An FDA advisory committee made up of outside experts voiced their unanimous support today for Intercept's ($ICPT) would-be blockbuster obeticholic acid (OCA) for rare cases of primary biliary cirrhosis. The final decision now moves to the agency, which tends to go along in cases like this.
The vote was 17 "yes" and 0 "no" on the vital question: Is there substantial evidence to support an accelerated approval?
That didn't come as a big shock to any of the analysts covering the company, and shares of Intercept surged about 10% in after-market trading.
The crux of the issue for the panel zeroed in on the type of pivotal data that is being used to gain an approval, following some skepticism in an internal FDA review that the data on a single biomarker for the disease--using alkaline phosphatase (ALP) as a surrogate endpoint for transplant-free survival--would be enough to warrant an approval. Data on another key biomarker, total bilirubin--which could come from a confirmatory study--was largely absent in the NDA.
During a general discussion at the end of the session today, members of the panel reached a consensus on using ALP as the best surrogate endpoint for predicting the clinical benefit of OCA. Seeing solid data on changes in total bilirubin would be helpful, but in early-stage disease that marker is generally normal. The panel also backed starting the dose at 5 mg and then titrating up to 10 mg in order to achieve a response while guarding against side effects. And many felt that there's enough data to support use of OCA as a monotherapy for a small number of patients who can't tolerate ursodeoxycholic acid.
The advisers also emphasized that more data from an ongoing confirmatory study was important for the long-term use of the drug.
There was some disagreement, though, on whether the drug should be used for moderate to advanced stages of the disease, where bilirubin data would be more important.
A green light would set the stage for New York-based Intercept to gain real-world knowledge about the drug, while earning the first round of revenue for what could be a much bigger franchise if an approval for NASH--fatty liver disease--is in the cards.
The PDUFA date, the agency's deadline for making a decision on marketing the drug for PBC, is May 29.