi syndrome (PWS), 6 and older. A pivotal study is one where results, if positive, are used to support applications for the therapy’s regulatory approval.

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…

People with Prader-Willi syndrome (PWS) caused by a genetic deletion are significantly more likely to have pale skin, seizures, and language development delays than PWS from other types of mutations. A PWS-causing genetic deletion was also associated with a higher frequency of excessive and…

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…

Acadia Pharmaceuticals has launched a Phase 3 clinical trial testing the safety and efficacy of ACP-101, an experimental carbetocin nasal spray, for the treatment of hyperphagia, or insatiable hunger, in people with Prader-Willi syndrome (PWS). The pivotal trial, dubbed COMPASS PWS, is expected to enroll up to…

The Prader-Willi Syndrome Association USA (PWSA) has again been named a winner of the Patients at the Heart awards, an effort by Harmony Biosciences to help fund nonprofits that work for people with sleep disorders and rare neurological diseases. The award will fund PWS Sleep Within Reach…