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FDA Approves Therapy for Rare Blood Disorder in Pediatric Patients 12 Years and Older
This action marks first drug approved for pediatric patients with acquired thrombotic thrombocytopenic purpura
Action
On Dec. 23, 2025, the U.S. Food and Drug Administration approved Cablivi (caplacizumab-yhdp) for injection to treat pediatric patients 12 years and older with acquired thrombotic thrombocytopenic purpura (aTTP) in combination with plasma exchange and immunosuppressive therapy. Cablivi was originally approved in 2019 to treat adults with this condition.
Disease or Condition
Acquired thrombotic thrombocytopenic purpura (aTTP) is a rare disorder that is even more uncommon in children, only occurring in about 1 per 10 million children annually. aTTP causes blood clots in the small blood 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.
Data Supporting Cablivi
The efficacy of Cablivi to treat pediatric patients with aTTP was evaluated in a retrospective chart review study of 30 patients ranging in age from 2-18 years old. Results showed that 80% 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.
Safety Information
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.
Designations
Cablivi received Orphan Drug Designation for this indication.
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