Volixibat

Volixibat

Clinical data
Routes of administration	Oral
Legal status
Legal status	Investigational
Identifiers
IUPAC name 3-O-Benzyl-N-({3-[(3S,4R,5R)-3-butyl-7-(dimethylamino)-3-ethyl-4-hydroxy-1,1-dioxido-2,3,4,5-tetrahydro-1-benzothiepin-5-yl]phenyl}carbamoyl)-6-O-sulfo-β-D-glucopyranosylamine
Synonyms	SHP626
CAS Number	1025216-57-2
ChemSpider	35099493
UNII	X2JZ0451H8
Chemical and physical data
Formula	C38H51N3O12S2
Molar mass	805.96 g·mol^−1
3D model (Jmol)	Interactive image
SMILES CCCC[C@@]1(CS(=O)(=O)c2ccc(cc2[C@H]([C@H]1O)c3cccc(c3)NC(=O)N[C@H]4[C@@H]([C@H]([C@@H]([C@H](O4)COS(=O)(=O)O)O)OCc5ccccc5)O)N(C)C)CC
InChI InChI=1S/C38H51N3O12S2/c1-5-7-18-38(6-2)23-54(46,47)30-17-16-27(41(3)4)20-28(30)31(35(38)44)25-14-11-15-26(19-25)39-37(45)40-36-33(43)34(51-21-24-12-9-8-10-13-24)32(42)29(53-36)22-52-55(48,49)50/h8-17,19-20,29,31-36,42-44H,5-7,18,21-23H2,1-4H3,(H2,39,40,45)(H,48,49,50)/t29-,31-,32-,33-,34+,35-,36-,38-/m1/s1 Key:ULVBLFBUTQMAGZ-RTNCXNSASA-N

Volixibat (INN; development code SHP626) is a medication under development as a possible treatment for nonalcoholic steatohepatitis (NASH), the most severe form of non-alcoholic fatty liver disease (NAFLD). No other pharmacotherapy yet exists for NASH, so there is interest in whether volixibat can prove to be both safe and effective. To encourage development and testing, the U.S. Food and Drug Administration (FDA) has issued fast track status.^[1]

Volixibat is an IBAT inhibitor, meaning that it blocks the function of the IBAT protein (ileal bile acid transporter), which is also called SLC10A2 (solute carrier family 10 member 2) or ASBT (apical sodium–bile acid transporter). IBAT is most highly expressed in the ileum, where it is found on the brush border membrane of enterocytes. It is responsible for the initial uptake of bile acids, particularly conjugated bile acids, from the intestine as part of their enterohepatic circulation.^[2]

References

1. Chitnis, Deepak (2016-08-03), "FDA grants fast track status to volixibat", Internal Medicine News Digital Network, retrieved 2016-08-14.
2. Dawson PA (2011). "Role of the intestinal bile acid transporters in bile acid and drug disposition". Handb Exp Pharmacol (201): 169–203. doi:10.1007/978-3-642-14541-4_4. PMC 3249407. PMID 21103970.