The U.S. Food and Drug Administration (FDA) announced that it has approved Soleno Therapeutics’ Vykat XR (diazoxide choline) to treat hyperphagia in individuals with Prader-Willi Syndrome (PWS).
PWS is a rare genetic disorder caused by the complete or partial deletion of chromosome 15. It is estimated that approximately 50,000 people in the U.S. are affected by this condition.
This syndrome affects multiple aspects of daily life, including appetite, behavior, and emotional well-being. Hyperphagia, or excessive hunger, is one of the key symptoms of PWS.
The symptoms of PWS appear from birth. Infants with PWS often have low muscle tone and struggle to breastfeed due to insufficient strength. As children with PWS grow older, they develop hyperphagia, experiencing constant hunger despite eating and never feeling full.
Hyperphagia typically begins around age 8 but can start as early as age 4, continuing into adulthood.
The abnormal increase in appetite can lead to rapid weight gain, which, if not carefully managed, can result in obesity, breathing difficulties, and heart complications.
Soleno Therapeutics’ Vykat XR is designed to alleviate hyperphagia by targeting specific pathways in the brain, reducing the secretion of appetite-regulating peptides.
In a later-stage clinical trial involving 127 patients with Prader-Willi Syndrome, Vykat XR was shown to reduce severe hunger in those with extreme hyperphagia. However, the results were not statistically significant compared to the placebo group.
Despite this, a long-term follow-up of patients who completed the trial showed that hyperphagia was significantly reduced after more than a year of treatment with Vykat XR.
Following FDA approval, Soleno Therapeutics plans to make Vykat XR available to patients aged 4 and older with hyperphagia due to PWS starting in April. The treatment will cost an average of $466,200, and dosage will be determined based on the patient’s weight.
The International Prader-Willi Syndrome Organization (IPWSO) announced that it will collaborate with Soleno and other global health organizations to ensure that Vykat XR becomes accessible worldwide.
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