FDA committee recommends approving rare liver disease drug

An FDA advisory committee recently voted to recommend approval of obeticholic acid (Ocaliva, Intercept Pharmaceuticals, Inc.) for the treatment of primary biliary cholangitis (PBC). If approved, Ocaliva would be the first new treatment for PBC in nearly 20 years.

Intercept is seeking accelerated approval of Ocaliva for the treatment of PBC in patients with an inadequate response to ursodeoxycholic acid (UDCA), the only approved therapy for the disease. A substantial percentage of patients treated with UDCA have a suboptimal response or are intolerant to treatment.

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PBC, formerly known as primary biliary cirrhosis, is a rare and chronic liver disease that results from autoimmune destruction of the bile ducts that transport bile acids out of the liver. When left untreated, PBC can progress to hepatic fibrosis, cirrhosis, liver failure and death unless the patient receives a liver transplant. 

The US FDA’s Gastrointestinal Drugs Advisory Committee voted 17 to 0 to recommended accelerated approval of Ocaliva for the treatment of patients with PBC. "We're pleased that the Advisory Committee strongly supported the approval of Ocaliva for people living with PBC. Today's positive recommendation is an encouraging step for the PBC community," said Mark Pruzanski, MD, CEO and president of Intercept.

The advisory committee’s recommendation is based on data from the clinical development program for Ocaliva. The program included a phase 3 trial (POISE), which evaluated the safety and efficacy of Ocaliva in 216 patients with PBC who had an inadequate response to, or were unable to tolerate, UDCA.

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The New Drug Application (NDA) for Ocaliva includes data for 432 PBC patients who received Ocaliva with a total of 675 patient years of exposure and some patients on therapy for over 5 years. Intercept is currently enrolling a global phase 4 long-term outcomes trial (COBALT) to confirm the clinical benefit of Ocaliva in people living with PBC.

The target date for FDA to take action under the Prescription Drug User Fee Act (PDUFA) is May 29, 2016. FDA takes the advice of the committee into consideration when reviewing new investigational drugs, but is not bound to the committee’s recommendation. If approved, Ocaliva would be the first new treatment for PBC in nearly 20 years. 

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