Soleno Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to diazoxide choline for the treatment of adults and children ages 4 years and older with genetically confirmed Prader-Willi syndrome (PWS) who have hyperphagia. The designation reflects the Agency's determination that, based on an assessment of the preliminary data from the Phase 3 clinical development program, diazoxide choline may demonstrate substantial improvement on a clinically significant endpoint(s) over available therapies. The FDA's Breakthrough Therapy Designation is intended to expedite the development and review of drugs in the U.S. that are intended to treat a serious condition, when preliminary clinical evidence indicates the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint (s).

With Breakthrough Therapy Designation, FDA provides intensive guidance and organizational commitment involving senior managers in a proactive, collaborative, cross-disciplinary review, and may also allow for priority review and other actions to expedite review. Diazoxide choline has Orphan Drug Designation for PWS in the U.S. and E.U., as well as Fast Track Designation in the U.S.