Eliglustat: A Review in Gaucher Disease Type 1.
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Scott LJ
Eliglustat: A Review in Gaucher Disease Type 1.
Drugs. 2015 Sep;75(14):1669-78. doi: 10.1007/s40265-015-0468-9.
- PubMed ID
- 26384672 [ View in PubMed]
- Abstract
Oral eliglustat (Cerdelga(R)) is approved in several countries for the long-term treatment of adults with Gaucher disease type 1 (GD1) who are cytochrome P450 (CYP) 2D6 extensive metabolizers (EMs), intermediate metabolizer (IMs) or poor metabolizers (PMs) [these three CYP categories encompass >90 % of individuals]. Eliglustat is a potent, selective inhibitor of glucosylceramide synthase, the rate-limiting enzyme in the synthesis of certain glycosphingolipids, and thus, reduces the rate of biosynthesis of glycosphingolipids to counteract the catabolic defect (i.e. substrate reduction therapy). In the 9-month phase 3 ENGAGE trial, eliglustat significantly improved haematological endpoints and reduced organomegaly compared with placebo in treatment-naive adults with GD1, with the bone marrow burden score (a marker of Gaucher cell infiltration) and GD1 biomarkers also improving from baseline in eliglustat recipients. After 12 months in the phase 3 ENCORE trial, oral eliglustat was noninferior to intravenous imiglucerase [an enzyme replacement therapy (ERT)] in maintaining disease stability in adults who had stable disease after receiving ERT for >/=3 years. During long-term treatment with eliglustat (
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- Drugs
- Drug Interactions
Drugs Interaction Integrate drug-drug
interactions in your softwareEliglustatCholecalciferol The metabolism of Eliglustat can be decreased when combined with Cholecalciferol. EliglustatAbiraterone The metabolism of Eliglustat can be decreased when combined with Abiraterone. EliglustatLumefantrine The metabolism of Eliglustat can be decreased when combined with Lumefantrine. EliglustatSulfaphenazole The metabolism of Eliglustat can be decreased when combined with Sulfaphenazole. EliglustatPhenylbutyric acid The metabolism of Eliglustat can be decreased when combined with Phenylbutyric acid.