Intercept Pharmaceuticals , Inc. ICPT announced that the FDA has granted accelerated approval to Ocaliva (obeticholic acid), in combination with ursodeoxycholic (UDCA), for the treatment of primary biliary cholangitis (PBC), previously known as primary biliary cirrhosis, in adults with an inadequate response to UDCA or as monotherapy in adults who are intolerant to UDCA.
The FDA has approved Ocaliva under its accelerated approval program based on a reduction in alkaline phosphatase (ALP), since an improvement in survival or disease-related symptoms has not been established yet. Continued approval in this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
Results from the phase III POISE trial showed that Ocaliva, in combination with UDCA (or as monotherapy in UDCA-intolerant patients), led to the primary composite endpoint in 46% of patients in the titration group, as compared to 10% on the placebo arm, when added to UDCA.
Ocaliva's approval for PBC will provide a major boost to Intercept Pharma after the recent setback. The company does not have any approved drug in its portfolio. We remind investors that the FDA had extended the Prescription Drug User Fee Act (PDUFA) date for priority review of Ocaliva by three months in Dec 2015. The agency was previously supposed to give a decision by Feb 29, 2016. However, it requested additional clinical data analyses, resulting in a delay.
In Apr 2016, the FDA's Gastrointestinal Drugs Advisory Committee (GDAC) voted unanimously (in the ratio of 17 to 0) in favor of Ocaliva's approval.
We note that UDCA is the only drug currently approved in the U.S. for the treatment of PBC. PBC affects approximately one in 1,000 women aged over 40 years. Hence, there is significant unmet need for therapies in this space.
Meanwhile, a marketing authorization application for Ocaliva for the treatment of PBC was accepted by the European Medicines Authority (EMA) in Jun 2015 and is currently under review. A decision is expected later in 2016.
Intercept Pharma currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the health care sector include Pfizer Inc. PFE , Bristol-Myers Squibb Company BMY and ANI Pharmaceuticals, Inc. ANIP . All the three stocks sport a Zacks Rank #1 (Strong Buy).
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