Rare Liver Disease Gets First New Rx in Years

The US Food and Drug Administration (FDA) has approved a new treatment for primary biliary cholangitis (PBC).

The FDA approved Ocaliva (obeticholic acid) to be given with ursodeoxycholic acid (UDCA) to adults who have not had an adequate response to UDCA. It was also approved to be given alone in adults who cannot tolerate UDCA.

PBC is a long-lasting disease that involves damaged bile ducts in the liver. Because the bile ducts are damaged, bile remains in the liver and can lead to the damage of liver cells as time goes on. Ultimately, PBC can lead to scarring of the liver, known as cirrhosis.

Ocaliva was granted fast-track designation after it appeared effective in reducing alkaline phosphatase (ALP), a biomarker. Although a confirmatory trial is ongoing, improvement in survival, a change in progression to cirrhosis and other disease-related symptoms have not yet been confirmed.

Ocaliva, from Intercept Pharmaceuticals, is the first new treatment for PBC in nearly 20 years. Ocaliva now joins UDCA, which was was approved to treat PBC in 1997. However, according to the FDA, up to 40 percent of patients given UDCA have an inadequate response. Furthermore, between 5 and 10 percent of patients cannot tolerate UDCA.

Common side effects of Ocaliva include itchy skin, feeling tired, stomach pain and joint pain. Ocaliva is not recommended in patients with complete biliary obstruction.