Intercept Pharmaceuticals Inches Closer to FDA Approval for First-Ever Nash Drug

The first-ever NASH drug may be close to being approved by the Food and Drug Administration and it belongs to Intercept Pharmaceuticals.

NASH, also called nonalcoholic steatohepatitis, is a serious liver disease that is caused by the accumulation of fat in the liver. It can lead to liver failure, cancer, and even death. It is one of the leading causes of liver transplants.

The pharma giant announced recently that a potential therapy for the fatty liver disease known as NASH had achieved its primary goal in a Phase 3 trial.

This news came just a week after Gilead Sciences Inc. announced a competing trial had failed.

Currently there are no approved treatments for NASH.

The company’s treatment, obeticholic acid (OCA), is meant to treat patients with liver fibrosis due to NASH.

In a Phase 3 trial that enrolled 931 patients with liver fibrosis who were randomly assigned to be treated with a placebo drug or one of two doses of OCA, Intercept found that after 18 months, the results were in: 23.1% of patients on the higher OCA dose showed a statistically significant reduction in liver fibrosis, compared to 17.6% of those on the lower dose and 11.9% of those on the placebo, achieving one of the two primary endpoints of the trial.

An agreement with the FDA stipulated that the company’s trial had to meet just one of its two primary goals to be considered successful. While patients on OCA showed a reduction in liver fat and inflammation compared to patients on placebo, the difference was not statistically significant. Thus the company failed to meet its secondary endpoint.

“I am very encouraged by these results that demonstrate OCA’s ability to significantly improve fibrosis in patients with advanced disease,” remarked Zobair M. Younossi, chair of the trial’s steering committee and professor at the Department of Medicine at Inova Fairfax Medical Campus. The company intends to file for approval in the U.S. and Europe in the second half of 2019.