Margarida Maia, PhD,  science writer—

Hypogonadism, a condition in which the body produces few or no sex hormones, is common in girls and young women with Prader-Willi syndrome (PWS). But while hormone replacement therapy (HRT) is often prescribed, many patients stop using it because of bothersome side effects, a U.S. study found. “These findings…

Aardvark Therapeutics plans to expand its ongoing Phase 3 clinical trial of ARD-101, an oral therapy for easing excessive hunger in Prader-Willi syndrome (PWS), to include patients younger than 13. The decision, supported by the PWS community, is based on historical data indicating that younger PWS patients are…

A year of treatment with somatropin safely maintains or improves body composition in Japanese children and adults with Prader-Willi syndrome (PWS). The findings come from a Pfizer-funded Phase 3 clinical trial (NCT04697381) that evaluated somatropin’s one-year safety and efficacy in 33 PWS patients on stable growth hormone treatment…

Most children and adolescents with Prader-Willi syndrome (PWS) experience frequent anxiety and engage in distress-related behaviors that become a regular part of daily life for their families. That’s according to results from a survey completed by caregivers at the start of the Phase 3 CARE-PWS clinical trial (NCT03649477),…

Vykat XR (diazoxide choline) can safely reduce hyperphagia — a feeling of insatiable hunger that is a hallmark of Prader-Willi syndrome (PWS) — in patients whose access to food has been restricted and in those with pre-diabetes or diabetes. These data were presented in two poster presentations at…

After discontinuing Vykat XR (diazoxide choline) for a few months as part of a clinical trial, Prader-Willi syndrome (PWS) patients who restarted the treatment showed sustained reductions in insatiable hunger, or hyperphagia, and behavioral problems. The results were presented in a poster, titled “Resuming diazoxide choline extended-release (DCCR)…

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…

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…

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…