First Treatment (Diazoxide Choline) Approved for Hyperphagia in Prader-Willi Syndrome

Описание: Ashley Shoemaker, MD, Associate Professor of Pediatrics and Pediatric Endocrinology at Vanderbilt University, discusses the approval of Vykat XR (diazoxide choline) for the treatment of hyperphagia in patients ages 4 years and older with Prader-Willi syndrome (PWS).

PWS is a rare genetic condition that affects many parts of the body but its most dominant symptom is overwhelming hyperphagia. Infants with PWS also have severe hypotonia, feeding difficulties, slow growth, short stature, hypogonadism, developmental delays, cognitive impairment, and distinctive behavioral characteristics such as temper tantrums, stubbornness, and obsessive-compulsive tendencies. PWS is caused by gene mutations on chromosome 15.

Diazoxide choline is a once-daily oral treatment that is capable of activating the adenosine triphosphate-sensitive potassium channel and crossing the blood-brain barrier. It is the first therapy to be approved for the treatment of hyperphagia in PWS.

The approval follows positive data from the phase 3, multienter, randomized withdrawal, double-blind, placebo-controlled clinical trial. Results demonstrated a statistically significant increase in hyperphagia for those randomized to switch to placebo compared to those who remained on diazoxide choline. Prior to this randomized withdrawal period, participants received double-blind and/or open-label diazoxide choline for a mean duration of 3.3 years.

Additionally, the safety profile of the therapy is favorable with the most common adverse events being hypertrichosis, edema, hyperglycemia, and rash. These occurred in greater than or equal to 10% of participants.

The treatment is expected to be available to patients in the U.S. in early April 2025.

Chapters:
Introduction 00:00
Prader-Willi Syndrome Overview 00:34
Current Management 1:48
Vykat (Diazoxide Choline) Overview 2:38
Phase 3 Clinical Trial 4:06
Take Home Message 7:37