Eliglustat information

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.^[6] Commonly used as the tartrate salt, the compound is believed to work by inhibition of glucosylceramide synthase.^[7]^[8] 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.^[9]

^ "Prescription medicines: registration of new chemical entities in Australia, 2015". Therapeutic Goods Administration (TGA). 21 June 2022. Retrieved 10 April 2023.
^ "Product monograph brand safety updates". Health Canada. February 2024. Retrieved 24 March 2024.
^ "Genetic disorders". Health Canada. 9 May 2018. Retrieved 13 April 2024.
^ "Cerdelga 84 mg Hard Capsules - Summary of Product Characteristics (SmPC)". (emc). 8 January 2020. Retrieved 30 January 2021.
^ "Cerdelga- eliglustat capsule". DailyMed. Retrieved 30 January 2021.
^ "FDA approves new drug to treat a form of Gaucher disease" (Press release). U.S. Food and Drug Administration. 19 August 2015. Archived from the original on 16 February 2017. Retrieved 18 July 2015.
^ Lee L, Abe A, Shayman JA (May 1999). "Improved inhibitors of glucosylceramide synthase". The Journal of Biological Chemistry. 274 (21): 14662–9. doi:10.1074/jbc.274.21.14662. PMID 10329660.
^ Shayman JA (August 2010). "ELIGLUSTAT TARTRATE: Glucosylceramide Synthase Inhibitor Treatment of Type 1 Gaucher Disease". Drugs of the Future. 35 (8): 613–620. doi:10.1358/dof.2010.035.08.1505566. PMC 3340614. PMID 22563139.
^ Mistry PK, Lukina E, Ben Turkia H, Amato D, Baris H, Dasouki M, et al. (February 2015). "Effect of oral eliglustat on splenomegaly in patients with Gaucher disease type 1: the ENGAGE randomized clinical trial". JAMA. 313 (7): 695–706. doi:10.1001/jama.2015.459. PMC 4962880. PMID 25688781.

[1] "Prescription medicines: registration of new chemical entities in Australia, 2015". Therapeutic Goods Administration (TGA). 21 June 2022. Retrieved 10 April 2023.

[2] "Product monograph brand safety updates". Health Canada. February 2024. Retrieved 24 March 2024.

[3] "Genetic disorders". Health Canada. 9 May 2018. Retrieved 13 April 2024.

[4] "Cerdelga 84 mg Hard Capsules - Summary of Product Characteristics (SmPC)". (emc). 8 January 2020. Retrieved 30 January 2021.

[5] "Cerdelga- eliglustat capsule". DailyMed. Retrieved 30 January 2021.

[6] "FDA approves new drug to treat a form of Gaucher disease" (Press release). U.S. Food and Drug Administration. 19 August 2015. Archived from the original on 16 February 2017. Retrieved 18 July 2015.

[7] Lee L, Abe A, Shayman JA (May 1999). "Improved inhibitors of glucosylceramide synthase". The Journal of Biological Chemistry. 274 (21): 14662–9. doi:10.1074/jbc.274.21.14662. PMID 10329660.

[8] Shayman JA (August 2010). "ELIGLUSTAT TARTRATE: Glucosylceramide Synthase Inhibitor Treatment of Type 1 Gaucher Disease". Drugs of the Future. 35 (8): 613–620. doi:10.1358/dof.2010.035.08.1505566. PMC 3340614. PMID 22563139.

[AMA_2015-9] Mistry PK, Lukina E, Ben Turkia H, Amato D, Baris H, Dasouki M, et al. (February 2015). "Effect of oral eliglustat on splenomegaly in patients with Gaucher disease type 1: the ENGAGE randomized clinical trial". JAMA. 313 (7): 695–706. doi:10.1001/jama.2015.459. PMC 4962880. PMID 25688781.

Eliglustat information

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