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FDA approves first therapy for acquired thrombotic thrombocytopenic purpura: Sanofi's Cablivi

Published on 07/02/19 at 11:17am

Following swiftly on the heels of the previous success with the announcement of positive Phase 3 data for Icaria in relapsed/refractory multiple myeloma, Sanofi has revealed the FDA approval of Cablivi (caplacizumab-yhdp), together with plasma exchange and immunosuppression, for the treatment of acquired thrombotic thrombocytopenic purpura (aTTP).

The decision makes Cablivi the first therapy to be awarded approval from the US agency in this indication, as well as the first based on Sanofi’s Nanobody technology. The manufacturer describes these Nanobody-based medicines as: “a novel class of proprietary therapeutic proteins based on single-domain antibody fragments that contain the unique structural and functional properties of naturally-occurring heavy chain only antibodies.”

The approval of Cablivi comes after the drug was awarded Fast Track designation by the FDA for tackling unmet need in aTTP, a rare autoimmune disorder which can be life-threatening and whose episodes kill around one in five sufferers despite the availability of treatments.

The FDA’s decision was made on the back of Phase 3 data which showed that the combo met its primary endpoint, significantly reducing the time to platelet count response compared to plasma exchange and immunosuppression alone.

“The US 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,” commented Sanofi CEO, Dr Olivier Brandicourt.  “Cablivi marks the first US 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.” 

Matt Fellows

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