Sanofi Submits NDA for Caplacizumab to Treat Acquired Thrombotic Thrombocytopenic Purpura in China

Intelligent Finance APP learned on May 8 that the CDE official website showed that Sanofi's "Injection of Caplacizumab" has been submitted for marketing authorization. The indication is for use in combination with plasma exchange and immunosuppressive therapy to treat acquired thrombotic thrombocytopenic purpura (aTTP) (also known as immune-mediated thrombotic thrombocytopenic purpura), applicable to adults and adolescent patients aged 12 years and above with a body weight of at least 40kg. This indication was previously included in the priority review.

aTTP is a rare and life-threatening autoimmune blood disorder. In aTTP, the accumulation of large amounts of von Willebrand factor (vWF) leads to extensive thrombosis in small blood vessels throughout the body, resulting in severe thrombocytopenia (extremely low platelet count), microangiopathic hemolytic anemia (loss of red blood cells due to destruction), and ischemia (restricted blood supply to parts of the body).

Caplacizumab is a nanobody targeting vWF, designed to inhibit the interaction between vWF and platelets. The drug was originally developed by Ablynx, which was acquired by Sanofi in 2018, bringing Caplacizumab under Sanofi's ownership.