Eliglustat

Eliglustat (INN, USAN; 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. 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. Eliglustat (INN, USAN; 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. 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. 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. 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.