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On February 6, the FDA approved the introduction of Cablivi (caplacizumab-yhdp) injection developed by Sanofi’s subsidiary Ablynx for plasma exchange and immunosuppressive therapy for adult acquired thrombotic thrombocytopenic purpura (aTTP). On September 3, 2018, the European Union has approved the application for the listing of Cablivi for this indication. Cablivi is the first new drug approved by the FDA for this indication and the world’s first new Nanobody drug.

aTTP is a rare but life-threatening coagulopathy disorder characterized by extensive coagulation of small blood vessels throughout the body, impeding normal oxygen and blood supply to major organs in the body, causing stroke and heart disease, which can lead to brain damage or death. Patients with cancer, AIDS, pregnancy, lupus, infection, postoperative, bone marrow transplant or chemotherapy may progress to aTTP.

Caplacizumab is an anti-von Willebrand Factor (vWF) Nanobody developed by Ablynx that inhibits the interaction between super-vWF multimers and platelets and avoids the appearance of platelets and coagulation and clots. Nano-antibodies are single-domain antibodies with only heavy chain variable regions, and contain minimally functional antigen-binding fragments. Compared with common antibodies, they have the advantages of small molecular weight, simple structure, easy modification, and good stability.

The efficacy of caplacizumab was confirmed in a randomized study involving 145 patients with aTTP. Patients received existing standard treatment, plasma exchange, and immunosuppressive drugs, on which they were randomized and given caplacizumab or placebo, respectively.

The results showed that patients receiving caplacizumab could rapidly improve platelet counts compared with placebo, and the number of aTTP-related deaths, the number of aTTP relapses during treatment, and the number of patients with at least one treatment-related major thrombotic event were lower than placebo. In the group, the proportion of patients with aTTP recurrence was significantly lower during the entire study period (dose period + 28 days follow-up period) than placebo (13% vs 38%). Common adverse reactions to caplacizumab include bleeding from the nose or gums and headache.

Sanofi made two major acquisitions in January 2018, first with a $11.6 billion acquisition of Bioverativ, a hemophilia and blood disease specialist drug company split from Biogen, followed by the acquisition of Ablynx for €3.9 billion.

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