Details
| Stereochemistry | ABSOLUTE |
| Molecular Formula | C40H56N3O4S |
| Molecular Weight | 674.955 |
| Optical Activity | UNSPECIFIED |
| Defined Stereocenters | 2 / 2 |
| E/Z Centers | 0 |
| Charge | 1 |
SHOW SMILES / InChI
SMILES
CCCCC1(CCCC)CS(=O)(=O)C2=CC=C(C=C2[C@H]([C@H]1O)C3=CC=C(OCC4=CC=C(C[N+]56CCN(CC5)CC6)C=C4)C=C3)N(C)C
InChI
InChIKey=STPKWKPURVSAJF-LJEWAXOPSA-N
InChI=1S/C40H56N3O4S/c1-5-7-19-40(20-8-6-2)30-48(45,46)37-18-15-34(41(3)4)27-36(37)38(39(40)44)33-13-16-35(17-14-33)47-29-32-11-9-31(10-12-32)28-43-24-21-42(22-25-43)23-26-43/h9-18,27,38-39,44H,5-8,19-26,28-30H2,1-4H3/q+1/t38-,39-/m1/s1
Maralixibat (SHP625; formerly LUM001 or lopixibat) is a potent, apical, sodium‐dependent, bile acid transporter competitive inhibitor with minimal systemic absorption. Maralixibat works by reducing systemic levels of bile acids. In animal models of cholestasis, maralixibat blocked reabsorption of bile acids in the terminal ileum, thereby reducing enterohepatic recirculation to the liver and increasing fecal excretion of bile acids. Maralixibat is being evaluated as a treatment for children with rare cholestatic liver diseases, including Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC).
Originator
Approval Year
PubMed
| Title | Date | PubMed |
|---|---|---|
| A Drug Regimen for Progressive Familial Cholestasis Type 2. | 2018 Jan |
|
| Placebo-Controlled Randomized Trial of an Intestinal Bile Salt Transport Inhibitor for Pruritus in Alagille Syndrome. | 2018 Oct |
|
| A Randomized, Controlled, Phase 2 Study of Maralixibat in the Treatment of Itching Associated With Primary Biliary Cholangitis. | 2019 Mar |
Sample Use Guides
Phase 3 study to evaluate the efficacy and safety of maralixibat in subjects with progressive familial intrahepatic cholestasis. Participants will be randomized to Maralixibat oral solution (up to 600 microgram per kilogram [mcg/kg]) orally twice daily for 26 weeks.
Route of Administration:
Oral
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FDA ORPHAN DRUG |
406013
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FDA ORPHAN DRUG |
705519
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SUB195692
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DTXSID001337103
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C170148
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UYB6UOF69L
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UYB6UOF69L
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9831643
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100000181871
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2571074
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CD-112
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716313-53-0
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ACTIVE MOIETY
SALT/SOLVATE (PARENT)
