Takeda Submits New Drug Application in China for Adzynma (Apadamtase Alfa), Pioneering a New Era in cTTP Treatment

On April 9, the CDE official website announced,Takeda's submission of the marketing application for Adzynma (Apadazyme Alfa) for injection has been accepted.

This innovative enzyme replacement therapy for the treatment of congenital thrombotic thrombocytopenic purpura (cTTP) has beenApproved by the U.S. FDA in November 2023, becoming the first enzyme replacement therapy for cTTP to receive FDA approval,For adult and pediatric patients.

Source: CDE official website

cTTP is a rare chronic coagulation disorder caused by the absence or dysfunction of the ADAMTS13 enzyme, leading to the accumulation of von Willebrand Factor (VWF) multimers in the blood, triggering platelet aggregation and adhesion, and subsequently causing abnormal clotting in small blood vessels. If left untreated, the mortality rate of acute TTP can exceed 90%.

Apadase α (TAK-755) is a recombinant ADAMTS13 protein that can replace missing or dysfunctional ADAMTS13 enzymes, restoring normal blood coagulation function, alleviating cTTP symptoms, and improving patients' quality of life and life expectancy. This therapy has been approved for marketing in the United States, the European Union, and Japan, and has received Fast Track designation, Orphan Drug designation, and Priority Review designation from the FDA.

In 2024, the phase 3 clinical trial data for Apadazyme Alfa was published in The New England Journal of Medicine (NEJM). The trial results showed that during the prevention period,No acute TTP events occurred in the Apadase α group, and the patients' ADAMTS13 activity averaged 101%, significantly higher than the 19% in the standard treatment group.In addition, no patients in the aparadase alfa treatment group discontinued or stopped medication due to adverse events, while 8 patients in the standard treatment group experienced such situations.

In November 2023, Takeda announced FDA approval of the marketing application for Apadagene Alfa. This application was supported by efficacy, pharmacokinetic, safety, and tolerability data from randomized controlled trials in cTTP, along with positive long-term safety and efficacy data. This Phase 3 trial aimed to evaluate the benefits of Apadagene Alfa across multiple clinically relevant endpoints. Interim results announced by Takeda in January 2023 showed that TAK-755 reduced the occurrence of thrombocytopenic events by 60% compared to plasma-based therapies, a key indicator of disease activity in cTTP and the most frequently observed clinical symptom.

Summary

Apada enzyme α was included in the priority review by the CDE in March 2023, for on-demand or prophylactic enzyme replacement therapy (ERT) in patients with cTTP.。The acceptance of the marketing application for Apada Enzyme α in China marks Takeda's continued progress in the development of innovative therapies and brings new hope to patients with cTTP.

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