VYKAT XR Shows Sustained Benefits in Prader-Willi Syndrome Patients

The findings, detailed in the C614 open-label long-term extension study, highlight the efficacy of the drug in managing the core symptoms of the rare genetic disorder. Participants who restarted treatment following a 16-week randomized withdrawal period showed marked improvements in their Hyperphagia Questionnaire for Clinical Trials scores by week 13. These benefits remained consistent for two years, mirroring the stability seen in patients who maintained continuous therapy.

Beyond clinical trial data, researchers compared the outcomes of 125 VYKAT XR-treated individuals against 229 subjects from the PATH for PWS Natural History Study. Over a three-year observation period, the treatment group demonstrated statistically significant superiority across all six behavioral domains, including anxiety, irritability, and compulsivity. These results suggest that the drug addresses the extreme food-seeking behaviors and emotional instability characteristic of PWS, which can otherwise lead to severe medical complications like obesity and cardiovascular disease.

Sanjay Keswani, Chief Medical Officer at Neurocrine Biosciences, noted that the data reinforces the role of VYKAT XR as a foundational long-term treatment for patients four years of age and older. While the drug is already commercially available following its 2025 FDA approval, these long-term insights provide clinicians with stronger evidence regarding the necessity of uninterrupted therapy for patients living with the complexities of Prader-Willi syndrome.