There is significant unmet medical need in the prevention and treatment of nonalcoholic steatohepatitis (NASH), primary biliary cholangitis, and primary sclerosing cholangitis. Despite its increasing prevalence and potential to cause severe liver-related morbidity (including liver cancer) and mortality, there are currently no drugs approved for the treatment of NASH. Recognizing that the complex etiologies, natural histories, and pathophysiologies of NASH and other cholestatic liver diseases are major impediments to ongoing drug development, the U.S. Food and Drug Administration (FDA) released two draft guidances to assist industry in the clinical development of drugs for the treatment of NASH with liver fibrosis and with compensated cirrhosis.
To further assist in drug development for NASH and cholestatic liver disease indications, Duke-Margolis and the FDA are convening this public meeting to discuss clinical pharmacology-driven considerations and promising approaches to optimize drug safety and efficacy. Discussion will encompass:
- The impact of liver dysfunction on drug pharmacokinetics, pharmacodynamics, patient safety and patient outcomes
- Emerging biomarkers, including non-invasive biomarkers, and their utility in the early-phase development of drugs for NASH and cholestatic liver disease
- Challenges associated with optimizing clinical trial design, including adequate characterization of pharmacologic effect, selection of study population, and bridging of trial endpoints to clinical meaningfulness
- Clinical pharmacology approaches for optimizing drug development programs as well as the safety and efficacy of new drugs
The views expressed in written meeting materials or publications and by speakers and moderators do not necessarily reflect the official policies of the Department of Health and Human Services nor does mention of trade names, commercial practices, or organizations imply endorsements by the U.S. Government.