FDA Denies Full Approval for Ocaliva in Primary Biliary Cholangitis, Accelerated Approval Stands
FDA Decision:
The FDA has denied full approval for Ocaliva (obeticholic acid) in treating primary biliary cholangitis (PBC), citing insufficient evidence to verify its clinical benefits and safety concerns135.
Accelerated Approval:
Despite the denial, Ocaliva will remain available under its accelerated approval status granted in 2016 for patients with PBC who do not respond adequately to or cannot tolerate ursodeoxycholic acid (UDCA)24.
Safety Concerns:
The FDA continues to consider safety data from post-marketing studies, including Study 747-302, due to concerns over liver decompensation or failure if patients are dosed incorrectly345.
Advisory Committee Vote:
The FDA's decision aligns with the Gastrointestinal Drugs Advisory Committee's vote in September 2024, where 13 out of 14 members did not believe the available data adequately proved Ocaliva's clinical benefit35.
Market Impact:
The denial comes as new treatments for PBC have been approved, including Gilead's Livdelzi (seladelpar), which received accelerated approval in August 20244.
Sources:
1. https://www.fiercepharma.com/pharma/fda-declines-grant-intercepts-ocaliva-full-approval-rare-liver-disease
2. https://endpts.com/fda-denies-ocaliva-full-approval-but-intercept-hopes-to-keep-drug-on-market/
3. https://www.healio.com/news/gastroenterology/20241112/fda-denies-ocaliva-full-approval-in-primary-biliary-cholangitis-safety-data-still-in-play
4. https://www.pharmaceutical-technology.com/news/intercept-loses-ocaliva-fda-full-approval-bid-in-rare-liver-disease/
5. https://www.hcplive.com/view/fda-issues-crl-obeticholic-acid-ocaliva-denies-full-approval-pbc