On February 6, the FDA approved Cablivi (caplacizumab-yhdp) Injection, developed by Sanofi subsidiary Ablynx, in combination with plasmapheresis and immunosuppressive therapy for acquired thrombotic thrombocytopenic purpura (aTTP) in adults.On September 3, 2018, the European Union has approved the marketing application of Cablivi for this indication.Cablivi is the first new drug approved by the FDA for this indication and the first new nanobody drug to be marketed worldwide.
aTTP is a rare but life-threatening blood clotting disorder characterized by extensive clotting of small blood vessels throughout the patient's body that impedes normal oxygen and blood supply to major organs in the body, causing stroke and heart disease, leading to brain damage or death.Cancer.AIDS.Pregnancy.Lupus.Infection.After surgery.Patients with bone marrow transplantation or chemotherapy are likely to progress to aTTP.
Caplacizumab is an anti-von Willebrand factor (vWF) nanobody developed by Ablynx that inhibits the interaction between large vWF multimers and platelets, avoiding platelets and the appearance of clots and clots.Nanobodies are single-domain antibodies with only the heavy chain variable region, containing the smallest functional antigen-binding fragment, and have a lower molecular weight than common antibodies.The structure is simple and easy to reform. The stability is good and so on.
The efficacy of caplacizumab was confirmed in a randomized study involving 145 patients with aTTP. Patients received existing standard treatments. Plasma exchange and immunosuppressive drugs were randomized and given caplacizumab or placebo, respectively.
The results showed that patients receiving caplacizumab were able to rapidly improve platelet counts compared with placebo, and the number of aTTP-related deaths. The number of aTTP relapses during treatment. The number of patients with at least one treatment-related major thrombotic event was lower than placebo. In the group, the proportion of patients with aTTP recurrence during the entire study period (dose period + 28 days follow-up period) was significantly lower than that of the placebo group (13% vs 38%). Common side effects of caplacizumab include nose or gum bleeding.
Sanofi made two major acquisitions in January 2018, first to acquire the hemophilia and blood disease specialist drug company Bioverativ, which was split from Biogen, and then to acquire Ablynx for 3.9 billion euros, which will be caplacizumab. In the pocket of income.