Intercept (ICPT) Announces Unanimous FDA Committee Recommendation for Accelerated Approval of OCA as PBC Treatment

April 7, 2016 5:02 PM EDT

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Intercept Pharmaceuticals, Inc. (Nasdaq: ICPT) announced that the U.S. Food and Drug Administration (FDA)’s Gastrointestinal Drugs Advisory Committee voted 17 to 0 to recommend accelerated approval of Ocaliva™ (obeticholic acid) for the treatment of patients with primary biliary cirrhosis, recently renamed primary biliary cholangitis (PBC). The target date for the FDA to take action under the Prescription Drug User Fee Act (PDUFA) is May 29, 2016. The FDA is not bound by the Advisory Committee's guidance, but takes its advice into consideration when reviewing investigational medicines. If approved, Ocaliva would be the first new treatment for PBC in nearly 20 years.

“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, M.D., Chief Executive Officer and President of Intercept. “We’d like to thank the many patients and physicians who took part in the research discussed in today’s meeting, as their participation and dedication has been – and remains – instrumental in evolving the treatment paradigm for PBC.”

Intercept is seeking accelerated approval of Ocaliva for the treatment of PBC in patients with an inadequate response to, or who are unable to tolerate, ursodeoxycholic acid (UDCA), the only approved therapy for this disease. While UDCA has a marked impact on clinical outcomes in PBC, a substantial percentage of UDCA-treated patients have a suboptimal response or are intolerant to treatment, leaving them at significantly increased risk of an adverse outcome.

The Advisory Committee’s recommendation is based on data from the clinical development program for Ocaliva in PBC, including the Phase 3 POISE trial, which assessed the safety and efficacy of Ocaliva in 216 PBC patients who had an inadequate therapeutic response to, or were unable to tolerate, UDCA. Intercept’s New Drug Application (NDA) includes data for 432 PBC patients who have received Ocaliva with an amassed total of 675 patient years of exposure and some patients on therapy for over five years. In accordance with the FDA guidelines for accelerated approval, Intercept is currently enrolling COBALT, a global Phase 4 long-term outcomes trial to confirm the clinical benefit of Ocaliva in people living with PBC.

PBC is a rare chronic liver disease, and if patients are left untreated or have an inadequate response to UDCA therapy, the disease typically progresses to hepatic fibrosis, cirrhosis, liver failure and death unless they receive a liver transplant.

The brand name Ocaliva has been provisionally approved by the FDA and European Medicines Agency, but Ocaliva is an investigational medicine that has not been granted marketing authorization or approval from any regulatory authority.

Conference Call Information

Intercept will host a conference call today, Thursday, April 7, 2016, at 5:30 p.m. ET to discuss the outcome of the Gastrointestinal Drugs Advisory Committee meeting. The live event will be available on the investor page of the Intercept website at http://ir.interceptpharma.com or by calling (855) 232-3919 (toll-free domestic) or (315) 625-6894 (international) five minutes prior to the start time (no passcode required). A replay of the call will be available on the Intercept website approximately two hours after the completion of the call and will be archived for two weeks.



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