FDA Approves Diazoxide Choline Extended-Release Tablets in Prader-Willi Syndrome

The FDA has announced the regulatory approval of diazoxide choline (DCCR, Vykat XR; Soleno Therapeutics) extended-release (ER) tablets for the treatment of individuals 4 years and older with Prader-Willi syndrome (PWS) who have associated hyperphagia, according to a news release from Soleno Therapeutics.¹

Image Credit: © Tada Images - stock.adobe.com

“The FDA approval of [DCCR] is an incredible achievement for the entire PWS community,” Jennifer Miller, MD, professor of pediatric endocrinology at the University of Florida, Gainesville, and principal investigator in the clinical development program of DCCR, said in a news release. “I am excited to have [DCCR] available to help treat hyperphagia, which is the most life-limiting aspect of PWS."¹

DCCR is designed as a novel, proprietary ER dosage of diazoxide choline, which is the crystalline salt of diazoxide, to be administered once daily. According to experts, diazoxide has been used for years in patients with rare diseases in neonates, infants, and adults but has not yet been approved for use in PWS until now. Solen Therapeutics studied DCCR in multiple completed clinical trials, in which the therapy showed promise in addressing hyperphagia, which is the hallmark symptom of PWS.^1,2

In one such trial, DESTINY PWS (NCT03440814), which was a 13-week, randomized, double-blind, placebo-controlled, phase 3 study, 127 participants with PWS aged 4 years and older with hyperphagia demonstrated improvements when treated with DCCR compared with placebo. Although DCCR did not significantly improve hyperphagia, it did so in participants with severe hyperphagia (-9.67 vs -4.26; P = .012). Furthermore, major improvements were observed in key secondary end points of body composition and clinician-reported outcomes and were well-tolerated among the participants.³

According to the Prader-Willi Syndrome Association USA, PWS occurs in 1 in every 15,000 live births and is considered very rare. Hyperphagia, the classic presentation of PWS, is a chronic and deadly condition characterized by feelings of intense and persistent hunger, food preoccupation, and a consistent drive to consume food, which can diminish quality of life for patients and their families. Other typical characteristics of PWS include behavioral problems, low muscle tone, short stature, and an excessive accumulation of body fat.²

Hyperphagia is especially concerning for patients with PWS and can lead to high rates of mortality through complications such as stomach rupture, choking, and accidental death due to food-seeking behavior. Longer-term comorbidities that may develop as a result of or in conjunction with PWS include diabetes, obesity, and cardiovascular disease. In a critical finding from a survey conducted by the Foundation for Prader-Willi Research, 96.5% of respondents rated hyperphagia and 92.9% rated body composition as the most important or very important symptoms to be relieved by a possible new medicine.²

In August 2024, the FDA accepted the new drug application and granted priority review to DCCR. In addition, the FDA granted breakthrough, fast track, and orphan drug designations for the treatment of patients with PWS, helping expedite its path to approval. Further studies should be conducted to evaluate the effectiveness of DCCR in patients with less severe disease to broaden the indication of DCCR and provide more patients with desperately needed relief.²

REFERENCES

1. Soleno Therapeutics. Soleno Therapeutics announces US FDA approval of VYKAT XR to treat hyperphagia in Prader-Willi syndrome. News Release. Released March 26, 2025. Accessed March 27, 2025. https://investors.soleno.life/news-releases/news-release-details/soleno-therapeutics-announces-us-fda-approval-vykattm-xr-treat

2. Miller KL, Gevers E, Bridges N, et al. Diazoxide choline extended-release tablet in people with Prader-Willi syndrome: A double-blind, placebo-controlled trial. J Clin Endocrinol Metab. 2023;108(7):1676-1685. doi:10.1210/clinem/dgad

3. Soleno Therapeutics. Soleno Therapeutics announces FDA extension of review period for DCCR (diazoxide choline) extended-release tablets in Prader-Willi syndrome. News Release. Released November 26, 2024. Accessed March 6, 2025.