Takeda's Innovative Therapy Adzynma (Apadamtase Alfa) Recommended for Priority Review in China, Restores Key Enzyme Levels to 100%

Mar 19, 2025 14:52 CST Updated 14:52

Takeda

Biopharmaceutical Manufacturer

▎WuXi AppTec Content Team Report

Today (March 19), the official website of the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) has just announced that Takeda Pharmaceutical Company Limited has submittedInjectable Apadase α (Adzynma）Proposed for Priority Review, Applicable toOn-demand or prophylactic enzyme replacement therapy (ERT) for pediatric and adult patients with congenital thrombotic thrombocytopenic purpura (cTTP). Public information shows that this was developed by Takeda.Enzyme Replacement Therapy Apadamtase Alfa, which was approved by the U.S. FDA in November 2023, is the first enzyme replacement therapy approved by the FDA for the treatment of congenital thrombotic thrombocytopenic purpura (cTTP) in adult and pediatric patients.

Screenshot source:CDE Official Website

cTTP is a rare chronic coagulation disorder,The cause of the disease is the absence of the ADAMTS13 enzyme, leading to the accumulation of von Willebrand Factor (VWF) multimers in the blood.. The accumulation of these VWF multimers leads to platelet aggregation and adhesion, causing abnormal clotting in small blood vessels, and presenting with hemolytic anemia and thrombocytopenia. Without treatment, the mortality rate for acute TTP exceeds 90%.[3,4]。

Apada enzyme α (TAK-755) is aRecombinant ADAMTS13 Protein, which can replace the missing or malfunctioning ADAMTS13 enzymeBy supplementing the function of ADAMTS13 enzyme, this product can effectively improve both acute and chronic symptoms of cTTP, as well as other complications caused by ADAMTS13 deficiency, thereby enhancing the quality of life and life expectancy of cTTP patients. This product has previously received Fast Track designation, Orphan Drug designation, and Priority Review designation from the FDA for the prevention and treatment of cTTP. Currently, the product has been approved for marketing in the United States, the European Union, and Japan.

In November 2023, Takeda announced FDAApprovalThe Marketing Application of Apadazyme Alfa.This application is supported by efficacy, pharmacokinetic, safety, and tolerability data from randomized controlled trials in cTTP, and is backed by positive long-term safety and efficacy data. This Phase 3 trial aims to evaluateAparadase αBenefits across multiple clinically relevant endpoints. Interim results announced by Takeda in January 2023 showed,Compared with plasma-based therapies, TAK-755 reduced the occurrence of thrombocytopenia events by 60%, an important indicator of disease activity in cTTP and the most frequently observed clinical symptom.。

In May 2024, data from a Phase 3 trial of Apadagene Alfa were published in The New England Journal of Medicine (NEJM). In this open-label, phase 3 crossover trial, patients were randomly assigned in a 1:1 ratio to receive either 6 months of treatment with velmanase alfa (40 IU per kilogram of body weight, intravenously) or standard care, followed by crossover to the alternative treatment; thereafter, all patients received velmanase alfa for an additional 6 months.

The results showed,No acute TTP events occurred during the apadase alfa prevention period, while one patient experienced an acute TTP event during the standard treatment prevention period.No patients in the Apadazyme Alfa group discontinued or interrupted treatment due to adverse events, compared to 8 cases in the standard treatment group.After treatment with Apadamate Alfa, the average maximum ADAMTS13 activity in patients was 101%, compared to 19% after standard treatment.According to the conclusions drawn by the researchers,In the treatment of patients with congenital TTPApada enzyme αDuring the prevention period, ADAMTS13 activity reached approximately 100% of the normal level.Adverse events are generally mild or moderate, and TTP events and manifestations are rare.

According to the official website of the Chinese Clinical Trial Registry and Information Disclosure Platform, Takeda has launched an international multicenter (including China) Phase 3b, open-label, continuation study for the prophylactic and on-demand treatment of severe congenital thrombotic thrombocytopenic purpura with TAK-755.The study targets the acute treatment and long-term prophylactic treatment of TTP in patients with congenital ADAMTS13 deficiency (cTTP).The study has now completed recruitment.

The inclusion of this innovative therapy in the priority review in China means that it is expected to reach patients more quickly.

References:

[1] Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) of China.Retrieved Mar 19，2025, From https://www.cde.org.cn/main/xxgk/listpage/4b5255eb0a84820cef4ca3e8b6bbe20c

[2]FDA Approves First Treatment for Patients with Rare Inherited Blood Clotting Disorder. Retrieved November 9, 2023 from https://www.prnewswire.com/news-releases/fda-approves-first-treatment-for-patients-with-rare-inherited-blood-clotting-disorder-301983923.html

[3]Van Dorland H et al. Haematologica. 2019;104:2107-16.

[4]Joly BS et al. Blood. 2017;129(21):2836–2846.

[5]Takeda Receives European Commission Approval for ADZYNMA®▼ (Recombinant ADAMTS13) as the First and Only Recombinant ADAMTS13 Replacement Therapy for Congenital Thrombotic Thrombocytopenic Purpura (cTTP). Retrieved Aug 7，2024, From https://www.takeda.com/newsroom/newsreleases/2024/european-commission-approval-for-adzynma/

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