The U.S. FDA has approved Soleno Therapeutics’ extended-release tablets for the treatment of hyperphagia in adults and children four years of age and older with Prader-Willi syndrome (PWS), marking the first drug to address the insatiable hunger associated with the condition.
PWS is a rare genetic condition usually caused by deletion of a part of chromosome 15 passed down by the father. The hallmark symptom of the disorder is hyperphagia, a chronic and life-threatening condition characterized by feelings of intense, persistent hunger, food pre-occupation, and an extreme drive to seek and consume food. Families of people with PWS have been prisoners in their own homes because of the need to provide constant, eyes-on supervision 24/7 with access to food being completely restricted.
Vykat XR, originally called DCCR, is a novel, proprietary extended-release dosage form containing diazoxide choline, the crystalline salt of diazoxide and is administered once-daily.
The FDA approval was based on data from a phase 3 multi-center, randomized, double-blind, placebo-controlled trial. Individuals randomized to switch to placebo demonstrated a statistically significant worsening of hyperphagia compared with individuals who remained on Vykat XR. Prior to participating in the randomized withdrawal period, all individuals received double-blind and/or open-label Vykat XR for a mean duration of 3.3 years.
Back in November, the FDA had extended the review period for the company’s new drug application, pushing the PDUFA date to March 27, 2025. The extension was based on the agency's determination that responses to recent information requests constituted a major amendment to the NDA. The FDA did not cite any safety, efficacy or manufacturing concerns in its correspondence.
Soleno expects Vykat XR to be available in the U.S. beginning in April 2025.