Healthcare company Sanofi (EURONEXT:SAN) (NASDAQ:SNY) reported on Wednesday the receipt of approval from the US Food and Drug Administration (FDA) for Cablivi (caplacizumab-yhdp) in combination with plasma exchange and immunosuppression for the treatment of acquired thrombotic thrombocytopenic purpura (aTTP) in adults.

According to the company, Cablivi is the first US FDA approved therapy indicated for the treatment of aTTP, a rare, life-threatening, rare blood-clotting autoimmune blood disorder. It received the US FDA's Fast Track designation and was evaluated under Priority Review and is expected to be available in the US late in the Q1.

The company said 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. It is an anti-vWF Nanobody, 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.

In aTTP, the accumulation of ultra-large vWF causes extensive clot formation in small blood vessels throughout the body, leading to severe thrombocytopenia (very low platelet count), microangiopathic hemolytic anemia (loss of red blood cells through destruction) and ischemia (restricted blood supply to parts of the body), disclosed the company.

This US FDA's approval of Cablivi is based on the results of the company's pivotal multicentre, randomised, double-blind, placebo-controlled Phase 3 clinical HERCULES study in 145 adults experiencing an episode of aTTP. The 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, added the company.