Prader-Willi syndrome (PWS) is a rare genetic disorder caused by deletions or disruptions on chromosome 15 that interfere with normal gene expression. It is estimated to affect 1 in 10,000 to 30,000 people worldwide and can affect various aspects of life, including appetite regulation, behavior, and emotional stability. One of its most notable symptoms is hyperphagia: an overwhelming and persistent feeling of hunger. While there is no current cure or way to prevent PWS, there are treatments available to manage the symptoms that come with the disorder.
A major step forward came with the recent FDA approval of VyKat XR, the first medication specifically developed to treat hyperphagia in individuals with PWS. VyKat Xr, formerly known as DCCR (Diazoxide Choline Controlled-Release), is the first commercial product produced by Soleno Therapeutics, a biopharmaceutical company focused on innovative treatments for rare diseases. VyKat XR is an extended-release version of the crystalline salt of diazoxide, designed to target the persistent hunger that characterizes PWS.
PWS is a rare genetic disorder caused by deletions or disruptions on chromosome 15.
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The FDA’s approval of the drug came after a lengthy review, largely due to mixed results from its late-stage clinical trial. That trial, which included 127 patients, showed that those with severe hyperphagia experienced a reduction in intense hunger. However, the drug did not demonstrate a statistically significant difference compared to the placebo group as a whole, which raised questions about its overall effectiveness.
Despite this, the FDA ultimately granted approval based on additional evidence from a long-term follow-up study. In that study, the same group of patients who completed the initial trial showed a significant reduction in hyperphagia after at least one year of treatment with VyKat XR.
Beginning in April 2025, Soleno plans to make VyKat XR available in the U.S. for patients ages four and older who have PWS with hyperphagia. This approval represents a major milestone not only in medical treatment but also in the day-to-day lives of patients and families dealing with Prader-Willi syndrome. For many, it brings long-awaited hope and a real possibility of improved quality of life.
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