FDA Approves Soleno’s Therapy for Hyperphagia in PWS

Rare Daily Staff

The U.S. Food and Drug Administration approved Soleno Therapeutics Vykat XR extended-release tablets for the treatment of hyperphagia in adults and children 4 years of age and older with Prader-Willi syndrome.

Prader-Willi syndrome (PWS) is a rare genetic neurodevelopmental disorder caused by an abnormality in the gene expression on chromosome 15. The defining symptom of PWS is hyperphagia, a chronic and life-threatening condition characterized by an intense persistent sensation of hunger accompanied by food preoccupations, an extreme drive to consume food, food-related behavior problems, and a lack of normal satiety, which can severely diminish the quality of life for individuals with PWS and their families. Hyperphagia can 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.

Vykat XR is a novel, proprietary extended-release dosage form containing diazoxide choline, the crystalline salt of diazoxide, and is administered once-daily. Vykat XR has received Orphan Drug designation for the treatment of PWS in the United States and European Union and Fast Track and Breakthrough designations in the United States.

The FDA approval of Vykat XR was based on study and safety data from the comprehensive clinical development program. Efficacy was established during the 16-week randomized withdrawal study period of Study 2-RWP, a phase 3 multi-center, randomized, double-blind, placebo-controlled trial. Individuals randomized to switch to placebo demonstrated a statistically significant worsening of hyperphagia compared with individuals 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 a well-established safety profile with over four years of data across four double-blind and/or open label studies. The most common adverse reactions occurring in greater than or equal to 10 percent of individuals receiving Vykat XR and at 2 percent greater than placebo included excessive hair growth, edema, hyperglycemia, and rash.

“The FDA approval of Vykat XR is an incredible achievement for the entire PWS community,” said Jennifer Miller, 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. “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.”