Intercept’s Ocaliva: ‘Dangling’ PBC Indication At Risk As Near-Term NASH Approval Looks Unlikely

Accelerated approval for primary biliary cholangitis in 2016 came with three postmarketing requirements, but studies were terminated early; firm working toward sNDA submission in 2023 for regular approval, but US FDA says reports already are overdue and it expects to take PBC indication back to an adcomm.

Dangling shoes
Ocaliva appears to fit FDA's definition of a 'dangling' accelerated approval - when prespecified postmarketing trials do not confirm benefit but marketing continues. • Source: Shutterstock

With the prospects for near-term approval of Intercept Pharmaceuticals, Inc.’s obeticholic acid (OCA) in nonalcoholic steatohepatitis (NASH) fibrosis considerably dimmed by a negative FDA review and advisory committee recommendation, the drug’s existing accelerated approval in a much smaller indication also may be at risk for lack of confirmation of clinical benefit after seven years on the market.

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