Draft NASH Guidance Out

FDA has published a draft guidance, Nonalcoholic Steatohepatitis with Compensated Cirrhosis: Developing Drugs for Treatment, to assist sponsors in the clinical development of drugs to treat nonalcoholic steatohepatitis (NASH) with compensated cirrhosis. The document contains the agency’s current recommendations on the important components of a drug development program for compensated NASH cirrhosis. FDA says it focuses on the enrollment criteria, trial design, efficacy endpoints, and safety considerations for Phase 3 trials. It also identifies knowledge gaps that represent important challenges in developing drugs for this indication.

The guidance does not address the clinical development of drugs for treating decompensated cirrhosis resulting from NASH. And it does not address drug development for patients with noncirrhotic NASH or provide general recommendations on early drug development in NASH, such as use of animal models and approaches to monitoring for potential liver toxicity.