FDA approves first therapy to treat hyperphagia in Prader-Willi syndrome
Key takeaways:
- Diazoxide choline extended-release is the first FDA-approved therapy to address hyperphagia in Prader-Willi syndrome.
- Soleno Therapeutics announced the medication will be available beginning in April.
The FDA has approved the first therapy to address hyperphagia for children and adults aged 4 years and older with Prader-Willi syndrome, Soleno Therapeutics announced.
Prader-Willi syndrome is a rare genetic neurodevelopmental disorder caused by an abnormality in the gene expression on chromosome 15, according to a press release from Soleno Therapeutics. The disorder commonly leads to hyperphagia, which can increase risk for mortality; long-term comorbidities such as obesity, diabetes and cardiovascular disease; and have a negative impact on the individual’s quality of life, according to Jennifer Miller, MD, professor of pediatric endocrinology at the University of Florida in Gainesville and a principal investigator in the Vykat XR clinical development program.
“Until this point, we have had no medications or treatments for the hyperphagia, which is the most life-limiting aspect of the syndrome,” Miller told Healio. “People literally had to lock their kitchens, alarm the individual’s bedroom door so if they snuck out to get food the parents would be alerted, put motion-activated cameras in the home to catch individuals trying to sneak into the kitchen and break locks to get to food. The hyperphagia prevents these individuals from living life.”
Trial findings
Diazoxide choline extended-release tablets (Vykat XR, Soleno Therapeutics) earned FDA approval based on findings from the withdrawal period of a phase 3 randomized, placebo-controlled trial. According to top-line results released in 2023 by Soleno Therapeutics, 77 patients with Prader-Willi syndrome who previously received open-label diazoxide choline extended-release for 2 to 4 years were randomly assigned, 1:1, to continue diazoxide choline or switch to placebo for 16 weeks. Participants who switched to placebo had worse hyperphagia-related behaviors at 16 weeks compared with those who remained on diazoxide choline extended-release.
Miller said the medication reduces insulin secretion within beta cells of the pancreas. This lowers excessive weight gain and hyperphagia, reduces secretion of two potent hunger hormones in the hypothalamus and inhibits neurons that allow improved insulin sensitivity and appetite, according to Miller.
In primary safety analyses, the most common adverse events with diazoxide choline extended release were hypertrichosis, edema, hyperglycemia and rash.
A ‘life-changing’ therapy
The FDA’s approval of diazoxide choline extended-release was applauded by two nonprofit organizations that advocate for Prader-Willi syndrome research and people who are affected by the disorder.
“Today marks a historic day for the Prader-Willi syndrome community,” Stacy Ward, CEO of the Prader-Willi Syndrome Association | USA, said in the release. “The FDA’s approval of Vykat XR represents a monumental step forward in addressing the longstanding unmet needs of individuals living with Prader-Willi syndrome and their families. 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," Susan Hedstrom, executive director of the Foundation for Prader-Willi Research, said in the release. "For years, families and researchers have worked towards a treatment option that truly addresses the complexities of Prader-Willi syndrome. Today, we take a major step forward in changing the future for individuals navigating hyperphagia associated with Prader-Willi syndrome.”
Miller said the medication had a positive impact on many trial participants with Prader-Willi syndrome, allowing them to go to college, maintain a job, live independently and perform life tasks that they were unable to do so previously due to hyperphagia symptoms.
“This therapy is nothing short of life-changing for patients and their families,” Miller said.
Soleno Therapeutics announced diazoxide choline extended release will be available in the U.S. beginning in April.
Reference:
- Soleno Therapeutics announces positive statistically significant topline results from randomized withdrawal period of study C602 of DCCR for Prader-Willi Syndrome. https://investors.soleno.life/news-releases/news-release-details/soleno-therapeutics-announces-positive-statistically-significant. Published Sept. 26, 2023. Accessed March 27, 2025.
For more information:
Jennifer Miller, MD, can be reached at millejl@peds.ufl.edu.
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