The FDA May Be Close to Approving Intercept Pharmaceuticals’ NASH Drug

The first-ever NASH drug may be around the corner from being approved by the Food and Drug Administration.

Intercept Pharmaceuticals announced recently that a potential therapy for the fatty liver disease known as NASH had achieved its primary goal in a Phase 3 trial. The news follows Gilead Sciences Inc. announcing that a competing trial had failed.

Currently there are no approved treatments for NASH. NASH, also called nonalcoholic steatohepatitis, is a serious liver disease that is caused by the accumulation of fat in the liver that can lead to liver failure and even death.
Intercept Pharmaceuticals’ 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, the company discovered 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. The company had failed the other endpoint but 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.

“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.

Disclaimer: We have no position in Intercept Pharmaceuticals Inc. (NASDAQ: ICPT) and have not been compensated for this article.