Eliglustat

Eliglustat

Systematic (IUPAC) name
N-[(1R,2R)-1-(2,3-Dihydro-1,4-benzodioxin-6-yl)-1-hydroxy-3-(1-pyrrolidinyl)-2-propanyl]octanamide
Clinical data
Trade names	Cerdelga
Legal status
Legal status	US: ℞-only
Identifiers
CAS Number	491833-29-5
ATC code	A16AX10 (WHO)
PubChem	CID 23652731
ChemSpider	28475348
ChEBI	CHEBI:82752
Chemical data
Formula	C23H36N2O4
Molar mass	404.543 g/mol
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 (INN, USAN;^[1] trade name Cerdelga) is a treatment for Gaucher's disease developed by Genzyme Corp that was approved by the FDA August 2014.^[2] Commonly used as the tartrate salt, the compound is believed to work by inhibition of glucosylceramide synthase.^[3][4] 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.^[5]

Cost

In 2014, the annual cost of Cerdelga hard gelatin capsules taken orally twice a day was $310,250. Genzyme’s flagship Imiglucerase (brand name Cerezyme) cost about $300,000 for the infusions if taken twice a month.^[6] Manufacturing costs for Cerdelga are slightly lower than for Cerezyme. Genzyme's maintains higher prices for orphan drugs—most often paid for by insurers— in order to remain financially sustainable.^[6]

References

1. Eligustat (PDF), AMA By subscription only
2. FDA approves new drug to treat a form of Gaucher disease, U.S. Food and Drug Administration, 19 August 2015, retrieved 18 July 2015
3. Lee, L.; Abe, A.; Shayman, J. A. (21 May 1999). "Improved Inhibitors of Glucosylceramide Synthase". Journal of Biological Chemistry 274 (21): 14662–14669. doi:10.1074/jbc.274.21.14662.
4. Shayman, JA (1 August 2010). "Eliglustat Tartrate: Glucosylceramide Synthase Inhibitor Treatment of Type 1 Gaucher Disease.". Drugs of the future 35 (8): 613–620. PMID 22563139.
5. Pramod K. Mistry, Elena Lukina, Hadhami Ben Turkia, Dominick Amato, Hagit Baris, Majed Dasouki, Marwan Ghosn, Atul Mehta, Seymour Packman, Gregory Pastores, Milan Petakov, Sarit Assouline, Manisha Balwani, Sumita Danda, Evgueniy Hadjiev, Andres Ortega, Suma Shankar, Maria Helena Solano, Leorah Ross, Jennifer Angell, M. Judith Peterschmitt (17 February 2015), "Effect of Oral Eliglustat on Splenomegaly in Patients With Gaucher Disease Type 1: The ENGAGE Randomized Clinical Trial", Journal of the American Medical Association 313 (7): 695–706, doi:10.1001/jama.2015.459
6. Robert Weisman (2 September 2014), New Genzyme pill will cost patients $310,250 a year, The Boston Globe, retrieved 18 July 2015