Eliglustat

Eliglustat (INN, USAN;[1] trade name Cerdelga) is a treatment for Gaucher's disease discovered at the University of Michigan and 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]

Eliglustat

Clinical data
Trade names	Cerdelga
License data	EU EMA: by INN
ATC code	A16AX10 (WHO)
Legal status
Legal status	US: ℞-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
ChemSpider	28475348
UNII	DR40J4WA67
KEGG	D09893
ChEBI	CHEBI:82752^Y
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

Cost

In 2014, the annual cost of eliglustat taken orally twice a day was $310,250. Genzyme's flagship imiglucerase (brand name Cerezyme) cost about $300,000 for the intravenous medication if taken twice a month.[6] Manufacturing costs for eliglustat are slightly lower than for imiglucerase. Genzyme 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 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.
4. 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.
5. 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.
6. Weisman R (2 September 2014), New Genzyme pill will cost patients $310,250 a year, The Boston Globe, retrieved 18 July 2015