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FDA approves first treatment for rare blood disorder in pediatric patients 12 and older

The U.S. Food and Drug Administration approved Cablivi as the first drug for pediatric patients aged 12 and older with acquired thrombotic thrombocytopenic purpura, authorizing its use with plasma exchange and immunosuppressive therapy.
Given how rare aTTP is in children, occurring in 1 per 10,000,000 annually, the U.S. Food and Drug Administration approved Cablivi for pediatric patients 12 and older.
A pediatric study of 30 children showed strong remission rates, with about 80% achieving clinical remission defined by normal platelet count and LDH < 1.5× upper limit for 30+ days.
Health officials cautioned about bleeding risks and advised stopping treatment if major bleeding occurs, noting common side effects like nosebleeds, headaches, and gum bleeding, with higher risk for patients on blood-thinning medicines.
Cablivi had prior adult approval in 2019, providing regulatory experience as children with aTTP often face anemia from red blood cell breakdown.

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