FDA Approves Ocaliva for Primary Biliary Cholangitis

James Radke, PhD

The FDA has approved Ocaliva (obeticholic acid) for the treatment of 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.

The approval comes as no surprise given that on April 7, 2016, the FDA’s Gastrointestinal Drugs Advisory Committee voted 17 to 0 in favor of granting accelerated approval of Ocaliva.

Primary biliary cholangitis [(PBC), also known as primary biliary cirrhosis] is a rare autoimmune liver disease that destroys the medium size bile ducts within the liver. Symptoms include abdomen pain, musculoskeletal pain, jaundice, edema, ascites, osteoporosis, elevated cholesterol and hypothyroidism.
The incidence of the disease has been estimated as 4.5 per 100,000 people.

In a press release, John Vierling, MD, FACP, FAASLD, Professor of Medicine and Surgery at Baylor College of Medicine and Past President of the American Association for the Study of Liver Diseases (AASLD) said, "Ocaliva fills an important unmet need for the many patients with PBC who have an inadequate response to or are intolerant of UDCA, which until now has been the only approved treatment." Dr Vierling added, "Ocaliva has demonstrated a clinically meaningful improvement in lowering ALP, a liver enzyme and biomarker that is used to track disease progression in patients with PBC. Importantly, Ocaliva maintained durable ALP reductions, which is critical for treatment of a chronic disease like PBC."
At the AASLD meeting in 2015, we talked with Dr Vierling about PBC and the effectiveness of Ocaliva. Clips from that interview are shown below.

What is Primary Biliary Cholangitis


Clinical Trials with Obeticholic Acid

Drug Availability

Ocaliva is expected to be available to PBC patients in the U.S. within 7-10 days and will be distributed through a specialty pharmacy network. For more information, visit  

Investor Conference Call Tuesday Morning

The makers of Ocaliva, Intercept Pharmaceuticals,  will host an investor conference call on Tuesday, May 31 at 8:30 a.m. ET to discuss the accelerated approval of Ocaliva. For more information, visit
Printer Printing...