US: The US Food and Drug Administration (FDA) has approved Soleno Therapeutics’ groundbreaking drug, Vykat XR, to treat hyperphagia- the persistent and intense hunger that defines Prader-Willi syndrome (PWS).
This marks the first treatment available for the debilitating genetic disorder, which affects approximately 50,000 people in the United States.
PWS is caused by full or partial deletions on chromosome 15, disrupting gene expression and leading to profound impacts on eating behavior, mood, and overall well-being.
Infants with the condition are typically born with low muscle tone and an inability to breastfeed. As they grow, children develop hyperphagia, which often manifests around age four and continues into adulthood, leading to excessive weight gain, obesity, and related health complications such as heart disease and respiratory issues.
Today, we announced that the FDA approved VYKAT™ XR (diazoxide choline) extended-release tablets for the treatment of hyperphagia in individuals aged 4 and older with Prader-Willi syndrome (PWS). We can proudly say that VYKAT XR is the first and only FDA-approved medicine to… pic.twitter.com/A5ga9NE2Hr
— SolenoTx (@SolenoTx) March 26, 2025
Vykat XR, a once-daily oral pill, targets a specific pathway in the brain to regulate appetite by reducing the secretion of a key peptide linked to hunger. Soleno plans to launch the drug in April 2025 for US patients aged four years and older with PWS-related hyperphagia. The treatment will cost an average of $466,200 per year and be administered based on the patient’s weight.
The FDA’s approval follows an extended review of the drug, which included mixed results from a late-stage clinical trial. In a study of 127 patients, the drug showed notable reductions in hyperphagia among those with severe symptoms, though it did not achieve a statistically significant difference compared to the placebo group. However, a long-term follow-up study demonstrated substantial benefits for patients who continued treatment for over a year.
Anthony Holland, president of the International Prader-Willi Syndrome Organisation (IPWSO), affirmed the group’s commitment to expanding global access to Vykat XR.
