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Intercept’s OCA In NASH: When Hitting Surrogate Endpoint Is Not Enough For Accelerated Approval

 
• By Sue Sutter

Although US FDA reaffirmed its support of surrogate endpoints described in a 2018 draft guidance on NASH fibrosis, advisory committee members questioned the link to clinical benefit and said obeticholic acid’s serious risks made it difficult to consider the effect on the surrogate in a vacuum.

Tire in mud
The degree to which an effect on a histopathologic surrogate endpoint will ultimately yield benefits 'remains mired in uncertainty.' • Source: Shutterstock

The US Food and Drug Administration is standing behind the surrogate endpoints laid out in its 2018 guidance on drug development for nonalcoholic steatohepatitis (NAS) fibrosis, but the recent advisory committee review of Intercept Pharmaceuticals, Inc.’s obeticholic acid shows sponsors need to do more than just hit those endpoints to gain accelerated approval.

The 25 mg dose of obeticholic acid met one of the two surrogate endpoints the FDA has deemed acceptable for accelerated approval, but the FDA and members of the Gastrointestinal Drugs Advisory Committee considered OCA’s effect

Key Takeaways

  • The FDA stands by the surrogate endpoints described in a 2018 draft guidance on NASH drug development despite questioning the “modest” effect of OCA 25 mg on one of those endpoints.

  • Hitting the mark on the prespecified surrogate endpoint was not enough for advisory committee members to support accelerated approval

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