FDA Approves Soleno's Vykat XR for Prader-Willi Hyperphagia Treatment

The US Food and Drug Administration (FDA) has recently approved Vykat XR (diazoxide choline) extended-release tablets, developed by Soleno Therapeutics, as the first treatment to combat the symptom of hyperphagia in patients suffering from Prader-Willi syndrome (PWS). This rare neurodevelopmental disorder, caused by a genetic anomaly on chromosome 15, affects approximately one in every 15,000 newborns in the United States.

Prader-Willi syndrome manifests through a diverse array of physical, mental, and behavioral challenges, with hyperphagia being its most pronounced symptom. The condition is characterized by an incessant and intense hunger that can lead to severe long-term health issues, including diabetes, obesity, and cardiovascular diseases. Vykat XR has gained approval for the oral, once-daily treatment of hyperphagia in both adult and pediatric patients with PWS, specifically those aged four years and above.

The decision by the FDA to endorse Vykat XR was significantly influenced by findings from the 2-RWP study, a phase 3 clinical trial. During a critical 16-week withdrawal period in the study, individuals who transitioned from Vykat XR to a placebo exhibited a statistically significant deterioration in hyperphagia symptoms, compared to those who continued receiving the medication. These results underscore the efficacy of Vykat XR in managing hyperphagia in PWS patients.

Vykat XR has also demonstrated a strong safety profile, with data backing its use for over four years across multiple studies, both double-blind and open-label in nature. This comprehensive safety and efficacy data played a crucial role in securing the FDA's approval.

Anish Bhatnagar, the chief executive officer of Soleno Therapeutics, hailed the FDA's approval as a significant achievement for the company and a monumental advancement for the PWS community, which until now did not have viable treatment options for managing hyperphagia, the most disruptive aspect of the disorder.

Echoing this sentiment, Stacy Ward, the chief executive officer of the Prader-Willi Syndrome Association USA, emphasized the historic nature of the approval. She highlighted that the introduction of Vykat XR addresses a longstanding unmet need for individuals living with PWS and their families, who face the relentless and disruptive challenges posed by hyperphagia. Vykat XR, she noted, offers a new ray of hope for many affected by the condition.

Soleno has indicated plans to make Vykat XR available in the US starting in April of this year. This development brings a much-needed therapeutic option to patients and families grappling with the relentless challenges of Prader-Willi syndrome, promising to improve the quality of life for many who have been waiting for effective treatment options.