Nonalcoholic steatohepatitis (NASH) is a severe form of nonalcoholic fatty liver disease (NAFLD) that occurs when excessive amounts of fat build up in the liver, damaging hepatocytes and causing the liver to become inflamed. Together, this can lead to fibrosis, the buildup of scar tissue that hinders the liver’s ability to function properly.
For patients with advanced fibrosis due to NASH, halting progression to cirrhosis is critical. After approximately 2.5 years, ~1 in 5 patients with advanced fibrosis due to NASH may progress to cirrhosis.*
Historically, the standard test to confirm the diagnosis of advanced fibrosis due to NASH has been a liver biopsy, in which a small sample of the liver is removed and examined. However, in addition to being a costly procedure, liver biopsy is associated with numerous limitations including the potential for inconsistent findings, pain, discomfort and the risk of rare but life-threatening complications. Today, noninvasive tests (NITs) play a much larger role in management of the disease: a large U.S. claims database showed that less than 10% of all NASH patients had a biopsy. Professional societies and regulatory authorities are encouraging NIT research to help the hepatology community advance patient care.
The treatment of advanced fibrosis due to NASH is currently limited to lifestyle changes and specific treatment of comorbidities. People with advanced fibrosis due to NASH are advised to make dietary changes and exercise to lose weight, and to avoid heavy alcohol consumption.
REGENERATE (Randomized Global Phase 3 Study to Evaluate the Impact on NASH with Fibrosis of Obeticholic Acid Treatment) is a randomized, double-blind, placebo-controlled, multicenter trial. It is assessing the safety and potential benefit of OCA on liver-related clinical outcomes in NASH patients with fibrosis. Read more about REGENERATE here.
REVERSE (Randomized Phase 3 Study Evaluating the Efficacy and Safety of Obeticholic Acid in Subjects with Compensated Cirrhosis Due to Nonalcoholic Steatohepatitis) is a randomized, double-blind, placebo-controlled, multicenter trial. It is evaluating the safety and efficacy of OCA in NASH patients with compensated cirrhosis. For more information, visit ClinicalTrials.gov (Identifier: NCT03439254).
OCA has not been approved for the treatment of NASH by any regulatory authority in any geography and is considered an investigational treatment for this indication.
*In a study of patients with biopsy-proven NASH, 48 of 217 patients (22%) with advanced fibrosis without cirrhosis progressed to advanced fibrosis with cirrhosis at around 29 months.