Clinical-stage biopharmaceutical firm CymaBay Therapeutics has received orphan drug designation from the US Food and Drug Administration (FDA) for its MBX-8025 to treat patients with hyperlipoproteinemia types I or V (Fredrickson classification).
Patients with types I and V hyperlipoproteinemias will have severely elevated levels of triglycerides in the blood.
MBX-8025 is a potent and selective peroxisome proliferator-activated receptor delta agonist of PPARd, a nuclear receptor important for lipid transport, storage and metabolism in liver and muscle.
CymaBay Therapeutics president and CEO Harold Van Wart said: "This second orphan drug designation is a validation of the company’s development strategy for MBX-8025 in which we are targeting indications with high unmet need and potentially expedited approval pathways.
"MBX-8025 potentially offers unique benefits for the treatment of metabolic disorders, including HoFH, severe hypertriglyceridemia, primary biliary cirrhosis and nonalcoholic steatohepatitis.
"We remain on-track to initiate a Phase II pilot study of MBX-8025 in HoFH this quarter, and look forward to announcing further details regarding the expansion of our development strategy of MBX-8025 in the near-term."
According to the company, MBX-8025 demonstrated favourable effects on lipid and metabolic parameters in a Phase II study in patients with mixed dyslipidemia.
MBX-8025 helped in lowering of LDL-C with selective depletion of pro-atherogenic dense LDL-C particles and increased high density lipoprotein, as well as decreased triglycerides and cardiovascular inflammation biomarker hsCRP.
CymaBay has recently obtained FDA orphan drug designation for MBX-8025 to treat patients with homozygous familial hypercholesterolemia (HoFH).
The company plans to initiate a pilot clinical study to assess the activity of MBX-8025 in patients with HoFH.
Image: Hyperlipidaemia – lipid in EDTA tube. Photo: courtesy of Mark-shea.