After years of regulatory struggles for Intercept Pharmaceuticals and its liver disease drug Ocaliva (obeticholic acid), the FDA has called time on the drug's run in the U.S. market.
The agency placed Intercept's trials of obeticholic acid on a clinical hold and requested that the company voluntarily withdraw the drug from the market, where it has been cleared to treat certain patients with primary biliary cholangitis (PBC) since winning an accelerated approval in 2016.
Despite Intercept's arduous efforts to obtain full approval for the drug, the company has obliged and will pull the med, it announced in a Sept. 11 press release.
“While our view of Ocaliva’s benefit-risk profile differs from FDA’s, we respect its request and have made this difficult decision to provide clear guidance for patients and prescribers,” Intercept’s U.S. president Vivek Devaraj said in a statement. “We remain committed to innovation in hepatology and to serving the needs of patients and physicians.”
The development marks the latest setback for Intercept's U.S. market prospects and comes after the FDA’s Gastrointestinal Drug Advisory Committee overwhelmingly voted against Intercept's data package at a meeting last year.
Committee members weren’t swayed by Intercept’s clinical trials, nor was the FDA in its pre-meeting briefing documents. In its assessment, the FDA flagged the potential for the drug to put patients at greater risk of needing a liver transplant or death.
Soon after the meeting, the FDA rejected Intercept’s bid for a traditional approval. But the agency did allow the drug to remain available for certain patients under its initial accelerated nod while it weighed safety data from a post-marketing study.
Still, the company didn’t give up and pledged to generate additional data in support of its drug.
But the hits kept on coming. Over in Europe, regulators ultimately snatched back Ocaliva’s marketing nod late last year after a holding period.
The repeated setbacks in PBC weren't exactly unfamiliar territory for Intercept. The company had at one point hoped to market the drug as the first treatment for nonalcoholic steatohepatitis, which is now known as metabolic dysfunction-associated steatohepatitis (MASH).
That situation saw a similar storyline play out, with two of the company's attempts to win the FDA’s favor falling short. Afterward, Intercept was forced to pivot from its MASH efforts to PBC, trimming a third of its workforce and winding down all MASH-related investments.
It was after the MASH rejection that Intercept sold itself to Italian drugmaker Alfasigma in a deal worth about $800 million. At the time in 2023, the company was boasting $285.7 million in full-year 2022 sales for Ocaliva, its lone product.
Intercept’s pipeline is made up largely of obeticholic acid, but it lists one other next-generation FXR agonist being tested in a phase 2a study for severe alcohol-associated hepatitis.