The US Food and Drug Administration (FDA) is a federal agency of the Department of Health and Human Services. Credit: Sergei Elagin via Shutterstock.

Following a negative Advisory Committee (AdCom) decision, the US Food and Drug Administration (FDA) has rejected Intercept’s Ocaliva (obeticholic acid) for the treatment of pre-cirrhotic fibrosis due to nonalcoholic steatohepatitis (NASH) in a complete response letter (CRL) to the company.

In a 23 June conference call, the company announced that it would begin the process of shutting down the REGENERATE trial (NCT02548351), with an aim to complete this process by the end of 2023.

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Intercept will continue to collect study data until the trial is fully terminated. Furthermore, the biotech has already begun “winding down” its NASH-related investment into research and development, commercial, medical affairs and administrative function.

The company will also cut down its workforce by a third to minimise operating expenses, beginning in the coming weeks with plans to complete this by the end of 2023.

Intercept will pivot its focus to Ocaliva’s use for primary biliary cholangitis (PBC), preserving the workforce in this department. The FDA granted Ocaliva accelerated approval in 2016 for the treatment of PBC in combination with ursodeoxycholic acid. The therapy will continue to be studied for real-world evidence data and to improve PBC management.

Intercept CEO Jerry Durso said: “Ocaliva is the only second-line therapy for PBC to demonstrate a positive impact on outcomes such as transplant-free survival.”

Intercept plans to send the FDA a regulatory submission, including data from the Phase III/IIIb Cobalt trial in 2023. This will fulfil its post-marketing requirements, stipulated during its 2016 approval for PBC. Intercept’s chief financial officer Andrew Saik announced that the company is also adjusting its 2023 Ocaliva net sales target from $310m to $340m to ensure the company’s profitability.

The FDA’s decision follows a May 2023 FDA AdCom meeting where panellists voted 12-4 against Ocaliva’s approval. The panel members blamed a lack of clear data on the drug’s safety risks, among other points. Treatment-emergent adverse events (TEAEs), treatment-emergent serious adverse events (TESAEs) and deaths were higher in the 25mg Ocaliva group at 55%, compared to 33% for 10mg and 24% in placebo. The therapy did not meet the other primary endpoint of NASH resolution with no worsening of liver fibrosis.

In the CRL, the FDA indicated the drug cannot be approved in its current form and any later NDA resubmissions would require long-term outcomes data from Intercept’s Phase III REGENERATE study (NCT02548351). In response to this decision, the New-Jersey based company has chosen to suspend all NASH-related investments and reconfigure the company’s future direction to focus more on rare and liver diseases.

In a press release, Durso said: “While this is clearly not the outcome that we have worked toward, I’m proud of the impact that Intercept has made to move the science of NASH forward and bring the field closer to a treatment option.”