The FDA’s Gastrointestinal Drugs Advisory Committee has voted unanimously (17 to 0) to recommend accelerated approval of Ocaliva (obeticholic acid, Intercept Pharmaceuticals) 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 is May 29, 2016.
The FDA is not bound by the advisory committee’s guidance but takes its advice into consideration when reviewing investigational medications. If approved, Ocaliva would be the first new treatment for PBC in nearly 20 years.
Intercept Pharmaceuticals is seeking accelerated approval of the medication for the treatment of PBC in patients who have an inadequate response to, or who are unable to tolerate, ursodeoxycholic acid (UDCA), the only approved therapy for PBC. While UDCA has a marked effect on clinical outcomes in PBC, a substantial percentage of UDCA-treated patients have a suboptimal response to or are intolerant of treatment, leaving them at increased risk of an adverse outcome.
The advisory committee’s recommendation was 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. The new drug application (NDA) for Ocaliva included data from 432 PBC patients who had received the drug, with a total of 675 patient-years of exposure. Some patients were treated for more than five years.
In accordance with the FDA guidelines for accelerated approval, Intercept is currently enrolling subjects into a global phase 4, long-term outcomes trial (COBALT) to confirm the clinical benefit of Ocaliva in patients 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, but obeticholic acid is an investigational medication that has not been granted marketing authorization or approval from any regulatory authority.
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