Ocaliva (obeticholic acid; Intercept Pharmaceuticals) is a selective farnesoid X receptor (FXR) agonist. FXR is a nuclear bile acid receptor involved in the regulation of bile acids, lipids, and possibly glucose. Ocaliva is a derivative of chenodeoxycholic acid, the endogenous FXR agonist found naturally in human bile acid. By activating FXR, Ocaliva improves insulin sensitivity and inhibits lipogenesis, which reduces fat buildup in the liver and results in decreased inflammation. Ocaliva has also been linked to weight loss. Ocaliva is currently approved in the US and EU for primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA, or as monotherapy in adults unable to tolerate UDCA.
Ocaliva is expected to initially experience rapid uptake in the F2/F3 population, as it will likely be the first drug approved for the treatment of non-alcoholic steatohepatitis (NASH), with Intercept planning to file for approval in the US and EU in the second half of 2019. The regulatory filings will be based on data from the pivotal REGENERATE study, in which a significantly higher percentage of Ocaliva-treated patients (25mg) demonstrated a fibrosis improvement of >1 stage with no worsening of NASH at month 18 than placebo-treated patients. While Ocaliva did not meet the co-primary endpoint of NASH resolution with no worsening of liver fibrosis at month 18, only achieving one of the endpoints was required for approval, and a demonstrated reduction in fibrosis will be sufficient to drive uptake given that the extent of fibrosis is an established predictor of mortality. Moreover, Ocaliva has the advantage of existing approval for primary biliary cholangitis (PBC), which has provided Intercept with an opportunity to familiarize physicians with the drug prior to its launch in NASH.
LIST OF FIGURES
11 Figure 1: Datamonitor Healthcare’s drug assessment summary of Ocaliva for NASH
12 Figure 2: Datamonitor Healthcare’s drug assessment summary of Ocaliva for NASH
15 Figure 3: Ocaliva sales for NASH across the US and five major EU markets, by country, 2018–27
LIST OF TABLES
6 Table 1: Ocaliva drug profile
8 Table 2: Late-phase trials of Ocaliva for NASH
10 Table 3: Ocaliva for NASH – SWOT analysis
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