FDA Grants Breakthrough Designation to Soleno's Diazoxide Choline for Prader-Willi Syndrome
Soleno Therapeutics, a company specializing in treatments for rare diseases, has announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to diazoxide choline.
This designation for treatment of Prader-Willi syndrome (PWS) in adults and children ages 4 and older with hyperphagia.
The FDA has found preliminary data from Phase 3 clinical trials suggest diazoxide choline may significantly improve clinical outcomes compared to current therapies.
The Breakthrough Therapy Designation aims to accelerate the development and review process for drugs targeting serious conditions when initial evidence suggests substantial improvement over existing treatments.
This condition is often accompanied by behavioral issues, cognitive delays, and low muscle tone, short stature, and excess body fat, developmental delays.
Hyperphagia can lead to serious health complications such as obesity, diabetes, cardiovascular disease, and life-threatening situations like stomach rupture or choking.
This designation provides enhanced guidance and a collaborative review process from senior FDA officials and may include priority review.
Diazoxide choline also holds Orphan Drug Designation for PWS in the U.S. and E.U., as well as Fast Track Designation in the U.S.
