Sanofi says FDA approves Cablivi for adults with aTTP

The U.S. Food and Drug Administration has approved Cablivi in combination with plasma exchange and immunosuppression for the treatment of acquired thrombotic thrombocytopenic purpura in adults. Cablivi is the first FDA approved therapy specifically indicated for the treatment of aTTP. “The U.S. approval of Cablivi provides a much-needed treatment option for people facing this challenging disease. There have been limited medicines available to treat aTTP until now,” says Olivier Brandicourt, M.D., CEO, Sanofi. “Cablivi marks the first U.S. approval in our newly formed rare blood disorders franchise, and we look forward to continuing to provide important medicines for people living with these very serious diseases.” Cablivi received FDA Fast Track designation and was evaluated under Priority Review, which is reserved for medicines that represent significant improvements in safety or efficacy in treating serious conditions. The approval of Cablivi in the U.S. is based on the results of the pivotal multicentre, randomized, double-blind, placebo-controlled Phase 3 clinical study known as HERCULES. This trial evaluated the efficacy of Cablivi in combination with plasma exchange and immunosuppressive therapy versus placebo, plasma exchange and immunosuppressive therapy in 145 adults experiencing an episode of aTTP. In the HERCULES study, treatment with Cablivi in combination with plasma exchange and immunosuppression resulted in a significantly shorter time to platelet count response versus plasma exchange and immunosuppression alone, the study’s primary efficacy endpoint; in secondary endpoints, Cablivi showed a significant reduction on a composite endpoint of aTTP-related death, recurrence of aTTP, or a major thromboembolic event during study drug treatment versus plasma exchange and immunosuppression alone; and a significantly lower percentage of aTTP recurrences in the overall study period versus plasma exchange and immunosuppression alone.

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