Evelien Gevers, PhD, MD, Consultant Pediatrician and Reader in Endocrinology and Diabetes at Barts Health NHS Trust, discusses effects of diazoxide choline extended release (DCCR) on hyperphagia in patients 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 missing or non-working genes on chromosome 15.
An abstract presented at ENDO 2026 evaluated long-term reductions in hyperphagia with DCCR in patients with PWS. DCCR is a potent activator of the adenosine triphosphate (ATP)-sensitive potassium channel. It was approved in March 2025 by the US Food and Drug Administration (FDA) as a treatment for hyperphagia in patients 4 years of age and older with PWS.
Results for up to 3 years from participants who received DCCR compared to real-world natural history data from the PATH for PWS study (PATH) were included.
A total of 352 participants were evaluated. Baseline Hyperphagia Questionnaire for Clinical Trials (HQ-CT) total scores were 21.6 ± 6.7 and 18.2 ± 5.0 for DCCR and PATH groups. Significant reductions in hyperphagia occurred at all time points for DCCR compared to the PATH cohort. Change in HQ-CT from Baseline between DCCR and PATH was -6.2 at year 1, -6.5 at year 2, and -6.2 at year 3.
To learn more about PWS and other rare endocrine conditions, visit https://checkrare.com/diseases/endocrine-disorders/
