Sanofi’s rare blood clotting drug approved by FDA

Sanofi’s Cablivi (caplacizumab-yhdp) received FDA Fast Track designation and was evaluated under Priority Review. It has now received FDA approval as the first treatment for adult patients with a rare blood clotting disorder, acquired thrombotic thrombocytopenic purpura. The decision was based on result from a clinical trial involving 145 patients where it was shown that platelet counts improved faster among patients treated with Cablivi versus placebo. Treatment with Cablivi also resulted in a lower total number of patients with either aTTP-related death and recurrence of aTTP during the treatment period, or at least one treatment-emergent major thrombotic event.
Richard Pazdur, director of the FDA’s Oncology Centre of Excellence, said: “Patients with aTTP endure hours of treatment with daily plasma exchange, which requires being attached to a machine that takes blood out of the body and mixes it with donated plasma and then returns it to the body. Even after days or weeks of this treatment, as well as taking drugs that suppress the immune system, many patients will have a recurrence of aTTP. Cablivi is the first targeted treatment that inhibits the formation of blood clots. It provides a new treatment option for patients that may reduce recurrences.”