.Pharmacolibrary.Drugs.ATC.A.A16AX10 .Pharmacolibrary.Drugs.ATC.A.A16AX10| name: | Eliglustat |
| ATC code: | A16AX10 | route: | oral |
| n-compartments | 2 |
Eliglustat is an oral substrate reduction therapy used for the long-term treatment of adults with Gaucher disease type 1, a lysosomal storage disorder. It inhibits glucosylceramide synthase, reducing the production of glycosphingolipids which accumulate in the disease. Eliglustat is approved and in current use for this indication.
Pharmacokinetic parameters as observed in healthy adult volunteers after oral administration; population includes both sexes, ages 18-55 years.
Wolthuis, DFGJ, et al., & Ter Heine, R (2025). Model-informed repurposing of eliglustat for treatment and prophylaxis of Shiga toxin-producing Escherichia coli hemolytic-uremic syndrome (STEC-HUS) in children. Pediatric nephrology (Berlin, Germany) 40(6) 2009–2019. DOI:10.1007/s00467-025-06688-3 PUBMED:https://pubmed.ncbi.nlm.nih.gov/39900743