Japan approves Takeda’s ADZYNMA to treat ‘ultra-rare’ disorder cTTP

Takeda announced that the Japanese Ministry of Health, Labour and Welfare has approved the use of ADZYNMAor apadamtase alfa /cinaxadamtase alfa, for the treatment of congenital thrombotic thrombocytopenic purpura or cTTP, for individuals 12 years of age and older.1 ADZYNMA is the first and only approved recombinant ADAMTS13 protein designed to address an unmet medical need in people with cTTP by replacing the deficient ADAMTS13 enzyme. cTTP is an ultra-rare, chronic blood clotting disorder caused by a deficiency in the ADAMTS13 enzyme.2 It is associated with acute events and debilitating chronic symptoms or thrombotic thrombocytopenic purpura or TTP, manifestations, which can include thrombocytopenia, microangiopathic hemolytic anemia, headache and abdominal pain.2,3,4 When left untreated, acute TTP events have a mortality rate of greater than90%.2,4 “The approval of ADZYNMA is an important milestone for people living with cTTP in Japan, who had limited treatment options and now have the first treatment option specifically approved to treat this ultra-rare condition,” said Yasushi Kajii, Head, R&D Japan Region at Takeda. “Developing innovative treatments that make a difference in the lives of patients is at the heart of what we do. With this approval, we are proud to support the cTTP community with new possibilities and continue our 70-plus year commitment to the rare disease community.”