News

The U.S. Food and Drug Administration (FDA) has delayed by three months its decision on whether to approve Soleno Therapeutics’ extended-release formulation of diazoxide choline — known as DCCR — for curbing excessive hunger in people with Prader-Willi syndrome (PWS). The therapy candidate is designed for use in…

Hospitalizations among people with Prader-Willi syndrome (PWS) are characterized by higher healthcare utilization and complexity compared with non-PWS patients, according to a large-scale analysis of a U.S.-based administrative claims database. In addition to high rates of obesity, PWS patients had longer hospital stays, higher associated costs, and more in-hospital…

The U.S. Food and Drug Administration (FDA) has decided that an advisory committee meeting is not needed at this point in the review process of Soleno Therapeutics‘ application seeking approval of its extended-release formulation of diazoxide choline (DCCR) to treat Prader-Willi syndrome (PWS). Soleno announced the regulatory update…

The U.S. Food and Drug Administration (FDA) will review Soleno Therapeutics’ application seeking approval of its extended-release formulation of diazoxide choline (DCCR) for Prader-Willi syndrome (PWS) in patients, 4 and older, with excessive hunger. The new drug application (NDA), filed in July, was granted priority review, shortening…

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…