New FDA Draft Guidance for Industry: Nonalcoholic Steatohepatitis with Compensated Cirrhosis: Developing Drugs for Treatment

New FDA Draft Guidance for Industry: Nonalcoholic Steatohepatitis with Compensated Cirrhosis: Developing Drugs for Treatment

Today the U.S. Food and Drug Administration (FDA) issued the draft guidance “Nonalcoholic Steatohepatitis with Compensated Cirrhosis: Developing Drugs for Treatment.” The purpose of this draft guidance is to provide the FDA’s current recommendations regarding the important components of a drug development program for nonalcoholic steatohepatitis (NASH) with compensated cirrhosis. This guidance focuses on clinical outcome trials, specifically on how to evaluate whether newly developed treatments can delay or reduce the frequency of decompensation (organ failure) events and other severe outcomes.

NASH is a clinical condition best described by histological findings including liver inflammation, hepatocyte damage and later fibrosis. Although patients have excessive fat accumulation in the liver (hepatic steatosis), most patients have a benign course, and only a minority of patients, who are not easily identifiable, develop NASH. NASH is not associated with alcohol use; rather NASH is associated with type 2 diabetes, hypertension, hypertriglyceridemia (high triglycerides) and obesity, among other diseases. NASH-related cirrhosis is becoming a major public health problem and is anticipated to be the leading indication for liver transplantation within a decade. Currently, no standard treatment exists for NASH.

This guidance accompanies a draft guidance published in 2018 which discusses the agency’s thinking for the design of clinical trials for patients who have NASH but do not have cirrhosis titled “Noncirrhotic Nonalcoholic Steatohepatitis with Liver Fibrosis: Developing Drugs for Treatment.”

FDA looks forward to receiving comments on this draft guidance.