CymaBay gets FDA orphan drug status for MBX-8025 to treat hypertriglyceridemia

MBX-8025 is a potent and selective peroxisome proliferator-activated receptor delta (PPARd) agonist currently being evaluated by the company in high unmet need and orphan diseases.

Recently, MBX-8025 also secured FDA orphan drug status for the treatment of patients with homozygous familial hypercholesterolemia (HoFH).

The company said that patients with types I and V hyperlipoproteinemias have severely elevated levels of triglycerides in the blood.

Type I is characterized by deficiencies related to lipoprotein lipase (LPL) and by an elevation of chylomicron particles, while type V is characterized by an elevation in very-low-density lipoprotein (VLDL).

CymaBay president and chief executive officer 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."

Both types of hyperlipoproteinia are associated with an increased risk of acute and chronic pancreatitis that is severe and life-threatening.

Earlier, MBX-8025 has shown favorable effects on lipid and metabolic parameters in a Phase II trial in patients with mixed dyslipidemia.