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10 Feb 2019

FDA approves first Nanobody-based medicine for adults with aTTP

Cablivi is the first US approval for Sanofi's new rare blood disorders franchise.

The FDA has approved Cablivi (caplacizumab-yhdp) in combination with plasma exchange and immunosuppression for the treatment of acquired thrombotic thrombocytopenic purpura (aTTP) in adults. Cablivi is the first FDA approved therapy specifically indicated for the treatment of aTTP.

"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," says Olivier Brandicourt, Chief Executive Officer, Sanofi. "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."

Cablivi targets von Willebrand factor (vWF), a protein in the blood involved in hemostasis. It is designed to inhibit the interaction between vWF and platelets. Cablivi is an anti-vWF Nanobody and Sanofi's first Nanobody-based medicine to receive approval in the US. Nanobodies are 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.

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.

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