CymaBay: FDA Grants Orphan Drug Designation For MBX-8025 - Quick Facts

(RTTNews) - CymaBay Therapeutics Inc. (CBAY) announced the U.S. FDA has granted the company orphan drug designation for MBX-8025 as a treatment for homozygous familial hypercholesterolemia.

MBX-8025 is a potent and selective peroxisome proliferator-activated receptor delta agonist being evaluated in high unmet need and orphan diseases. CymaBay is planning a pilot Phase 2 study of MBX-8025 in HoFH starting in the first half of 2015.

Harold Van Wart, CEO of CymaBay, said: "MBX-8025 may provide meaningful clinical benefit to patients across a number of diseases and disorders, including HoFH, primary biliary cirrhosis, severe refractory hypertriglyceridemia and nonalcoholic steatohepatitis, or NASH. As we move into a Phase 2 pilot study, we also look forward to providing additional guidance on the expansion of our development strategy into a second indication."