Major developments are unfolding in the treatment to tackle non-alcoholic steatohepatitis (NASH), a rapidly growing and costly disease affecting millions of adults in the United States. In the absence of an FDA-approved treatment, NASH patients face elevated risks of liver fibrosis, cardiovascular disease and liver cancer.
Until recently, there were two investigational medications for NASH in late-stage development. However, in June, the FDA rejected Intercept Pharmaceutical’s second attempt at approval of obeticholic acid (OCA) for the treatment of NASH with stage 2 or 3 fibrosis. Intercept's clinical trial data demonstrated only a moderate benefit of OCA as compared to placebo in improving fibrosis in NASH patients and there were notable safety concerns. In response to the second FDA rejection of OCA, Intercept has decided to discontinue all NASH-related investments. OCA will continue to be available as Ocalvia® and is FDA approved to treat primary biliary cholangitis (PBC). As a result of OCA’s FDA rejection, resmetirom (MGL-3196), from Madrigal Pharmaceuticals, now has the potential to be the first approved therapy for NASH.
Resmetirom is an investigational medication designed to address the underlying causes of NASH. Positive results from the Phase 3 MAESTRO-NASH trial demonstrate resmetirom's potential, with significant improvements in NASH resolution and fibrosis reduction. If granted priority review, resmetirom could be approved as early as Q1 2024.
But that's not all: the combination therapy of efruxiferim and Ozempic is showing promise. Efruxiferim, an investigational fibroblast growth factor, has demonstrated its effectiveness in treating NASH, and when combined with Ozempic—an FDA-approved medication for type 2 diabetes—a remarkable 65% relative reduction in liver fat was observed. Furthermore, this combination therapy showed improvements in liver enzymes, non-invasive fibrosis markers and glycemic control.
As the NASH patient population continues to rise, these advancements come as a beacon of hope, offering potential relief from the burdensome clinical and economic impacts of NASH.
Stay tuned for the arrival of resmetirom—the first approved therapy for NASH—and the ongoing development of innovative treatment strategies for this complex disease.
To learn more about NASH and current treatments, click here to read our clinical bulletin.