An anti-von Willebrand factor humanized single-variable-domain immunoglobulin (Nanobody), inhibits the interaction between ultralarge von Willebrand factor multimers and platelets.
Inhibits interaction between vpn Willebrand factor multimers and platelets.
In acquired thrombocytopenic purpura, an immune-mediated deficiency of von Willebrand factor-cleaving protease ADAMTS13 allows unrestrained adhesion of von Willebrand factor multimers to platelets and microthrombosis, which result in thrombocytopenia, hemolytic anemia, and tissue ischemia.
Caplacizumab-yhdp injection is the first therapy specifically indicated for the treatment of adults with acquired thrombotic thrombocytopenic purpura, a rare and life-threatening blood clotting disorder.
Trade name Cablivi, Ablynx
Caplacizumab-yhdp is indicated for use in combination with plasma exchange and immunosuppressive therapy.
It is the first targeted treatment that inhibits the formation of blood clots, and provides a new treatment option for patients that may reduce recurrences.
Acquired thrombotic thrombocytopenic purpura can develop due to cancer, HIV, pregnancy, lupus or infections, or after having surgery, bone marrow transplantation or chemotherapy.
Acquired thrombotic thrombocytopenic purpura is characterized by extensive blood clots in small blood vessels.
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: faster improvement in platelet counts among patients treated with caplacizumab-yhdp.
Fewer patients assigned caplacizumab-yhdp experienced disease recurrence, defined as a treatment-emergent major thrombotic event, during the treatment period.
Further, caplacizumab-yhdp conferred a lower rate of aTTP-related death.
Bleeding of the nose or gums and headache are the most common adverse event among patients assigned caplacizumab-yhdp.
Acquired thrombotic thrombocytopenic purpura (TTP) is caused by aggregation of platelets on ultralarge von Willebrand factor multimers.
The process causes microvascular thrombosis and multiorgan ischemia with potentially life-threatening complications.
Failing plasma exchange and immunosuppressive therapies induced remission, but mortality and morbidity due to microthrombosis remain high.
Patients with acquired TTP randomly assigned to subcutaneous caplacizumab (10 mg daily) or placebo during plasma exchange and for 30 days afterward.
Caplacizumab induced a faster resolution of the acute TTP episode than did placebo.
Caplacizumab was associated with an increased tendency toward bleeding, as compared with placebo.