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FDA Greenlights Soleno Therapeutics’ VYKAT XR for PWS Hyperphagia
Soleno Therapeutics Inc. announced that the U.S. Food and Drug Administration (FDA) has approved VYKAT XR (diazoxide choline) extended-release tablets to treat hyperphagia in patients with Prader-Willi syndrome (PWS) aged four and older. Previously known as DCCR, VYKAT XR is set to launch in the U.S. in April 2025.
Approval of VYKAT XR Supported by Positive Phase 3 Trial Results
The FDA approved VYKAT XR based on data from a well-controlled study and a comprehensive clinical development program. Efficacy was demonstrated in the 16-week randomized withdrawal phase of Study 2-RWP (Study C602-RWP), a Phase 3 multicenter, double-blind, placebo-controlled trial. Patients who switched to placebo showed a statistically significant worsening of hyperphagia compared to those who remained on VYKAT XR. Before entering the withdrawal phase, all participants had received double-blind and/or open-label VYKAT XR for an average of 3.3 years.
VYKAT XR has a well-established safety profile, supported by over four years of data from four double-blind and open-label studies. Primary safety analyses from Study 1 (Study C601) identified the most common adverse reactions (≥10% incidence and at least 2% higher than placebo), including hypertrichosis, edema, hyperglycemia, and rash.
Soleno Therapeutics submitted the New Drug Application (NDA) for VYKAT XR on June 27, 2024. In August, the FDA accepted the submission and granted it priority review. The agency also designated VYKAT XR as a breakthrough therapy, fast track, and orphan drug.
“Today marks a historic day for the PWS community. The FDA’s approval of VYKAT XR represents a monumental step forward in addressing the longstanding unmet needs of individuals living with PWS and their families,” said Stacy Ward, Chief Executive Officer of the Prader-Willi Syndrome Association | USA. “Our families experience the constant and disruptive challenges of hyperphagia, and VYKAT XR offers hope to so many.”
PWS Affects 1 in 15,000 Live Births with Life-Threatening Hyperphagia
PWS is a rare genetic neurodevelopmental disorder resulting from abnormal gene expression on chromosome 15. According to the Prader-Willi Syndrome Association USA, PWS affects approximately one in 15,000 live births. The hallmark symptom, hyperphagia, is a chronic and life-threatening condition marked by an intense, persistent hunger, obsessive food-related behaviors, and an impaired sense of satiety.
This condition severely impacts the quality of life for individuals with PWS and their families, leading to increased mortality risks such as stomach rupture, choking, and accidental death from food-seeking behaviors. Long-term complications include obesity, diabetes, and cardiovascular disease.
Currently, treatment options for PWS focus on managing symptoms rather than addressing the underlying genetic cause. Growth hormone therapy is the most widely used treatment, helping to improve body composition, muscle tone, and overall physical development. Other interventions include behavioral therapies and structured nutritional plans to manage hyperphagia and prevent obesity. Medications such as GLP-1 receptor agonists, traditionally used for diabetes and weight management, are being explored for their potential to reduce appetite in PWS patients. Additionally, ongoing clinical trials are investigating novel pharmacological approaches, including oxytocin, to address hyperphagia and associated behavioral challenges.
“This approval is a testament to the power of persistence, science, and advocacy,” said Susan Hedstrom, Executive Director of the Foundation for Prader-Willi Research. “For years, families and researchers have worked towards a treatment option that truly addresses the complexities of PWS. Today, we take a major step forward in changing the future for individuals navigating hyperphagia associated with PWS.”
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