Trade names Cerdelga Legal status US: ℞-only PubChem CID 23652731 Formula C23H36N2O4 | ATC code A16AX10 (WHO) CAS Number 491833-29-5 ChemSpider 28475348 Molar mass 404.543 g/mol | |
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Cerdelga eliglustat helps gaucher type 3 patient
Eliglustat (INN, USAN; trade name Cerdelga) is a treatment for Gaucher's disease developed by Genzyme Corp that was approved by the FDA 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.
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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 infusions if taken twice a month. Manufacturing costs for eliglustat are slightly lower than for imiglucerase. Genzyme's maintains higher prices for orphan drugs —most often paid for by insurers— in order to remain financially sustainable.