FDA approves first therapy for the treatment of adult patients with a rare blood clotting disorder

FDA approved on Wednesday caplacizumab-yhdp (Cablivi—Ablynx) injection, the first therapy specifically indicated, in combination with plasma exchange and immunosuppressive therapy, for the treatment of adults with acquired thrombotic thrombocytopenic purpura (aTTP).

FDA approved on Wednesday caplacizumab-yhdp (Cablivi—Ablynx) injection, the first therapy specifically indicated, in combination with plasma exchange and immunosuppressive therapy, for the treatment of adults with acquired thrombotic thrombocytopenic purpura (aTTP). The rare and life-threatening disorder causes extensive blood clotting in the small blood vessels throughout the body. "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," said Richard Pazdur, MD, director of FDA's Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in FDA's Center for Drug Evaluation and Research. "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." A clinical trial found that platelet counts improved faster among patients treated with caplacizumab-yhdp compared with placebo. Treatment with the drug 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. Additionally, the proportion of patients with a recurrence of aTTP in the study period was 13% in the caplacizumab-yhdp group, compared with 38% among placebo recipients. Common adverse events reported by patients in clinical trials of the drug were bleeding of the nose or gums and headache.