Ocaliva EC decision suspension of conditional marketing authorization

On September 5^th, 2024, Advanz announced that the EU's General Court has temporarily suspended the European Commission’s decision to revoke conditional marketing authorization (CMA) for Ocaliva (obeticholic acid) to treat 2L PBC.

Key Details:

• Just 2 days after CMA withdrawal, legal action by Advanz has temporarily reinstated Ocaliva’s marketing authorization “until further notice”
• Advanz states that Ocaliva “remains fully available for new and existing patients across Europe”

CI Assessment:

• Ocaliva will remain available in the EU for now; the timing for the appeals process is unclear
• Legal action by Advanz was anticipated, especially given strong support from PBC patient and physician groups  (eg, PBC Foundation)
• Despite a temporary legal victory, it seems unlikely that Advanz will secure a permanent reprieve; the EC decision was based on Ocaliva’s failed placebo-controlled outcomes study and “insufficient” supporting real-world evidence (RWE)
• Ocaliva could still be made available for patients who need it through compassionate use or similar programs
• FDA will soon deliberate and decide Ocaliva’s fate in the U.S. (FDA Ad Comm Friday, 13 Sep 2024; PDUFA, 15 Oct 2024)