Takeda announced that the Japanese Ministry of Health, Labour and Welfare has approved the use of ADZYNMA (apadamtase alfa /cinaxadamtase alfa) for the treatment of congenital thrombotic thrombocytopenic purpura (cTTP) for individuals 12 years of age and older. 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. ADZYNMA is an ultra-rare, chronic blood clotting disorder caused by a deficiency in the ADAMTS13 enzyme.

It is associated with acute events and debilitating chronic symptoms or thrombotic thromssocytopenic purpura ("TTP") manifestations, which can include thrombocytopenia, microangiopathic hemolytic anemia, headache and abdominal pain. When left untreated, acute TTP events have a mortality rate of >90%. ADZYNMA is also approved by the U.S. Food and Drug Administration (FDA) for the prophylactic and on-demand treatment of adult and pediatric patients with congenital thrombocytopenic Purpura (cTTP).

ADZYNMA was previously granted Orphan Drug Designation (ODD) by the U.S. FDA for the treatment and prevention of TTP, including its acquired idiopathic and secondary forms, and by the European Medicines Agency (EMA) and Japan's Ministry of Health, Labour and welfare (MHLW) for the treatment of TTP.