Clinical Liver Injury

FEASIBILITY1 SCOPING2 RESEARCH3 SUBMITTED4 QUALIFIED5

blue U.S. Food & Drug Administration (FDA)

green European Medicines Agency (EMA)

Overview

Drug-induced liver injury (DILI) is one of the most frequent adverse events and can sometimes result in liver failure. Rare (e.g. < 1/10,000) liver safety events have resulted in the withdrawal of drugs from the market that were efficacious for the overwhelming majority of patients. Identifying which patients might be susceptible or succumb to DILI is an active and complex area of research. PSTC is focused specifically on biomarkers that would predict early in the course of therapeutic treatment (i.e. earlier than meeting a Hy's Law definition) whether a patient might progress to serious liver injury or adapt, and thus safely continue with therapeutic treatment. These biomarkers would be enormously useful in deciding whether to continue or alter the course of treatment, while simultaneously improving patient safety and enabling continued therapy for patients in need.

  • First set: GLDH, MDH, SDH, GST-alpha, ARG-1, miR122