FDA committee backs accelerated approval of Intercept’s Ocaliva liver drug

The target date for the FDA to take action under the Prescription Drug User Fee Act is 29 May.

If approved by FDA, the drug would be the first new treatment for PBC in nearly 20 years.

Intercept Pharmaceuticals CEO and president Mark Pruzanski said: "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."

The company is seeking accelerated approval of Ocaliva to treat PBC in patients with an inadequate response to, or who are unable to tolerate, ursodeoxycholic acid (UDCA), the only approved therapy for the disease.

Intercept said even though 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, leading to increased risk of an adverse outcome.

The committee’s recommendation was based on data from the clinical development program for Ocaliva in PBC, including the phase 3 POISE trial, under which the safety and efficacy of Ocaliva was evaluated in 216 PBC patients who had an inadequate therapeutic response to, or were unable to tolerate, UDCA.

Intercept’s new drug application includes data for 432 PBC patients who received Ocaliva with a total of 675 patient years of exposure and several patients on therapy for more than five years.

As per the FDA guidelines for accelerated approval, Intercept is presently enrolling COBALT, a global phase 4 long-term outcomes trial for confirmation of the clinical benefit of Ocaliva in people living with PBC.

PBC is a chronic disease of the liver, which leads to progressive cholestasis and generally end-stage liver disease.