Takeda's (TAK.US) Adzynma, an injectable apadazyme alpha, has been designated for priority review by the regulatory authorities.

On March 19, the National Medical Products Administration's Center for Drug Evaluation and Research (CDE) officially announced that Takeda's (TAK.US) Adzynma (apadamtase alfa) for injection was proposed to be included in the priority review, which is indicated for on-demand or prophylactic enzyme replacement therapy (ERT) in children and adults with congenital thrombotic thrombocytopenic purpura (cTTP). According to public information, it is Takeda's enzyme replacement therapy apadamtase alfa, which was approved by the US FDA in November 2023, becoming the first enzyme replacement therapy approved by the FDA for the treatment of congenital thrombotic thrombocytopenic purpura (cTTP) in adults and children. cTTP is a rare chronic coagulation disorder caused by the deficiency of ADAMTS13 enzyme, leading to the accumulation of multimers of von Willebrand factor (VWF) in the blood. Adzynma (apadamtase alfa) is a recombinant ADAMTS13 protein that can replace the deficient or dysfunctional ADAMTS13 enzyme. By supplementing the function of ADAMTS13 enzyme, the product can effectively improve the acute and chronic symptoms of cTTP, as well as other complications caused by ADAMTS13 deficiency, thus improving the quality of life and lifespan of cTTP patients.