奥贝胆酸疗效好吗？

(Obeticholic acid, OCA, trade name Ocaliva), developed by Intercept, is a farnesoid X receptor-specific agonist. On May 27, 2016, the US FDA conditionally accelerated its approval for the treatment of primary biliary cholangitis, making it the first drug approved for the treatment of primary biliary cholangitis in the past 20 years.

Obeticholic acid (OCA) is a semi-synthetic bile acid analogue. Its mechanism of action is to indirectly inhibit the gene expression of cytochrome 7A1 (CYP7A1, the rate-limiting enzyme for bile acid biosynthesis) by activating farnesoid X receptor (FXR), thereby reducing the production of bile acid and reducing bile damage to the liver. Is obeticholic acid effective?

Results from the REGENERATE trial, a phase 3 trial, studying its investigational new drug obeticholic acid (OCA) versus placebo in the treatment of patients with liver fibrosis caused by nonalcoholic steatohepatitis (NASH). In terms of primary efficacy, a planned 18-month interim analysis showed that OCA 25 mg once daily met the primary endpoint of improvement in fibrosis (≥Grade 1) with statistical significance and no worsening of NASH (p = 0.0002 compared with placebo).

The trial showed that in the primary efficacy analysis, a greater proportion of patients in the treatment group achieved the primary endpoint of NASH resolution without worsening of liver fibrosis compared with placebo, but this did not reach statistical significance. The REGENERATE data tell us that obeticholic acid is expected to be the first approved drug for patients with liver fibrosis caused by NASH. Currently, in the field of research on fibrosis caused by NASH, obeticholic acid is still the only drug under development that has received FDA breakthrough therapy designation.