Italian pharma Alfasigma will face an FDA advisory committee next month to discuss the full approval of its rare liver disease treatment Ocaliva, a drug the company acquired less than a year ago.
The Sept. 13 advisory meeting was disclosed in the Federal Register on Friday, teeing up a major benchmark for the pharma and the drug after Alfasigma bought Intercept Pharmaceuticals for $800 million at the end of 2023. The deal centered on Ocaliva, which snagged accelerated approval to treat primary biliary cholangitis (PBC) in 2016. The condition causes the body’s immune system to attack and injure bile ducts.
A spokesperson for Alfasigma did not immediately respond to a request for comment on the meeting. Intercept had a supplemental new drug package accepted in February of this year, and the FDA is expects to decide on the application by Oct. 15.
Since reaching the market, Ocaliva has been dogged by label warnings and a failure to expand into other liver conditions.
A black box warning was added to the drug in 2018 after it was being incorrectly dosed on a daily basis, as opposed to a weekly basis, possibly increasing the risk of more severe liver damage. The FDA began formally investigating the risk of additional liver damage in 2020, which an Intercept spokesperson said at the time would likely spur a 12-month review.
But it was outside of PBC where the active ingredient of Ocaliva caused greater disappointment. Intercept had tried for years to get the FDA to greenlight obeticholic acid as a treatment for metabolic-associated steatohepatitis (MASH), an advanced form of fatty liver disease that until this year had no approved treatments. (The condition was previously referred to as nonalcoholic steatohepatitis or NASH.)
The company reported positive Phase 3 results in 2019 but received a complete response letter in 2020 after the agency felt it couldn’t back reduction in liver fibrosis as an adequate surrogate endpoint. Intercept said that the FDA conducted an “incomplete review” of the data. Intercept tried again, submitting more biopsy and safety data in 2023, but that too was knocked down by the FDA following scrutiny from outside advisors. The second rejection led Intercept to fully divest from its MASH ambitions, causing layoffs amid a wider restructuring.
By the time the September advisory meeting rolls around, Gilead may be nipping at Alfasigma’s heels. The FDA is days away from a decision on Gilead’s PBC candidate, seladelpar, the centerpiece of the $4.3 billion acquisition of CymaBay Therapeutics earlier this year. Analysts expect the drug’s peak sales to be at least $500 million.