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FDA reviewers mull surrogate endpoint for Intercept's OCA

April 6, 2016 12:10 AM UTC

FDA reviewers questioned the utility of alkaline phosphatase (ALP) as a surrogate endpoint for approval of obeticholic acid ( OCA; DSP-1747) from Intercept Pharmaceuticals Inc. (NASDAQ:ICPT) to treat primary biliary cholangitis (PBC), but appeared amenable to its use. The concerns were raised in briefing documents posted ahead of Thursday's meeting of FDA's Gastrointestinal Drugs Advisory Committee to discuss an NDA for OCA, which is under Priority Review with a May 29 PDUFA date.

Reviewers will ask members of the committee to discuss whether ALP reduction alone is likely to predict clinical benefit in treating early stage PBC, and vote on whether data showing OCA's effects on ALP would support accelerated approval for PBC. FDA had recommended that pivotal studies examine a composite endpoint based on ALP and total bilirubin (TB) reduction, but noted that the majority of patients in a Phase III trial included in the NDA (92%) had normal TB at enrollment. The reviewers modeled ALP reduction thresholds that could predict clinical benefit, and said both dose cohorts in OCA's Phase III study met those thresholds more often than placebo in the agency's exploratory analysis. ...