Eliglustat tartrateProduct ingredient for Eliglustat

Name
Eliglustat tartrate
Drug Entry
Eliglustat

Eliglustat is a glucosylceramide synthase inhibitor used for the long-term treatment of type 1 Gaucher disease.2,6 Gaucher disease is a rare genetic disorder characterized by the deficiency of acid β-glucosidase, an enzyme that converts glucosylceramide into glucose and ceramide. In patients with Gaucher disease, the accumulation of glucosylceramide leads to the formation of Gaucher cells that infiltrate the liver, spleen, bone marrow and other organs. This leads to complications such as anemia and thrombocytopenia.6,4 By inhibiting glucosylceramide synthase, eliglustat reduces the accumulation of glucosylceramide.6

Eliglustat is mainly metabolized by CYP2D6.6 Patients selected for eliglustat treatment undergo an FDA-cleared genotyping test to establish if they are CYP2D6 extensive metabolizers (EMs), intermediate metabolizers (IMs), or poor metabolizers (PMs). The results of this test dictate eliglustat dosing recommendations for each type of patient. There are no dosing recommendations for CYP2D6 ultra-rapid or indeterminate metabolizers.6,1 Eliglustat was approved by the FDA in August 2014 as an oral substrate reduction therapy for the first-line treatment of type 1 Gaucher disease.6,1 Enzyme replacement continues to be the standard of care for the treatment of type 1 Gaucher disease (imiglucerase, velaglucerase alfa, taliglucerase alfa); however, oral substrate reduction therapies with favourable safety profiles, such as eliglustat, represent a treatment alternative.5,1

Accession Number
DBSALT001106
Structure
Synonyms
Eliglustat tartrate
External IDs
GENZ 112638 / GENZ-112638
UNII
N0493335P3
CAS Number
928659-70-5
Weight
Average: 959.188
Monoisotopic: 958.551453208
Chemical Formula
C50H78N4O14
InChI Key
KUBARPMUNHKBIQ-VTHUDJRQSA-N
InChI
InChI=1S/2C23H36N2O4.C4H6O6/c2*1-2-3-4-5-6-9-22(26)24-19(17-25-12-7-8-13-25)23(27)18-10-11-20-21(16-18)29-15-14-28-20;5-1(3(7)8)2(6)4(9)10/h2*10-11,16,19,23,27H,2-9,12-15,17H2,1H3,(H,24,26);1-2,5-6H,(H,7,8)(H,9,10)/t2*19-,23-;1-,2-/m111/s1
IUPAC Name
(2R,3R)-2,3-dihydroxybutanedioic acid; bis(N-[(1R,2R)-1-(2,3-dihydro-1,4-benzodioxin-6-yl)-1-hydroxy-3-(pyrrolidin-1-yl)propan-2-yl]octanamide)
SMILES
O[C@H]([C@@H](O)C(O)=O)C(O)=O.CCCCCCCC(=O)N[C@H](CN1CCCC1)[C@H](O)C1=CC=C2OCCOC2=C1.CCCCCCCC(=O)N[C@H](CN1CCCC1)[C@H](O)C1=CC=C2OCCOC2=C1
ChemSpider
30790872
ChEBI
83353
Wikipedia
Eliglustat
Predicted Properties
PropertyValueSource
Water Solubility0.113 mg/mLALOGPS
logP3.44ALOGPS
logP3.21Chemaxon
logS-3.6ALOGPS
pKa (Strongest Acidic)13.51Chemaxon
pKa (Strongest Basic)8.17Chemaxon
Physiological Charge1Chemaxon
Hydrogen Acceptor Count5Chemaxon
Hydrogen Donor Count2Chemaxon
Polar Surface Area71.03 Å2Chemaxon
Rotatable Bond Count25Chemaxon
Refractivity113.76 m3·mol-1Chemaxon
Polarizability46.82 Å3Chemaxon
Number of Rings6Chemaxon
Bioavailability0Chemaxon
Rule of FiveNoChemaxon
Ghose FilterNoChemaxon
Veber's RuleNoChemaxon
MDDR-like RuleYesChemaxon