Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of Oral Venglustat in Healthy Volunteers

• Published in: Clinical pharmacology in drug development Vol. 10; no. 1; pp. 86 - 98
• Main Authors: Peterschmitt, M. Judith, Crawford, Nigel P. S., Gaemers, Sebastiaan J. M., Ji, Allena J., Sharma, Jyoti, Pham, Theresa T.
• Format: Journal Article
• Language: English
• Published: United States Wiley Subscription Services, Inc 01.01.2021; John Wiley and Sons Inc
• Subjects: Administration, Oral; Adolescent; Adult; Carbamates - administration & dosage; Carbamates - adverse effects; Carbamates - pharmacokinetics; Cross-Over Studies; Disease; Double-Blind Method; Enzyme Inhibitors - adverse effects; Enzyme Inhibitors - pharmacokinetics; Enzyme Inhibitors - pharmacology; Female; Gangliosides - blood; globotriaosylceramide (GL‐3); glucosylceramide (GL‐1); glucosylceramide synthase (GCS) inhibition; Glucosylceramides - blood; glucosylsphingosine (lyso‐GL‐1); Glucosyltransferases - antagonists & inhibitors; Healthy Volunteers; Humans; Kidney diseases; Male; Middle Aged; monosialodihexosylganglioside (GM3); Original Manuscript; Pharmacodynamics; Pharmacokinetics; Quinuclidines - administration & dosage; Quinuclidines - adverse effects; Quinuclidines - pharmacokinetics; substrate reduction therapy; venglustat (GZ/SAR402671; Genz‐682452); Young Adult; glucosylceramide (GL-1); glucosylceramide synthase (GCS) inhibition; venglustat (GZ/SAR402671; Genz-682452); substrate reduction therapy; globotriaosylceramide (GL-3); glucosylsphingosine (lyso-GL-1); monosialodihexosylganglioside (GM3)

Venglustat is a small‐molecule glucosylceramide synthase (GCS) inhibitor designed to reduce the production of glucosylceramide (GL‐1) and thus is expected to substantially reduce formation of glucosylceramide‐based glycosphingolipids. Because of its effect on glycosphingolipid formation, GCS inhibit...