New Drugs and Therapeutics Update: VYKAT™ XR (diazoxide choline)
Prepared on behalf of PES Drugs and Therapeutics Committee by Dania Al-Hamad, MD, and Neha Patel, DO.
On March 26, 2025, the U.S. Food and Drug Administration (FDA) approved VYKATTM XR (diazoxide choline extended-release) for the treatment of hyperphagia in patients 4 years and older with Prader-Willi syndrome (PWS).
Diazoxide choline extended release reduced hyperphagia in individuals with PWS. It was also associated with significant improvements in body composition, as well as reduced leptin, acylated ghrelin, and insulin levels compared to placebo. The most common adverse events included hypertrichosis, peripheral edema, and hyperglycemia, although these rarely led to treatment discontinuation. VYKATTM XR is administered once daily, with dosing based on body weight with a maximum dose of 5.8 mg/kg/day or 525 mg/day.