Soleno Therapeutics Announces FDA Extension of Review Period for DCCR (Diazoxide Choline) Extended-Release Tablets in Prader-Willi Syndrome

Core Summary - The FDA has extended the review period for Soleno Therapeutics' NDA for DCCR (diazoxide choline) extended-release tablets, with the new PDUFA target action date set for March 27, 2025 [1] - The extension was due to the FDA's determination that responses to recent information requests constituted a major amendment to the NDA, requiring additional review time [2] - Soleno submitted the NDA on June 27, 2024, and it was granted Priority Review in August 2024 [3] - DCCR has received Breakthrough, Fast Track, and Orphan Drug Designations in the U S and E U for the treatment of Prader-Willi syndrome (PWS) [3] About PWS - PWS occurs in approximately 1 in 15,000 live births and is characterized by hyperphagia, a chronic and life-threatening condition [4] - Hyperphagia leads to severe quality of life issues and can cause significant mortality and long-term comorbidities such as diabetes, obesity, and cardiovascular disease [4] - 96 5% of respondents in a global survey rated hyperphagia as the most important or very important symptom to be relieved by a new medicine [4] - There are currently no approved therapies to treat hyperphagia, metabolic, cognitive function, or behavioral aspects of PWS [4] About DCCR - DCCR is a novel, proprietary extended-release dosage form of diazoxide choline, administered once daily [5] - The parent molecule, diazoxide, has been used for decades in other rare diseases but is not approved for PWS [5] - Soleno has established extensive patent protection for the therapeutic use of diazoxide, diazoxide choline, and DCCR in PWS [5] - The DCCR development program is supported by data from five Phase 1 and three Phase 2 clinical studies, including one in PWS patients [5] - In Phase 3 trials, DCCR showed promise in addressing hyperphagia and other symptoms such as aggressive behaviors and metabolic parameters [5] About Soleno Therapeutics - Soleno is focused on developing and commercializing novel therapeutics for rare diseases [6] - The company's lead candidate, DCCR, is currently under FDA review for the treatment of PWS and has been granted Priority Review [6]