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.
Go deeper with GlobalData
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."
How well do you really know your competitors?
Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.
Thank you!
Your download email will arrive shortly
Not ready to buy yet? Download a free sample
We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form
By GlobalDataAccording 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.
