- A FDA advisory panel on Thursday voted unanimously in favor of Intercept's drug Ocaliva (obeticholic acid or OCA), recommending the regulator approve the drug for treatment of primary biliary cirrhosis (PBC), an autoimmune liver disease.
- Intercept is also pursing an indication for non-alcoholic steato-hepatitis (NASH), a fatty liver disease. OCA is in late-stage development for treatment of NASH, with a phase 3 trial underway in 2,000 patients.
- The advisory board meeting discussed dosing concerns as well as questions of whether use of alkaline phosphatase as a surrogate endpoint is an acceptable basis for an accelerated approval.
The current standard of care for primary biliary cirrhosis is ursodeoxycholic acid. However, not all patients can tolerate it. Intercept aims to develop OCA as a monotherapy in patients who can't tolerate ursodeoxycholic acid, or as combination therapy in those who can. Despite concerns about the use of a surrogate endpoint, and concerns about dosing—which have largely been resolved—it' appears OCA will most likely be approved for PBC, with qualifications and post-marketing requirements likely.
The FDA doesn't always follow the recommendations of advisory committees on approval but usually does. OCA's target action date is May 29, 2016.
As PBC is a slow-moving disease which can lead to liver failure and death, there is a need to evaluate OCA in a longer term clinical trial context.
The opportunity for OCA as a treatment for NASH is larger, however. Roughly 2% to 3% of the population is estimated to have NASH, compared with one in 1,000 women over 40 who have PBC. So far, Intercept's phase 3 results have been positive.
Thomson Reuters Cortellis has forecast 2020 sales of OCA at over $2.6 billion. If OCA is approved, it would be the first new treatment for PBC in over 20 years.