Soleno Therapeutics Announces FDA Priority Review of NDA for DCCR in Prader-Willi Syndrome

Soleno Therapeutics, Inc., a biopharmaceutical company based in Redwood City, California, has announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for DCCR, a treatment for Prader-Willi syndrome (PWS). This rare disorder primarily affects individuals aged four and older who suffer from hyperphagia. The FDA has granted Priority Review for this NDA and has established a Prescription Drug User Fee Act (PDUFA) target action date of December 27, 2024. Priority Review is given to drugs that, if approved, would significantly improve the treatment, prevention, or diagnosis of serious conditions.

In its Priority Review Designation letter, the FDA indicated that it plans to hold an advisory committee meeting to discuss the application for DCCR. Anish Bhatnagar, M.D., the CEO of Soleno, expressed his gratitude towards the PWS community, including patients, caregivers, and advocacy groups, for their support. He emphasized that the FDA's acceptance of their NDA is a significant milestone and confirmed the agency's recognition of PWS as a serious condition.

PWS is a rare genetic disorder estimated to occur in one out of every 15,000 live births, according to the Prader-Willi Syndrome Association USA. The disorder is characterized by hyperphagia, which involves an intense and persistent hunger, preoccupation with food, and an extreme drive to seek and consume food. This condition severely affects the quality of life for individuals with PWS and their families. Other symptoms include behavioral issues, cognitive disabilities, low muscle tone, short stature (if untreated with growth hormone), excess body fat, developmental delays, and incomplete sexual development. Hyperphagia can lead to significant mortality risks, such as stomach rupture, choking, and accidental death due to food-seeking behaviors. Long-term complications can include diabetes, obesity, and cardiovascular diseases. A global survey by the Foundation for Prader-Willi Research found that 96.5% of respondents considered hyperphagia a critical symptom needing treatment, and 92.9% viewed body composition issues similarly. Currently, there are no approved therapies for managing the hyperphagia, metabolic, cognitive, or behavioral aspects of PWS.

DCCR (Diazoxide Choline) Extended-Release Tablets represent a novel, proprietary extended-release formulation containing diazoxide choline, a crystalline salt of diazoxide. This medication is administered once daily. The parent compound, diazoxide, has been used for decades to treat a few rare diseases in various age groups but is not yet approved for use in PWS. Soleno has established extensive patent protection for the therapeutic use of diazoxide, diazoxide choline, and DCCR in individuals with PWS. The development program for DCCR is supported by data from five Phase 1 clinical studies in healthy volunteers and three Phase 2 clinical studies, one of which included individuals with PWS. In its Phase 3 clinical development program for PWS, DCCR showed promise in addressing hyperphagia, the hallmark symptom of PWS, as well as other symptoms like aggressive and destructive behaviors, fat mass, and other metabolic parameters.

Soleno Therapeutics, Inc. focuses on developing and commercializing novel therapeutics for treating rare diseases. The company's lead candidate, DCCR, is currently under FDA review and has been granted Priority Review.