News

Thanks to strong enrollment in a Phase 3 clinical trial of Acadia Pharmaceuticals’ ACP-101 — an experimental nasal spray of carbetocin for treating hyperphagia in Prader-Willi syndrome (PWS) — the company announced that it now expects to have top-line data later this year. That trial, COMPASS PWS…

Parents and siblings of people with Prader-Willi syndrome (PWS) commonly experience stress and other mental health challenges, but greater family cohesion, or bonding, helped buffer against these adverse outcomes, according to a study that surveyed people from around the world. In questionnaires completed by parents, parental depression, anxiety, and…

May is Prader-Willi Syndrome (PWS) Awareness Month and the worldwide PWS community is coming together to spread awareness about the rare genetic condition. Also, May 15 is PWS Awareness Day in the U.S. The Prader-Willi Syndrome Association USA (PWSA USA) is encouraging the community to engage in…

The first eligible Prader-Willi syndrome (PWS) patients in the U.S. have received their prescriptions for Vykat XR (diazoxide choline) — the once-daily, extended-release tablets now approved as a treatment for hyperphagia, or insatiable hunger, in people with PWS, ages 4 and older. Soleno Therapeutics’ Vykat XR became…

The U.S. Food and Drug Administration (FDA) has approved Soleno Therapeutics’ extended-release formulation of diazoxide choline (DCCR) for easing hyperphagia — marked by insatiable hunger — in Prader-Willi syndrome (PWS) patients ages 4 and older. The newly authorized oral therapy will be marketed under the brand name…

Researchers have successfully used the gene-editing tool CRISPR to reactivate certain maternally-inherited genes that can compensate for the loss of the corresponding paternally-inherited genes in Prader-Willi syndrome (PWS). Done using lab-grown human stem cells with a PWS-causing mutation, the work showed that the one-time approach resulted in a sustained…

Rhythm Pharmaceuticals plans to start a new Phase 2 clinical study in the first quarter of 2025 to test setmelanotide in adults and children with Prader-Willi syndrome (PWS). An investigational therapy for PWS, setmelanotide is approved in the U.S. to treat certain types of genetic obesity under the…

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…