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
Molecular Formula | C40H55N3O4S |
Molecular Weight | 673.947 |
Charge | 0 |
Count |
|
Stereochemistry | EPIMERIC |
Additional Stereochemistry | No |
Defined Stereocenters | 2 / 2 |
E/Z Centers | 0 |
Optical Activity | UNSPECIFIED |
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
In Vivo Use Guide
Sources: https://clinicaltrials.gov/ct2/show/NCT03905330
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
Substance Class |
Chemical
Created
by
admin
on
Edited
Sat Dec 16 08:35:56 GMT 2023
by
admin
on
Sat Dec 16 08:35:56 GMT 2023
|
Record UNII |
UYB6UOF69L
|
Record Status |
Validated (UNII)
|
Record Version |
|
-
Download
Name | Type | Language | ||
---|---|---|---|---|
|
Official Name | English | ||
|
Code | English | ||
|
Common Name | English | ||
|
Common Name | English | ||
|
Code | English | ||
|
Common Name | English | ||
|
Common Name | English | ||
|
Systematic Name | English |
Classification Tree | Code System | Code | ||
---|---|---|---|---|
|
FDA ORPHAN DRUG |
406013
Created by
admin on Sat Dec 16 08:35:56 GMT 2023 , Edited by admin on Sat Dec 16 08:35:56 GMT 2023
|
||
|
FDA ORPHAN DRUG |
705519
Created by
admin on Sat Dec 16 08:35:56 GMT 2023 , Edited by admin on Sat Dec 16 08:35:56 GMT 2023
|
Code System | Code | Type | Description | ||
---|---|---|---|---|---|
|
SUB195692
Created by
admin on Sat Dec 16 08:35:56 GMT 2023 , Edited by admin on Sat Dec 16 08:35:56 GMT 2023
|
PRIMARY | |||
|
DTXSID001337103
Created by
admin on Sat Dec 16 08:35:56 GMT 2023 , Edited by admin on Sat Dec 16 08:35:56 GMT 2023
|
PRIMARY | |||
|
C170148
Created by
admin on Sat Dec 16 08:35:56 GMT 2023 , Edited by admin on Sat Dec 16 08:35:56 GMT 2023
|
PRIMARY | |||
|
UYB6UOF69L
Created by
admin on Sat Dec 16 08:35:56 GMT 2023 , Edited by admin on Sat Dec 16 08:35:56 GMT 2023
|
PRIMARY | |||
|
UYB6UOF69L
Created by
admin on Sat Dec 16 08:35:56 GMT 2023 , Edited by admin on Sat Dec 16 08:35:56 GMT 2023
|
PRIMARY | |||
|
9831643
Created by
admin on Sat Dec 16 08:35:56 GMT 2023 , Edited by admin on Sat Dec 16 08:35:56 GMT 2023
|
PRIMARY | |||
|
100000181871
Created by
admin on Sat Dec 16 08:35:56 GMT 2023 , Edited by admin on Sat Dec 16 08:35:56 GMT 2023
|
PRIMARY | |||
|
2571074
Created by
admin on Sat Dec 16 08:35:56 GMT 2023 , Edited by admin on Sat Dec 16 08:35:56 GMT 2023
|
PRIMARY | |||
|
CD-112
Created by
admin on Sat Dec 16 08:35:56 GMT 2023 , Edited by admin on Sat Dec 16 08:35:56 GMT 2023
|
PRIMARY | |||
|
716313-53-0
Created by
admin on Sat Dec 16 08:35:56 GMT 2023 , Edited by admin on Sat Dec 16 08:35:56 GMT 2023
|
PRIMARY |
Related Record | Type | Details | ||
---|---|---|---|---|
|
SALT/SOLVATE -> PARENT |
|
||
|
TARGET -> INHIBITOR |
|
Related Record | Type | Details | ||
---|---|---|---|---|
|
ACTIVE MOIETY |
|