The FDA has approved Vyktur XR, an extended-release formulation of diazoxide choline, as the first drug to manage hyperphagia in Prader-Willi syndrome (PWS). Hyperphagia is intense, chronic hunger that is a major barrier for those with PWS, a rare genetic disorder found in approximately one of every 15,000 US births.
Until now, managing hyperphagia involved strict diets and off-label use of obesity medications, but Vykat XR offers a new approach. It’s taken orally once daily and works by activating a specific channel in the brain, pancreas, and fat tissue to reduce the intense hunger associated with PWS.
University of Florida affiliate Dr. Jennifer Miller emphasized the significance of this approval for individuals with PWS, pointing to the way in which hyperphagia profoundly impacts daily functioning, often requiring round-the-clock monitoring and restricted food availability.
In clinical trials, Vykat XR showed efficacy in reducing hyperphagia compared to a placebo over 16 weeks. Common side effects included increased hair growth, swelling, high blood sugar, and rash. The drug is not recommended for patients with certain allergies or conditions like kidney or liver problems, and careful monitoring of blood sugar levels is required before and during treatment.
Vykat XR is available in three tablet dosages and is on the verge of release in April, with the new medication showing potential to dramatically improve the quality of life of PWS patients and families.
Source: www.medpagetoday.com/endocrinology/growthdisorders/114856
