Here’s a polished English translation of the Chinese title: **”China’s First and Currently Only¹ Recombinant ADAMTS13, Apadamtase Alfa / Cinaxadamtase Alfa for Injection, Approved”** This translation maintains the original structure and emphasis while ensuring clarity and professional tone.

ShanghaiJune 23, 2026 /PRNewswire/ — Takeda China today announced that its innovative drug, ADZYNMA^® (generic name: Apadamtase alfa/Cinaxadamtase alfa for injection), has been officially approved by the China National Medical Products Administration (NMPA) for on-demand or prophylactic enzyme replacement therapy (ERT) in children and adults with congenital thrombotic thrombocytopenic purpura (cTTP). Previously, Apadamtase alfa/Cinaxadamtase alfa for injection had been included in the priority review program of the Center for Drug Evaluation (CDE) of the NMPA^[2]. This approval makes it the first and currently only^[1] recombinant ADAMTS13 drug in China, offering a new ADAMTS13 enzyme replacement therapy option for Chinese cTTP patients and advancing cTTP diagnosis and treatment into a new stage of targeted etiology. At the same time, it will also bring a new option for preventive treatment for domestic cTTP patients, potentially reducing the risk of acute episodes and supporting long-term stable disease management^[3].

Dr. Liu Yan, President of Takeda China, stated: “Rare diseases are one of the key therapeutic areas where Takeda has long been deeply involved. Adhering to the original intention of ‘patient-first,’ we continue to introduce global innovative therapies to China, helping to improve the level of rare disease diagnosis and treatment and promoting high-quality medical resources to benefit more patients. Against the backdrop of the country’s continuous improvement of the rare disease diagnosis and treatment guarantee system and encouragement of faster access to innovative drugs, Apadamtase alfa/Cinaxadamtase alfa for injection has been accelerated for approval in China, bringing new treatment options for cTTP patients. In the future, Takeda will continue to collaborate with various parties to promote the construction of a standardized diagnosis and treatment system for cTTP, allowing more patients to embrace a better life.”

Extremely Rare and Potentially Life-Threatening: Urgent Treatment Needs for cTTP Patients

Congenital thrombotic thrombocytopenic purpura (cTTP) is an extremely rare and potentially life-threatening thrombotic microvascular disease caused by mutations in the ADAMTS13 gene^[4]. According to statistics, the annual incidence of thrombotic thrombocytopenic purpura (TTP) is 2 to 6 cases per million population^[4]. The disease has been included in China’s Second Batch of Rare Disease Catalog^[5], with cTTP accounting for approximately 5% of total cases^[4].

Due to genetic defects, cTTP patients have a severe deficiency of ADAMTS13 protease in their bodies, making them prone to the formation of microthrombi in the blood, which can rapidly lead to thrombocytopenia, hemolytic anemia, and multi-organ ischemic damage. The age of onset for this disease spans a wide range, from early childhood to adulthood and even during pregnancy^[6]. Its daily symptoms are insidious and easily overlooked, including occasional headaches, forgetfulness, skin bruising, abdominal pain, diarrhea, or nausea and vomiting^{[6, 7, 8, 9]}. When symptoms suddenly worsen, indicating entry into an acute episode, manifestations such as extreme fatigue, confusion, shortness of breath, rapid heart rate, yellowing of the skin or whites of the eyes, and dark urine may occur, requiring immediate medical attention. Without effective treatment, the mortality rate during acute episodes can exceed 90%; severe patients may suffer from persistent organ damage due to recurrent thrombotic events, leading to disease deterioration and even premature death^{[8, 10]}.

Traditionally, treatment for cTTP patients has primarily relied on fresh frozen plasma to increase ADAMTS13 activity levels in the body, thereby controlling the disease process. However, plasma therapy still has certain limitations. Data show that patients receiving traditional plasma therapy only achieve ADAMTS13 enzyme activity recovery to 19%^[3], with individual variability, which is not conducive to achieving long-term disease control nor meeting the core needs of preventing acute episodes and reducing the risk of long-term organ damage. Additionally, because treatment depends on allogeneic blood supply, it may also pose risks such as infection, allergies, and transfusion-related reactions, leaving the long-term disease management needs of patients yet to be fully addressed.

Professor Hu Yu, Chairman of the Hematology Branch of the Chinese Medical Association and President of Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, stated:“cTTP is a high-risk, rapidly progressing rare thrombotic microvascular disease. Patients not only face the risk of acute episodes but also need long-term attention to disease recurrence and organ damage, posing many challenges in clinical management. Although traditional plasma therapy can supplement ADAMTS13 activity, it still has limitations in maintaining stable enzyme activity levels, safety, and long-term prevention of recurrence. The approval of Apadamtase alfa/Cinaxadamtase alfa for injection provides a new treatment option for disease intervention in domestic cTTP. By compensating for the lack of ADAMTS13 enzyme activity in patients, this drug offers a new intervention pathway for disease management at the pathogenic mechanism level, further promoting the transformation of the diagnosis and treatment model from passive rescue to preventable and controllable long-term standardized management, providing a new clinical solution for improving the prognosis of high-risk patients.”

From Intervention to Prevention: Recombinant ADAMTS13 Opens a New Option for Precise Prevention and Treatment of cTTP

Apadamtase alfa/Cinaxadamtase alfa for injection is a recombinant ADAMTS13 protein drug that specifically supplements ADAMTS13 activity in the plasma of cTTP patients. By regulating the metabolism of von Willebrand factor (vWF) multimers, it reduces or eliminates the formation of vWF-platelet microthrombi^[11], thereby lowering the risk of acute/subacute TTP events and their related clinical manifestations^[12].

Results from a prospective, randomized, controlled, multicenter Phase III clinical study showed that patients (n=45) receiving prophylactic treatment with Apadamtase alfa/Cinaxadamtase alfa for injection experienced no acute TTP events during the treatment period, while among patients receiving plasma-based therapy (n=46), one acute TTP event occurred^[3]. Compared with plasma-based therapy, Apadamtase alfa/Cinaxadamtase alfa for injection reduced the incidence of thrombocytopenic events by 60%. After treatment with Apadamtase alfa/Cinaxadamtase alfa for injection, the mean maximum ADAMTS13 activity in patients was 101%, compared to 19% after standard therapy^[3]. According to the researchers’ conclusions, during prophylaxis with Apadamtase alfa/Cinaxadamtase alfa for injection in congenital TTP patients, ADAMTS13 activity reached approximately 100% of normal levels, adverse events were generally mild or moderate, the most common adverse reactions (incidence >10%) included headache, diarrhea, dizziness, upper respiratory tract infection, nausea, and migraine, and TTP events and manifestations were rare^[12].

Professor Wu Depei, Director of the National Clinical Research Center for Hematologic Diseases and Director of the Hematology Department at the First Affiliated Hospital of Soochow University, stated: “The treatment of cTTP requires long-term continuous intervention. The approval of Apadamtase alfa/Cinaxadamtase alfa for injection is of great value both for patient benefit and for the construction of a standardized diagnosis and treatment system for cTTP in China. Traditional plasma infusion and plasma exchange therapies have extremely stringent requirements for medical equipment, blood sources, and operational standards. As the world’s first recombinant ADAMTS13 protein drug^[13], Apadamtase alfa/Cinaxadamtase alfa for injection enables targeted therapy addressing the cause of the disease, can reduce the difficulty of clinical treatment and daily management, improve patient treatment compliance, and help China’s cTTP diagnosis and treatment enter a stage of precise treatment with equal emphasis on prevention and treatment, continuously and steadily improving the level of rare disease diagnosis and treatment in our country.”

Professor Wu Runhui, Chief Physician of the Hematology Department at Beijing Children’s Hospital, Capital Medical University, stated:“As a rare hematological disease, cTTP poses a high risk of morbidity in newborns and can lead to severe complications in early life, presenting certain challenges in clinical management. During clinical research and exploration, the application of Apadamtase alfa/Cinaxadamtase alfa for injection has provided new ideas for intervention in pediatric cTTP patients, and to some extent, helps enhance the medical community’s understanding of early identification and intervention strategies for cTTP, offering a reference for the exploration of diagnosis and treatment in pediatric rare diseases.”

Dr. Wang Lin, Regional Head of Global R&D and Head of Takeda R&D China and Asia Pacific Center, stated: “We are very pleased to see that Apadamtase alfa/Cinaxadamtase alfa for injection has been approved in China, bringing a new treatment option for cTTP patients. As the world’s first recombinant ADAMTS13 enzyme replacement therapy^[13], it intervenes in the cause of cTTP while covering both acute episode management and long-term preventive treatment, potentially providing Chinese patients with a precise disease management plan and improving their quality of life. As a research-driven biopharmaceutical company, Takeda will continue to leverage its global R&D advantages to accelerate the delivery of more innovative achievements to Chinese patients earlier, continuously contributing to improving the level of diagnosis and treatment and drug accessibility in the field of rare diseases.”

Approval Date: June 2026
Approval Number: C-ANPROM/CN/ADZ/0005

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