Drug intelligence / Profile preview

volixibat

Development stage
Phase 2
Lead developer
Mirum Pharmaceuticals
Modality
Small Molecules
Administration
Oral
01

Overview

Volixibat is an orally bioavailable, highly potent and selective inhibitor of the apical sodium-dependent bile acid transporter (ASBT), also known as the ileal bile acid transporter (IBAT or SLC10A2). This transmembrane protein is primarily expressed by enterocytes in the ileum and is responsible for the enterohepatic recirculation of bile acids. By inhibiting ASBT/IBAT, volixibat reduces the reabsorption of bile acids from the intestine, leading to increased fecal excretion and decreased return of bile acids to the liver. This mechanism helps regulate hepatic lipid and glucose metabolism. Volixibat has been developed primarily for nonalcoholic steatohepatitis (NASH) and cholestatic pruritus associated with primary biliary cholangitis (PBC). The drug has received Breakthrough Therapy designation from the FDA for PBC-related pruritus due to positive clinical trial results showing significant improvement in itch severity and reduction in serum bile acids[2][3][5][8].

Other names
volixibat potassium
02

Targets

SLC10A2 (Solute carrier family 10 member 2)

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