Intercept announced receipt of a Complete Response Letter (CRL) from FDA for its Ocaliva

Intercept announced receipt of a Complete Response Letter (CRL) from FDA for its supplemental New Drug Application (sNDA) for standard (full) approval of Ocaliva (obeticholic acid) in  2L PBC.

• A CRL was expected following the negative FDA AdComm (Gastrointestinal Drugs Advisory Committee, GIDAC) in September 2024
• FDA is "unable to approve the sNDA in its current form”
• FDA is “continuing to consider safety data” from Ocaliva's long-term confirmatory outcomes study (COBALT) and other sources
• Ocaliva continues to be available for the treatment of 2L PBC in the US under its accelerated approval status

• This CRL would be expected to trigger an FDA proposal to withdraw Ocaliva from the US Market, but the certainty of this outcome, and potential timing are unclear
• If FDA proposes to withdraw Ocaliva’s Accelerated Approval, Intercept could accept or appeal
  • In 2023, FDA implemented a new expedited withdrawal process under the Consolidated Appropriations Act (thus, there are few relevant analogs to consider)
  • An appeals process could take ~6 to 12 months, during which time Ocaliva is expected to remain available in the US