Intercept Withdraws Ocaliva from US Market Amid Regulatory Challenges
Intercept Pharmaceuticals has announced the withdrawal of its liver disease drug Ocaliva (obeticholic acid) from the US market, marking the end of a tumultuous journey for the once-promising treatment. This decision comes in response to a request from the US Food and Drug Administration (FDA) and follows a series of regulatory setbacks for the company.
FDA Concerns and Clinical Hold
The FDA has not only requested the market withdrawal of Ocaliva but has also placed a clinical hold on all trials of obeticholic acid being conducted under a US investigational new drug application. This action stems from mounting safety concerns and efficacy doubts surrounding the drug.
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 History and Setbacks
Ocaliva, a small-molecule drug that activates the farnesoid X receptor, received accelerated approval in 2016 for the treatment of primary biliary cholangitis (PBC), a rare and progressive autoimmune liver disease. However, the drug's journey has been fraught with challenges:
- In September 2024, FDA staffers flagged safety concerns, noting "unfavorable trends" in death and liver transplantation rates among Ocaliva users.
- The FDA's Gastrointestinal Drug Advisory Committee voted against Ocaliva, citing an inability to verify a favorable risk/benefit profile in confirmatory studies.
- In November 2024, the FDA refused to grant Ocaliva full approval, maintaining its accelerated approval status.
- December 2024 saw the FDA reveal instances of serious liver injury in patients treated with Ocaliva, along with an apparent increased risk of death or need for liver transplantation in indicated patients.
Broader Implications for Intercept
The withdrawal of Ocaliva from the US market is the latest in a series of setbacks for Intercept Pharmaceuticals. The company had previously attempted to expand Ocaliva's indications to include metabolic dysfunction-associated steatohepatitis (MASH), formerly known as nonalcoholic steatohepatitis. These efforts failed in both 2020 and 2023, leading to significant corporate restructuring.
In September 2023, Intercept agreed to be acquired by Italian pharmaceutical company Alfasigma for $800 million. At the time, Ocaliva was Intercept's sole product, generating $285.7 million in full-year 2022 sales.
As Intercept navigates this challenging period, the company maintains its commitment to innovation in hepatology. However, with Ocaliva's withdrawal and the clinical hold on obeticholic acid trials, the future direction of Intercept's research and development efforts remains uncertain.
References
- Intercept Withdraws Embattled Liver Drug from US Market
Ocaliva won accelerated approval for primary biliary cholangitis in 2016, which the FDA refused to upgrade to full approval last year after an advisory committee was unable to verify a favorable risk/benefit profile.
- Intercept, after many setbacks, pulls liver disease drug Ocaliva in US
The FDA has officially put its foot down on Intercept Pharmaceuticals’ rare liver disease drug Ocaliva, requesting that the company pull the med from the market and putting all of its related trials on a clinical hold.
Explore Further
What specific safety concerns and efficacy doubts led to the clinical hold on obeticholic acid trials?
What were the reasons for the FDA's Gastrointestinal Drug Advisory Committee voting against Ocaliva in confirmatory studies?
How did Intercept Pharmaceuticals' previous attempts to expand Ocaliva's indications impact their corporate restructuring efforts?
What are the financial implications of Alfasigma's acquisition of Intercept Pharmaceuticals for $800 million?
Who are the main competitors of Ocaliva in the treatment of primary biliary cholangitis (PBC), and what are their market shares?