FDA

Vykat XR approved for Prader-Willi syndrome-related hyperphagia

April 1, 2025

Brand name: Vykat XR

Generic name: diazoxide choline

Manufacturer: Soleno Therapeutics, Inc.

Approval date: March 26, 2025

FDA approved Vykat XR (diazoxide choline) extended-release tablets, previously referred to as DCCR, for the treatment of hyperphagia in adults and children ≥4 years of age with Prader-Willi syndrome (PWS).

Efficacy

Approval was based on data from a 16-week, double-blind, placebo-controlled, randomized withdrawal study (NCT03714373) that followed an open-label study period of diazoxide choline ER. Participants with hyperphagia and PWS were randomly assigned to receive diazoxide choline ER (n=38) or placebo (n=39).

The primary endpoint was the change from baseline to week 16 in the Hyperphagia Questionnaire for Clinical Trials (HQ-CT) total score. The HQ-CT is a 9-item, observer-reported outcome measure that assesses a range of hyperphagic and food-related behaviors during the prior two weeks. An item score of 0 indicates an absence of behaviors, with a score of 4 indicating the most frequent or severe behaviors. The HQ-CT Total Score may range from 0 to 36, with higher scores indicating greater overall severity of hyperphagic and food-related behaviors.

At the end of the 16-week randomized withdrawal study period, there was statistically significant worsening of hyperphagia in the placebo group relative to the diazoxide choline ER group, as assessed by the HQ-CT Total Score.

Safety

The most common adverse reactions (incidence ≥10% and ≥2% greater than placebo) in the trial were hypertrichosis, edema, hyperglycemia, and rash.

Sources:

Vykat XR (diazoxide choline) [package insert]. U.S. Food and Drug Administration. https://www.accessdata.fda.gov/drugsatfda_docs/label/2025/216665s000lbl.pdf Revised March 2025. Accessed March 31, 2025.

Soleno Therapeutics announces US FDA approval of Vykat(TM) XR to treat hyperphagia in Prader-Willi syndrome. [News release] 2025. https://investors.soleno.life/news-releases/news-release-details/soleno-therapeutics-announces-us-fda-approval-vykattm-xr-treat