Heo Young-A
Springer Nature, Private Bag 65901, Mairangi Bay, Auckland, 0754, New Zealand.
Drugs. 2024 Apr;84(4):467-472. doi: 10.1007/s40265-024-02007-6.
Apadamtase alfa (ADAMTS13, recombinant-krhn; ADZYNMA), a human recombinant form of a disintegrin and metalloproteinase with thrombospondin motifs 13 (ADAMTS13), is being developed by Takeda under license from KM biologics for thrombotic thrombocytopenic purpura (TTP) and sickle cell disease. On 9 November 2023, apadamtase alfa was approved in the USA for prophylactic and on-demand enzyme replacement therapy (ERT) in paediatric and adult patients with congenital TTP. Apadamtase alfa is under regulatory review for congenital TTP in the EU and Japan, and is under clinical development for immune-mediated TTP in several countries worldwide. Clinical development of apadamtase alfa for vaso-occlusive crisis related to sickle cell anaemia is underway in the USA. This article summarizes the milestones in the development of apadamtase alfa leading to this first approval in the USA for congenital TTP.
阿帕达马酶α（ADAMTS13，重组人源化；ADZYNMA）是一种具有血小板反应蛋白基序的去整合素和金属蛋白酶13（ADAMTS13）的重组人源形式，由武田公司根据KM生物制品公司的许可进行开发，用于治疗血栓性血小板减少性紫癜（TTP）和镰状细胞病。2023年11月9日，阿帕达马酶α在美国被批准用于先天性TTP儿科和成人患者的预防性和按需酶替代疗法（ERT）。阿帕达马酶α正在欧盟和日本接受先天性TTP的监管审查，并在全球多个国家进行免疫介导TTP的临床开发。阿帕达马酶α用于镰状细胞贫血相关血管闭塞性危机的临床开发正在美国进行。本文总结了阿帕达马酶α开发过程中的里程碑事件，这些事件促成了其在美国首次获批用于先天性TTP。
