FDA Approves First Therapy To Treat Hyperphagia in Prader-Willi Syndrome

Soleno Therapeutics, Inc., a biopharmaceutical company developing novel therapeutics for the treatment of rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved VYKAT™ XR (diazoxide choline) extended-release tablets, previously referred to as DCCR, for the treatment of hyperphagia in adults and children 4 years of age and older with Prader-Willi syndrome (PWS). Soleno expects VYKAT XR to be available in the U.S. beginning in April 2025.

“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, M.D., Chief Executive Officer of Soleno. “We are deeply grateful to the many individuals with PWS, their caregivers and clinical sites who participated in our trials, the advocacy groups, including FPWR and PWSA | USA, the advocates who have tirelessly supported the approval of VYKAT XR, the FDA for a collaborative review process, and our employees who have been committed to delivering VYKAT XR to those with PWS.”

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

“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.”

"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."

The FDA approval of VYKAT XR was based on an adequate and well-controlled 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 (Study C602-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 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.

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