Failed Confirmatory Trial Won’t Force Withdrawal Of Zepzelca’s Accelerated Approval, US FDA Says

The US Food and Drug Administration denied a citizen petition request that it withdraw accelerated approval of Jazz Pharmaceuticals plc/PharmaMar, S.A.’s Zepzelca (lurbinectedin) as a second-line treatment for small cell lung cancer (SCLC) because a confirmatory trial failed to meet its primary endpoint of overall survival. The agency cited aspects of the study, including use of a lower dose of Zepzelca, that could have led to its failure, and noted that the sponsor is conducting two additional confirmatory trials with a different design.

The agency’s response provides further perspective on its handling of accelerated approval when a confirmatory trial fails