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This action marks first drug approved for pediatric patients with acquired thrombotic thrombocytopenic purpura.
The U.S. Food and Drug Administration (FDA), has approved Cablivi (caplacizumab - yhdp) for injection to treat pediatric patients 12 years and older with acquired thrombotic thrombocytopenic purpura (aTTP) in connection with plasma exchange and immunosuppressive therapy.
Cablivi was originally approved in 2019 to treat adults with this condition.
Acquired thrombotic thrombocytopenic purpura (aTTP) is a rare disorder that is even more uncommon in children, only occurring in about 1 to 10 million annually, said FDA.
aTTP causes blood clots in the small vessels. These clots can slow or block the flow of blood to parts of the heart, brain, kidneys, and other organs. In this disorder, platelets (blood cells involved in clotting) clump together, leaving fewer platelets in the rest of the body to help with clotting. In addition, red blood cells may break apart, causing anemia.
The FDA affirmed that the efficacy of Cablivi to treat pediatric patients with aTTP was evaluated in a reproductive chart review study of 30 patients ranging from 2-8 years old. Results showed that 80 percent of patients achieved clinical remission, defined as achieving a normal platelet count and having LDH levels (a marker of red blood cell breakdown) less than 1.5 times the upper limit of normal for 30 days or more.
Because Cablivi works by targeting the abnormal blood clotting, it can also cause serious and fatal bleeding. The risk of bleeding is higher in patients with underlying coagulopathies (impaired clotting abilities) or who are also taking blood thinning medicines (antiplatelet agents or anticoagulants).
Patients should stop treatment if clinically significant bleeding occurs. Patients should also not take Cablivi 7 days before elective surgery, dental procedures or other invasive procedures. In addition, patients should not take Cablivi if they have had a severe allergic reaction to any of its ingredients.
The most common side effects are nose bleeds, headache, and gingival (gum) bleeding.
Cablivi received Orphan Drug Designation for this indication.
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