Lucerastat

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 Search

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

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Description
Sources: https://fabrydiseasenews.com/lucerastat/ | https://www.ncbi.nlm.nih.gov/pubmed/7929454 | https://www.ncbi.nlm.nih.gov/pubmed/29982630

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.

Originator

Approval Year

PubMed

Title	Date	PubMed
N-butyldeoxygalactonojirimycin inhibits glycolipid biosynthesis but does not affect N-linked oligosaccharide processing.	1994 Oct 28	7929454
Structure-activity relationship of a new class of anti-hepatitis B virus agents.	2002 Dec	12435712

Patents

Sample Use Guides

In Vivo Use Guide

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

Name

Type

Language

Lucerastat

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