Intercept's Ocaliva Withdrawn from US Market Amid Regulatory Challenges

Intercept Pharmaceuticals has announced the withdrawal of its liver disease drug Ocaliva (obeticholic acid) from the US market following a request from the Food and Drug Administration (FDA). This development marks the latest in a series of setbacks for the company and its flagship product.

FDA Calls for Market Withdrawal and Clinical Hold

The FDA has not only requested that Intercept voluntarily withdraw Ocaliva from the market but has also placed all related trials on clinical hold. This decision comes after years of regulatory struggles for the drug, which received accelerated approval in 2016 for the treatment of primary biliary cholangitis (PBC).

Vivek Devaraj, Intercept's US president, stated, "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."

Regulatory Hurdles and Safety Concerns

The withdrawal follows a series of regulatory challenges for Ocaliva:

• In 2022, the FDA's Gastrointestinal Drug Advisory Committee voted against Intercept's data package, citing concerns over the drug's safety profile.
• The FDA subsequently rejected Intercept's bid for traditional approval, although it allowed the drug to remain available under its initial accelerated approval.
• European regulators revoked Ocaliva's marketing authorization in late 2024.

The FDA's concerns primarily stem from data suggesting that Ocaliva may increase patients' risk of needing a liver transplant or death. These safety issues have overshadowed the drug's potential benefits in treating PBC.

Impact on Intercept Pharmaceuticals

The withdrawal of Ocaliva from the US market represents a significant blow to Intercept Pharmaceuticals. The company had previously faced setbacks in its efforts to market the drug for nonalcoholic steatohepatitis (NASH), now known as metabolic dysfunction-associated steatohepatitis (MASH).

Following these challenges, Intercept:

• Sold itself to Italian drugmaker Alfasigma in a deal worth approximately $800 million in 2023.
• Reported full-year 2022 sales of $285.7 million for Ocaliva, its only marketed product.
• Has refocused its pipeline, which now includes a next-generation FXR agonist in phase 2a studies for severe alcohol-associated hepatitis.

As the pharmaceutical industry continues to navigate complex regulatory landscapes, the case of Ocaliva serves as a reminder of the challenges faced in bringing novel therapies to market, particularly in areas with significant unmet medical needs.