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Sponsors Aim To Avoid Confirmatory Trial Hurdles That Plagued Ocaliva In Primary Biliary Cholangitis

 
• By Sue Sutter

The FDA and sponsors of two new accelerated approval drugs for PBC have taken steps in study design, initiation and reporting transparency to ensure timely completion.

Hurdles
Sponsors of new PBC drugs are hoping to avoid some of the hurdles that plagued Ocaliva's confirmatory trial. • Source: Shutterstock

The US Food and Drug Administration and sponsors of two new accelerated approval drugs for primary biliary cholangitis appear to have taken steps to avoid some of the problems that tripped up the confirmatory trial for Intercept Pharmaceuticals, Inc.’s Ocaliva (obeticholic acid).

Key Takeaways

  • The confirmatory trial for Intercept’s Ocaliva began before accelerated approval, but was negatively impacted by safety-related labeling changes and the crossover to commercial drug.

  • The FDA pressed sponsors of two new PBC drugs, elafibranor and seladelpar, to initiate placebo-controlled confirmatory trials before NDA submission

A close look at the postmarketing requirements for Ipsen SA/Genfit SA’s Iqirvo (elafibranor) and Gilead Sciences, Inc

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