The U.S. Food and Drug Administration has approved Cablivi (caplacizumab-yhdp) for pediatric patients 12 years and older suffering from acquired thrombotic thrombocytopenic purpura (aTTP), marking the first time a treatment has been authorized for this extremely rare condition in children.
aTTP is an uncommon but potentially life-threatening blood disorder characterized by tiny clots forming in small blood vessels, which can impede blood flow to vital organs such as the heart, brain, or kidneys. The condition also leads to a shortage of circulating platelets and the destruction of red blood cells, resulting in anemia and serious complications if untreated.
Originally approved in 2019 for adult patients with aTTP, Cablivi’s new pediatric indication expands its use in combination with plasma exchange and immunosuppressive therapy. This decision follows a retrospective review of clinical data showing that a significant portion of treated children achieved remission, defined by normalized platelet counts and stable markers of red blood cell health.
Despite its benefits, the therapy carries risks, including serious and potentially fatal bleeding, particularly for patients with clotting problems or those on other blood-thinning medications. FDA guidance recommends discontinuing treatment before planned surgeries and advises against its use in individuals with known severe allergies to any components of the drug.
In recognition of its role in treating a rare disease, Cablivi received Orphan Drug Designation, which provides incentives to encourage the development of treatments for conditions affecting small patient populations.
