A panel of outside advisers will vote Thursday on whether to recommend the FDA approve Intercept's obeticholic acid. OCA is in late-stage development for treatment of primary biliary cirrhosis, secondary to autoimmune disease. It also is in late-stage development for NASH, a form of fatty liver disease.
- FDA staff reviewers previously examined Intercept's data from its late-stage trial. Despite some safety concerns, overall the reviewers did not seem to have any potentially deal-breaking concerns.
Because of the positive feedback, Intercept's stock jumped following release of the documents.
Intercept's OCA is emerging as a star lead candidate with positive data for both of the major indications it is pursuing--primary biliary cirrhosis and NASH, a condition for which there is no approved treatment. Currently, the standard of care for primary biliary cirrhosis is ursodeoxycholic acid, but many patients are nonresponsive. Intercept is developing OCA as monotherapy and as part of combination therapy for biliary cirrhosis.
Intercept's clinical trial results for use of OCA for treatment of NASH have been very positive. Early phase 3 results from the FLINT trial showed 22% of OCA-treated patients had a NASH resolution, versus 13% of placebo-treated patients. However, the results were not deemed statistically significant. In addition, 35% of OCA-treated patients had an improvement in fibrosis, compared to 19% of placebo-treated patients.
There are some lingering safety concerns, notably, the effect of OCA on lipid levels. The FDA reviewers did not notice a major change in lipid levels during their review, but they said that ultimately OCA's, effect on lipid levels will be factored into the overall risk-benefit analysis.
Last year, Thomson Reuters Cortellis put OCA on a 'drugs to watch' list. OCA could generate $2.62 billion in revenue by 2020, according to their forecast.