Soleno Therapeutics Provides Regulatory Update on DCCR for Prader-Willi Syndrome

REDWOOD CITY, Calif., Oct. 08, 2024 – Soleno Therapeutics, Inc. (NASDAQ: SLNO), a clinical-stage biopharmaceutical company, has provided an update regarding its interactions with the U.S. Food and Drug Administration (FDA). The FDA's Review Division has indicated that there is currently no necessity for an advisory committee meeting for Soleno's New Drug Application (NDA) concerning DCCR (diazoxide choline) extended-release tablets, a proposed treatment for Prader-Willi syndrome (PWS). Despite this, the review team will continue to evaluate the potential requirement for such a meeting throughout their ongoing assessment process. Previously, the DCCR NDA was granted Priority Review, with a Prescription Drug User Fee Act (PDUFA) target action date set for December 27, 2024.

Prader-Willi syndrome (PWS) is a rare genetic disorder occurring in approximately one in every 15,000 live births, as estimated by the Prader-Willi Syndrome Association USA. The primary symptom of PWS is hyperphagia, characterized by an intense and persistent hunger, food preoccupation, and a compelling drive to seek and consume food. This condition significantly impacts the quality of life for individuals with PWS and their families. Other symptoms include behavioral problems, cognitive impairments, low muscle tone, short stature (if not treated with growth hormone), excessive body fat accumulation, developmental delays, and incomplete sexual development. Hyperphagia can lead to severe health issues, including stomach rupture, choking, accidental death due to food-seeking behavior, and long-term complications such as diabetes, obesity, and cardiovascular disease.

A global survey conducted by the Foundation for Prader-Willi Research revealed that 96.5% of respondents (parents and caregivers) considered hyperphagia the most crucial symptom to address with new treatments, while 92.9% emphasized the importance of managing body composition. Currently, there are no approved therapies specifically targeting the hyperphagia, metabolic disorders, cognitive dysfunction, or behavioral aspects associated with PWS.

DCCR (diazoxide choline) extended-release tablets are a unique, proprietary formulation containing diazoxide choline, the crystalline salt of diazoxide. Administered once daily, diazoxide has been historically used in various rare diseases across neonates, infants, children, and adults, although it has not been approved for use in PWS. Soleno Therapeutics has developed extensive patent protection for the therapeutic application of diazoxide, diazoxide choline, and DCCR for individuals with PWS.

The development program for DCCR includes data from five completed Phase 1 clinical studies in healthy volunteers and three completed Phase 2 clinical studies, one of which involved individuals with PWS. In the Phase 3 clinical development program for PWS, DCCR demonstrated potential in addressing hyperphagia and several other symptoms, such as aggressive and destructive behaviors, fat mass reduction, and improvements in other metabolic parameters.

Soleno Therapeutics, Inc. is dedicated to the development and commercialization of innovative treatments for rare diseases. The NDA for its lead candidate, DCCR (diazoxide choline) extended-release tablets, designed for once-daily oral administration for PWS treatment, is currently under FDA review and has received Priority Review designation.