U.S. Department of Health & Human Services Divider Arrow National Institutes of Health Divider Arrow NCATS

Details

Stereochemistry ABSOLUTE
Molecular Formula C10H21NO4
Molecular Weight 219.278
Optical Activity ( - )
Defined Stereocenters 4 / 4
E/Z Centers 0
Charge 0

SHOW SMILES / InChI
Structure of Lucerastat

SMILES

CCCCN1C[C@H](O)[C@@H](O)[C@@H](O)[C@H]1CO

InChI

InChIKey=UQRORFVVSGFNRO-XFWSIPNHSA-N
InChI=1S/C10H21NO4/c1-2-3-4-11-5-8(13)10(15)9(14)7(11)6-12/h7-10,12-15H,2-6H2,1H3/t7-,8+,9+,10-/m1/s1

HIDE SMILES / InChI

Molecular Formula C10H21NO4
Molecular Weight 219.278
Charge 0
Count
Stereochemistry ABSOLUTE
Additional Stereochemistry No
Defined Stereocenters 4 / 4
E/Z Centers 0
Optical Activity UNSPECIFIED

Lucerastat inhibits glycolipid biosynthesis. Lucerastat is an orally bioavailable inhibitor of glucosylceramide synthase. Lucerastat is being developed by the biopharmaceutical company Idorsia for the treatment of Fabry disease.

Approval Year

PubMed

PubMed

TitleDatePubMed
N-butyldeoxygalactonojirimycin inhibits glycolipid biosynthesis but does not affect N-linked oligosaccharide processing.
1994 Oct 28
Structure-activity relationship of a new class of anti-hepatitis B virus agents.
2002 Dec
Patents

Sample Use Guides

Phase 3 study aimed to determine the efficacy and safety of lucerastat oral monotherapy in adult subjects with Fabry disease. Hard gelatin capsules containing 250 mg of lucerastat and inactive excipients; 1000 mg (4 capsules) b.i.d.; dose adjusted for renal function.
Route of Administration: Oral
Substance Class Chemical
Created
by admin
on Sat Dec 16 01:40:57 GMT 2023
Edited
by admin
on Sat Dec 16 01:40:57 GMT 2023
Record UNII
GVS3YDM418
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
Lucerastat
INN   USAN  
Official Name English
LUCERASTAT [USAN]
Common Name English
(2R,3S,4R,5S)-1-Butyl-2-(hydroxymethyl)-3,4,5-piperidinetriol
Systematic Name English
LUCERASTAT [JAN]
Common Name English
lucerastat [INN]
Common Name English
ACT-434964
Code English
N-Butyldeoxygalactonojirimycin
Common Name English
3,4,5-Piperidinetriol, 1-butyl-2-(hydroxymethyl)-, (2R,3S,4R,5S)-
Systematic Name English
Classification Tree Code System Code
FDA ORPHAN DRUG 495815
Created by admin on Sat Dec 16 01:40:57 GMT 2023 , Edited by admin on Sat Dec 16 01:40:57 GMT 2023
EU-Orphan Drug EU/3/12/1033
Created by admin on Sat Dec 16 01:40:57 GMT 2023 , Edited by admin on Sat Dec 16 01:40:57 GMT 2023
Code System Code Type Description
EPA CompTox
DTXSID60161601
Created by admin on Sat Dec 16 01:40:57 GMT 2023 , Edited by admin on Sat Dec 16 01:40:57 GMT 2023
PRIMARY
SMS_ID
100000177224
Created by admin on Sat Dec 16 01:40:57 GMT 2023 , Edited by admin on Sat Dec 16 01:40:57 GMT 2023
PRIMARY
INN
9539
Created by admin on Sat Dec 16 01:40:57 GMT 2023 , Edited by admin on Sat Dec 16 01:40:57 GMT 2023
PRIMARY
DRUG BANK
DB14872
Created by admin on Sat Dec 16 01:40:57 GMT 2023 , Edited by admin on Sat Dec 16 01:40:57 GMT 2023
PRIMARY
PUBCHEM
501391
Created by admin on Sat Dec 16 01:40:57 GMT 2023 , Edited by admin on Sat Dec 16 01:40:57 GMT 2023
PRIMARY
FDA UNII
GVS3YDM418
Created by admin on Sat Dec 16 01:40:57 GMT 2023 , Edited by admin on Sat Dec 16 01:40:57 GMT 2023
PRIMARY
USAN
GH-14
Created by admin on Sat Dec 16 01:40:57 GMT 2023 , Edited by admin on Sat Dec 16 01:40:57 GMT 2023
PRIMARY
CAS
141206-42-0
Created by admin on Sat Dec 16 01:40:57 GMT 2023 , Edited by admin on Sat Dec 16 01:40:57 GMT 2023
PRIMARY
NCI_THESAURUS
C170135
Created by admin on Sat Dec 16 01:40:57 GMT 2023 , Edited by admin on Sat Dec 16 01:40:57 GMT 2023
PRIMARY
Related Record Type Details
TARGET -> INHIBITOR
Related Record Type Details
ACTIVE MOIETY