Eliglustat

Eliglustat
Clinical data
Trade names	Cerdelga
AHFS/Drugs.com	Monograph
MedlinePlus	a618038
License data	US DailyMed: Eliglustat;
Routes of; administration	By mouth
ATC code	A16AX10 (WHO) ;
Legal status
Legal status	AU: S4 (Prescription only); CA: ℞-only; UK: POM (Prescription only); US: ℞-only; EU: Rx-only;
Identifiers
	IUPAC name N-[(1R,2R)-1-(2,3-Dihydro-1,4-benzodioxin-6-yl)-1-hydroxy-3-(1-pyrrolidinyl)-2-propanyl]octanamide;
CAS Number	491833-29-5;
PubChem CID	23652731;
DrugBank	DB09039;
ChemSpider	28475348;
UNII	DR40J4WA67;
KEGG	D09893;
ChEBI	CHEBI:82752 ;
ChEMBL	ChEMBL2110588;
CompTox Dashboard (EPA)	DTXSID20964175 ;
Chemical and physical data
Formula	C23H36N2O4
Molar mass	404.551 g·mol−1
3D model (JSmol)	Interactive image;
	SMILES CCCCCCCC(=O)N[C@H](CN1CCCC1)[C@@H](C2=CC3=C(C=C2)OCCO3)O;
	InChI InChI=1S/C23H36N2O4/c1-2-3-4-5-6-9-22(26)24-19(17-25-12-7-8-13-25)23(27)18-10-11-20-21(16-18)29-15-14-28-20/h10-11,16,19,23,27H,2-9,12-15,17H2,1H3,(H,24,26)/t19-,23-/m1/s1; Key:FJZZPCZKBUKGGU-AUSIDOKSSA-N;

Eliglustat, sold under the brand name Cerdelga, is a medication used for the treatment of Gaucher's disease. It was discovered at the University of Michigan, developed by Genzyme Corp, and was approved by the FDA in August 2014. Commonly used as the tartrate salt, the compound is believed to work by inhibition of glucosylceramide synthase. According to an article in Journal of the American Medical Association the oral substrate reduction therapy resulted in "significant improvements in spleen volume, hemoglobin level, liver volume, and platelet count" in untreated adults with Gaucher disease Type 1.