Soleno Therapeutics announced positive data from its clinical development program for VYKAT XR (diazoxide choline) extended-release tablets, a treatment for hyperphagia in Prader-Willi syndrome (PWS). The data, presented at the 2025 Pediatric Endocrine Society Annual Meeting, showed significant improvements in hyperphagia and behavioral symptoms in PWS patients who resumed VYKAT XR treatment after a 16-week withdrawal period. The study reinforces the drug’s effectiveness in managing crucial PWS symptoms and underscores the need for continuous, long-term treatment.
This news is particularly encouraging for PWS patients and their families, as hyperphagia, a defining characteristic of PWS, presents significant challenges and health risks. The demonstrated improvements in both hyperphagia and behavioral symptoms suggest a potential for improved quality of life and reduced long-term health complications associated with the disorder. The reaffirmation of VYKAT XR’s effectiveness strengthens its position as a vital treatment option, offering a tangible benefit to those affected by this rare disease.
Participants who restarted VYKAT XR after the withdrawal period experienced a 6.3-point reduction in the Hyperphagia Questionnaire for Clinical Trials (HQ-CT) total score after one year, returning to pre-withdrawal baseline levels. Furthermore, behavioral improvements were observed across all six assessed domains throughout the year. Notably, VYKAT XR received FDA approval in March 2025 and is commercially available in the US.
This positive data further validates the clinical utility of VYKAT XR and strengthens its market position as a recently approved treatment for PWS. Continued positive outcomes may lead to increased adoption of VYKAT XR, benefiting a larger population of PWS patients and potentially driving further research and development in this therapeutic area. This solidifies the drug’s role in addressing the unmet needs of PWS patients and offers hope for a better future in managing this challenging condition.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
