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FDA Approves New Drug for Rare, Chronic Liver Disease

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Ocaliva™ (obeticholic acid) of US based Intercept Pharmaceuticals received accelerated approval from US FDA for treatment of an autoimmune disorder of liver called primary biliary cholangitis (PBC).

logo Intercept PharmaOcaliva™ became the first new medicine for PBC in about 20 years. Till now ursodeoxycholic acid (UDCA) was the only approved treatment for PBCFDA approved Ocaliva™ in combination with UDCA for the treatment of adults who showed inadequate response to UDCA or as monotherapy for UDCA intolerant adults.

Primary biliary cholangitis formerly known as primary biliary cirrhosis is a rare, autoimmune cholestatic liver disorder. It is indicated by slow progressive ruination of the small bile ducts of the liver with the earlier damage of intralobular ducts and Canals of Hering.

Primary biliary cholangitis

Figure 1. Effects on bile duct and surrounding tissues in PBC. 

PBC primarily affects women with about 9:1 women to male ratio. The prevalence of the disease in some areas of US and UK is around 1 in 4000.

FDA Approves New Liver Disease Drug

Ocaliva™ is a semisynthetic analog of naturally occurring bile acid chenodeoxycholic acid (CDCA). Ocaliva™ is a farnesoid X receptor (FXR) agonist which is a nuclear receptor expressed in the liver and intestine and a key regulator of bile acid, inflammatory, fibrotic and metabolic pathways. Ocaliva™ helps in reduction of the liver enzyme, alkaline phosphatase (ALP) which plays a crucial role in the treatment of the PBC and other liver related disorders.

primary biliary cholangitis sign and symptoms

Figure 2. Sign and symptoms of PBC.

In the Phase III trial, POISE, Ocaliva™ demonstrated clinically significant improvement in lowering of ALP and the biomarker which was used for tracking the disease progression in PBC patients. 46% patients receiving Ocaliva™ in combination with UDCA or as monotherapy due to UDCA intolerance met the primary endpoint compared to only 10% of those receiving UDCA with placebo. The common side effect seen in the Ocaliva™ treated patients was pruritus (itching).

PBC is an unmet medical condition and the Intercept Pharmaceuticals believe that targeting FXR can solve various liver disorder problems for which very limited or less treatment options are available like non-alchoholic steatohepatitis (NASH).

Ocaliva™ will be made available to the PBC patients of the US within 7 to 10 days through a speciality pharmacy network.

PBC affects people in the prime of their lives and, for some, the potential need for a liver transplant is a constant concern during these important years,” said Linie Moore, a PBC patient and President of the PBCers Organization, the leading PBC patient support group in the U.S. “After nearly two decades with only one approved treatment, we are thrilled to welcome this important new medicine for people living with PBC“.

Featured image credit: human anatomy background. Molecules and human organs © guniita (Stock Photo ID: 65123620)

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