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FDA approves Cablivi, first treatment for acquired thrombotic thrombocytopenic purpura
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The FDA today approved caplacizumab-yhdp injection as the first therapy specifically indicated for the treatment of adults with acquired thrombotic thrombocytopenic purpura, a rare and life-threatening blood clotting disorder.
Caplacizumab-yhdp (Cablivi, Ablynx) is indicated for use in combination with plasma exchange and immunosuppressive therapy.
“Patients with acquired thrombotic thrombocytopenic purpura [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,” 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, said in a press release. “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.”
aTTP — which patients can develop due to cancer, HIV, pregnancy, lupus or infections, or after having surgery, bone marrow transplantation or chemotherapy — is characterized by extensive blood clots in small blood vessels, which can prevent oxygen and blood from reaching major organs and can cause strokes and heart attacks that lead to brain damage or death.
The approval was based, in part, on data from 145 patients randomly assigned to receive caplacizumab-yhdp or placebo along with plasma exchange and immunosuppressive therapy.
Results showed faster improvement in platelet counts among patients treated with caplacizumab-yhdp.
Further, fewer patients assigned caplacizumab-yhdp experienced disease recurrence, defined as a treatment-emergent major thrombotic event, during the treatment period. Thirteen percent of patients in the study-drug group experienced a recurrence of aTTP during the drug treatment period and 28-day follow-up period after discontinuation compared with 38% of placebo patients.
Further, caplacizumab-yhdp conferred a lower rate of aTTP-related death.
Bleeding of the nose or gums and headache were the most common adverse event among patients assigned caplacizumab-yhdp. Health care providers should monitor patients closely for bleeding when administering the injection to patients currently on anticoagulants, according to the FDA.
This application also received priority review and orphan drug designations.