March 27, 2025 – The FDA has approved a drug to treat a key symptom of a rare life-threatening genetic disorder known as Prader-Willi syndrome (PWS) in adults and children ages 4 or older. It marks the first treatment for an extreme, uncontrollable urge to eat, also known as hyperphagia.
PWS affects about 1 in 15,000 babies and disrupts the brain’s control over hunger, growth, and development. Newborns often have weak muscles, feeding difficulties, developmental delays, and hormone imbalances. As they grow, they have constant hunger and struggle to control their eating, leading to excessive weight gain and increased risks of obesity, diabetes, and heart disease. While supportive treatments are available, none effectively manage hyperphagia – the most challenging aspect of PWS.
The newly approved drug, known as diazoxide choline but sold as VYKAT XR (previously called DCCR), helps manage hunger by reducing brain chemicals that trigger appetite, improving fullness, and balancing hunger hormones. It also lowers insulin levels and reduces body fat. The once-a-day extended-release tablet maintains steady blood levels for continuous hunger control throughout the day.
Stacy Ward, CEO of the Prader-Willi Syndrome Association, USA, believes the approval addresses “longstanding unmet needs” of people with PWS and their families.
“Our families experience the constant and disruptive challenges of hyperphagia, and VYKAT XR offers hope to so many,” she said in a news release from Soleno Therapeutics, the drug’s maker.
The FDA approved VYKAT XR based on a clinical study that showed its effectiveness in hyperphagia. In the 16-week study, people who stopped taking VYKAT XR and switched to a placebo had worsening hunger symptoms, compared to those who stayed on the drug. Before this phase, all people in the study had already been taking VYKAT XR for about 3.3 years, confirming its long-term benefits.
Common side effects include unusual excessive hair growth, swelling, high blood sugar levels, and rash. The drug can raise blood sugar, requiring monitoring before and during treatment. It should be used with caution in people with heart problems. Patients should inform their doctor if they get swelling; if they have diabetes, high blood pressure, or heart conditions; or if they take medications for these issues, especially water pills like thiazide.
The treatment is expected to be available in the U.S. in April.