FDA grants orphan drug status for CymaBay’s MBX-8025 to treat HoFH

Currently, MBX-8025 is being evaluated in high unmet need and orphan diseases.

HoFH is characterized by loss-of-function mutations in both alleles of the LDL receptor (LDL-R) gene and this loss results in marked elevations in the plasma levels of LDL cholesterol (LDL-C) in patients with this disease.

The loss of LDL-R activity causes premature cardiovascular disease that often presents during the first decades of life and which can result in myocardial infarction, ischemic stroke and premature death.

In clinical trials, MBX-8025 has been shown to reduce LDL-C in patients with mixed dyslipidemia,.

In addition, the company had recently reported data showing LDL-C lowering activity independent of fully functional LDL-R using MBX-8025 in a preclinical model of human HoFH.

CymaBay president and chief executive officer Harold Van Wart said: "Orphan designation is an important milestone for CymaBay as we advance the development of MBX-8025 in HoFH.

"As a potent, selective PPAR-d agonist, we believe 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 II pilot study of MBX-8025 in HoFH in the first half of this year, we also look forward to providing additional guidance on the expansion of our development strategy into a second indication."

The company is planning for a pilot Phase II trial of MBX-8025 in HoFH starting in the first half of 2015.