eliglustat and Liver-Diseases

Eliglustat is a first-line oral therapy for adults with Gaucher disease type 1 (GD1) who have extensive (EM), intermediate (IM), or poor (PM) CYP2D6 metabolizer phenotypes. It was initially not recommended in GD1 patients with hepatic or renal impairment due to insufficient data. Two Phase 1 studies (NCT02536937/NCT02536911) evaluated the effects of hepatic and renal impairment on pharmacokinetics and tolerability following a single 84-mg dose of eliglustat. Compared to matched healthy EM subjects (n = 7 for both studies), geometric means for eliglustat maximum concentration (C

Topics: Administration, Oral; Adolescent; Adult; Aged; Drug Tolerance; Female; Gaucher Disease; Humans; Kidney; Liver; Liver Diseases; Male; Middle Aged; Pyrrolidines; Renal Insufficiency; Young Adult