Ocaliva, Azelaprag, And The Continuing Challenge Of Drug-Induced Liver Injury: A Call For Human-Relevant Models

Drug-induced liver injury remains a major threat to patient safety and drug development. Recent liver safety concerns involving Ocaliva and the obesity drug candidate azelaprag demonstrate how toxicity can emerge even after conventional preclinical testing. Such setbacks can halt clinical trials, delay promising therapies, and expose patients to serious risks, including liver transplantation and death. They also reveal the limitations of animal studies and 2D cell cultures, which do not always predict how the human liver will respond to a drug. More human-relevant preclinical approaches, including Organ-on-a-Chip technology, could help researchers identify liver toxicity earlier and make better-informed development decisions.

Examine why improving the prediction of drug-induced liver injury is critical to advancing safer therapies.