Obeticholic Acid in Primary Biliary Cholangitis

While Primary Biliary Cholangitis (PBC) (previously called primary biliary cirrhosis) is seen mainly in adult hepatology practices, a new treatment may be emerging and this same medication is likely to be considered for several liver conditions.

Reference: F Nevens et al. NEJM 2016; 375: 631-43.

This 12 month, double-blind, placebo-controlled phase 3 “POISE” trial with 217 patients examined the use of obeticholic acid with or without ursodeoxycholic acid.  Surrogate markers of PBC were followed & the treatment groups improved compared to placebo.  However, adverse effects, particularly itching, were more common in the obeticholic acid groups; serious adverse effects were 11-16% in the treatment groups compared with 4% in the placebo group.

Results below:


My take: It will be nice when important clinical endpoints can be assessed for this therapy like progression to cirrhosis.  For now, the cost of this treatment is ~$70,000 yearly.

Related blog post:

Breakthrough for Fatty Liver Disease? | gutsandgrowth


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