Soleno Therapeutics has announced that the FDA has approved VYKAT XR (diazoxide choline) extended-release tablets for the treatment of hyperphagia in adults and children 4 years of age and older with Prader-Willi syndrome (PWS).
Hyperphagia is the defining symptom of PWS. It is a chronic and life-threatening condition characterized by an intense persistent sensation of hunger.
“The approval of VYKAT XR is a significant milestone for Soleno and, most importantly, for the PWS community who have had no options to treat the most disruptive aspect of this disease,” said Anish Bhatnagar, MD, CEO of Soleno.
PWS is a rare genetic neurodevelopmental disorder caused by an abnormality in gene expression on chromosome 15. The Prader-Willi Syndrome Association USA estimates that the condition occurs in one in every 15,000 live births. Rare genetic causes of obesity make up a small proportion of causes of the condition, but there is still commercial interest in them.
Hyperphagia is characterized by intense persistent hunger accompanied by food preoccupations, an extreme drive to consume food, food-related behavior problems, and a lack of normal satiety.
This condition can severely diminish the quality of life for individuals with PWS and their families, as the patient must be under constant watch. It can also lead to significant mortality (e.g., stomach rupture, choking, accidental death due to food-seeking behavior) and longer term, co-morbidities such as diabetes, obesity, and cardiovascular disease.
Soleno expects VYKAT XR to be available in the United States beginning in April 2025. The company has launched Soleno One, a comprehensive patient support program.
“The FDA approval of VYKAT XR is an incredible achievement for the entire PWS community,” said Jennifer Miller, MD, professor of pediatric endocrinology at the University of Florida, Gainesville, who specializes in treating children and adults with PWS and is a principal investigator in the VYKAT XR clinical development program.
“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.”
VYKAT XR efficacy was established during a 16-week randomized withdrawal period of Study 2-RWP (Study C602-RWP), a Phase III multi-center, randomized, double-blind, placebo-controlled trial.
Individuals randomized to switch to placebo demonstrated a statistically significant worsening of hyperphagia compared with those who remained on VYKAT XR. Prior to participating in the randomized withdrawal period, all individuals received double-blind and/or open-label VYKAT XR for a mean duration of 3.3 years.
VYKAT XR has over four years of data across four double-blind and/or open-label studies. The primary safety analyses are based on Study 1 (Study C601) and the most common adverse reactions occurring in greater than or equal to 10% of individuals receiving VYKAT XR and at 2% greater than placebo included hypertrichosis, edema, hyperglycemia, and rash.
“I am excited to have VYKAT XR available to help treat hyperphagia, which is the most life-limiting aspect of PWS. Families of people with PWS have been prisoners in their own homes because of the need to provide constant, eyes-on supervision 24/7 with access to food being completely restricted,” said Hedstrom.
