ON FEBRUARY 6, 2019, the U.S. Food and Drug AdministrationFDA approved caplacizumab-yhdp (Cablivi) injection, in combination with plasma exchange and immunosuppressive therapy, for the treatment of adult patients with acquired thrombotic thrombocytopenic purpura (aTTP).
Richard Pazdur, MD
“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,” said Richard Pazdur, MD, Director of the FDA’s Oncology Center of Excellence and Acting Director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “Caplacizumab-yhdp is the first targeted treatment that inhibits the formation of blood clots. It provides a new treatment option for patients that may reduce recurrences.”
Hercules Clinical Trial
THE EFFICACY of caplacizumab-yhdp was studied in the Hercules clinical trial (Clinical Trials. gov Identifier: NCT02553317) of 145 patients who were randomly assigned to receive either caplacizumab-yhdp or a placebo. Patients in both groups received the current standard of care of plasma exchange and immunosuppressive therapy. The results of the trial demonstrated that platelet counts improved faster among patients treated with caplacizumab-yhdp than with placebo.
Treatment with caplacizumab-yhdp 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. The proportion of patients with a recurrence of aTTP in the overall study period (the drug treatment period plus a 28-day follow-up period after discontinuation of drug treatment) was lower in the caplacizumab-yhdp group (13%) than in the placebo group (38%), a finding that was statistically significant.
Common side effects of caplacizumab-yhdp reported by patients in clinical trials were bleeding of the nose or gums and headache. The prescribing information for caplacizumab-yhdp includes a warning to advise health-care providers and patients about the risk of severe bleeding.
Health-care providers are advised to monitor patients closely for bleeding when administering caplacizumab-yhdp to patients who currently take anticoagulants.
For more information, visit FDA.gov. ■