Rare Disease Report

Pivotal Primary Biliary Cholangitis Study Published in NEJM

AUGUST 18, 2016
James Radke, PhD
The data that led to the approval of Ocaliva (obeticholic acid) to treat primary biliary cholangitis (PBC) (also referred to as primary biliary cirrhosis) has been published in the New England Journal of Medicine.  
The Phase 3 POISE Trial evaluated the safety and efficacy of once-daily treatment with Ocaliva in PBC patients with an inadequate therapeutic response to, or who are unable to tolerate, ursodeoxycholic acid (UDCA).
The trial randomly assigned 217 patients who had an inadequate response to ursodiol or who found the side effects of ursodiol unacceptable to receive 10 mg of obeticholic acid, 5 mg of obeticholic acid (with an option to adjust to 10 mg), or placebo.
The trial`s primary endpoints were liver biomarkers that predict liver failure in PBC patients (reduction in alkaline phosphatase (ALP) to below a threshold of 1.67 times the upper limit of the normal range and a total bilirubin level at or below the upper limit of the normal range after 12 months of Ocaliva therapy).
At the end of the 12-month study, 46-47% of the patients in the groups taking Ocaliva achieved the primary endpoint compared to only 10% of the patients in the placebo group (P < .001).
In a press release, lead author Frederik Nevens, MD, PhD, University Hospitals Leuven & KU Leuven, Belgium said:
 "For nearly 20 years, we only had one option to help our PBC patients. The introduction of Ocaliva provides physicians and patients with an opportunity to rethink treatment goals and take action when ALP and/or bilirubin remain elevated despite UDCA therapy."
At the AASLD meeting in 2015, we talked with 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) about PBC and about the clinical trial for PBC involving Ocaliva.

What is Primary Biliary Cholangitis

Clinical Trials with Obeticholic Acid


Nevens F, Andreone P, Mazzella G, et al. Placebo-Controlled Trial of Obeticholic Acid in Primary Biliary Cholangitis. N Engl J Med. 2016; 375:631-643. DOI: 10.1056/NEJMoa1509840

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