期刊
DRUGS OF THE FUTURE
卷 35, 期 8, 页码 613-620出版社
PROUS SCIENCE, SAU-THOMSON REUTERS
DOI: 10.1358/dof.2010.035.08.1505566
关键词
Genz-112638
资金
- NIAMS NIH HHS [R01 AR056991-04, R01 AR056991] Funding Source: Medline
- NIDDK NIH HHS [R01 DK055823, R01 DK055823-11] Funding Source: Medline
Eliglustat tartrate (Genz-112638) is a novel, orally administered agent currently in development for the treatment of lysosomal storage disorders, including type 1 Gaucher disease and Fabry disease. This glucosylceramide analogue acts as an inhibitor of glucosylceramide synthase, a Golgi complex enzyme that catalyzes the formation of glucosylceramide from ceramide and UDP-glucose and is the first step in the formation of glucocerebroside-based glycosphingolipids. Preclinical pharmacological studies demonstrate that the agent has a high therapeutic index, excellent oral bioavailability and limited toxicity. Phase I studies in healthy volunteers revealed limited toxicity with an excellent pharmacodynamic response, as measured by decreased plasma glucosylceramide concentrations. Phase II studies in patients with type 1 Gaucher disease have demonstrated promising clinical responses, as measured by decreases in spleen size, improvement in hemoglobin concentrations and increased platelet counts. Two randomized phase Ill trials testing the efficacy and safety of eliglustat tartrate are currently in progress.
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