Nanobody drug for aTTP

April 9, 2019 0 By FM

The US FDA cleared caplacizumab-yhdp (Cablivi), an anti-vWF nanobody, in combination with plasma exchange and immunosuppression for the treatment of acquired thrombotic thrombocytopenic purpura (aTTP) in adults.

Caplacizumab is the first FDA approved therapy specifically indicated for the treatment of aTTP.

Caplacizumab targets von Willebrand factor (vWF), a protein in the blood involved in hemostasis. It is designed to inhibit the interaction between vWF and platelets.

Caplacizumab is 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. The treatment received FDA Fast Track designation and was evaluated under Priority Review.