Tuesday, April 30, 2024
Soleno Therapeutics, Inc. (Soleno) (NASDAQ: SLNO) has announced that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation to diazoxide choline for the treatment of adults and children aged 4 years and older with genetically confirmed Prader-Willi syndrome (PWS) who have hyperphagia. This designation acknowledges the FDA's assessment of preliminary data from the Phase 3 clinical development program, suggesting that diazoxide choline may offer significant improvement over existing therapies on clinically significant endpoints.
Soleno Therapeutics, noted that this designation is a significant milestone for their DCCR clinical development program, emphasizing the seriousness of PWS as a condition and the potential of DCCR to be an effective treatment. The focus remains on preparing the New Drug Application (NDA) submission for DCCR in PWS, expected in mid-2024.
The Breakthrough Therapy Designation from the FDA accelerates the development and review of drugs for serious conditions. It involves intensive guidance and organizational commitment, potentially leading to priority review and other actions to expedite the process.
Diazoxide choline has Orphan Drug Designation for PWS in both the U.S. and E.U., as well as Fast Track Designation in the U.S.
Prader-Willi Syndrome (PWS) affects approximately one in every 15,000 live births, characterized by hyperphagia, a chronic and life-threatening hunger. Current therapies do not effectively address the core symptoms of PWS.
DCCR (Diazoxide Choline) Extended-Release Tablets offer a novel once-daily formulation. Supported by extensive clinical data, including Phase 1 and Phase 2 studies, DCCR has shown promise in addressing hyperphagia and other symptoms associated with PWS. With multiple regulatory designations, including Orphan Drug, Fast Track, and Breakthrough Therapy, DCCR holds considerable potential as a therapeutic option for PWS patients.
Source: globenewswire.com
