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Apadamtase alfa /cinaxadamtase alfa (TAK-755) receives Orphan Drug Designation

- Apadamtase alfa /cinaxadamtase alfa receives orphan drug designation from the Ministry of Health, Labour and Welfare for the expected indication of Thrombotic thrombocytopenic purpura (TTP)

 

Takeda today announced that apadamtase alfa/cinaxadamtase alfa (TAK-755) has received orphan drug designation for the treatment of thrombotic thrombocytopenic purpura (TTP) from the Ministry of Health, Labour and Welfare.

TTP is a fatal and rare type of thrombotic microangiopathy associated with von Willebrand factor (VWF)-induced platelet adhesion and aggregation. The disease is designated as intractable. As the first recombinant ADAMTS13 drug targeting TTP, apadamtase alfa/cinaxadamtase alfa (TAK-755) is being developed globally for the treatment of congenital TTP (cTTP) and acquired (immune) TTP (iTTP).

Thrombotic Thrombocytopenic Purpura (TTP)

TTP is characterized by a congenital or acquired (immune) deficiency of ADAMTS13 activity that causes a large accumulation of VWF polymer. It was designated as intractable disease number 64 in Japan on January 1, 2015. The VWF polymer binds to platelets, causes them to aggregate and generates microthrombi, resulting in a variety of clinical manifestations ranging from recurrent thrombocytopenia, microangiopathic hemolytic anemia and mild neurological symptoms to ischemic organ damage, including cerebral and cardiovascular disorders with severe neurological symptoms and renal damage. The symptoms of TTP can sometimes be severe, with skin hemorrhage (purpura) and long-term complications such as depression, cognitive dysfunction and hypertension, which markedly affect the prognosis. The Department of Transfusion Medicine of Nara Medical University is currently collecting blood samples to place in the Thrombotic Microangiopathy Register. At present, the register lists 725 patients with an ADAMTS13 activity of less than 10% of the normal value. These individuals are diagnosed as having TTP, with 69 having cTTP and 656 having iTTP.1

Apadamtase alfa/cinaxadamtase alfa (TAK-755)

Apadamtase alfa/cinaxadamtase alfa (TAK-755) is the first recombinant ADAMTS13 product to be developed anywhere in the world for the treatment of congenital TTP (cTTP) and acquired (immune) TTP (iTTP). As there is no drug currently approved for the treatment of cTTP, fresh-frozen plasma is widely used for the treatment or prevention of acute symptoms, and plasma exchange therapy is recommended for the acute treatment of iTTP, however both forms of treatment234 come with complications – complications that, it is hoped, TAK-755 will reduce. A Phase III clinical study of cTTP is underway in Japan in collaboration with international partners, and a Phase II clinical trial is currently underway for iTTP overseas. TAK-755 was granted orphan drug designation for the treatment and prevention of TPP, including the congenital, acquired, idiopathic and secondary thrombocytopenic purpura forms of the disease in the US in July 2008 and for the treatment of TPP in Europe in December 2008.

About Orphan Drugs 

Drugs are given orphan drug designation by the Ministry of Health, Labour and Welfare upon consultation with the Pharmaceutical Affairs and Food Sanitation Council in accordance with Article 77-2, Paragraph 1 of the Pharmaceuticals and Medical Devices Act if they meet all of the following criteria.

  • There must be fewer than 50,000 patients in Japan for whom the drug is intended. However, if the drug is for a designated intractable disease, the number of patients is instead limited to the number specified in Article 5, Paragraph 1 of the Intractable Diseases Act (approximately one-thousandth of the population).
  • In addition to being indicated for serious illnesses, there should be no appropriate alternative drug or treatment, or the product should have a particularly high medical value, such as being expected to have a significantly higher level of efficacy or safety than existing drugs.
  • There should be a theoretical rationale for the use of the product for the target disease, and the development plan should be appropriate.

 

About Takeda Pharmaceutical Company Limited 

Takeda Pharmaceutical Company Limited (TSE: 4502/NYSE: TAK) is a global, values-based, R&D-driven biopharmaceutical leader headquartered in Japan, committed to discover and deliver life-transforming treatments, guided by our commitment to patients, our people and the planet. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Rare Genetics and Hematology, Neuroscience, and Gastroenterology (GI). We also make targeted R&D investments in Plasma-Derived Therapies and Vaccines. We are focusing on developing highly innovative medicines that contribute to making a difference in people’s lives by advancing the frontier of new treatment options and leveraging our enhanced collaborative R&D engine and capabilities to create a robust, modality-diverse pipeline. Our employees are committed to improving quality of life for patients and to working with our partners in health care in approximately 80 countries and regions. For more information, visit https://www.takeda.com

Disclaimer 

The drug information contained herein is intended to disclose corporate information. Nothing contained in this document should be considered a solicitation, promotion, or indication for any prescription drug, including those currently under development. 

 

1 MHLW Grant-in-Aid for Scientific Research on Intractable Disease Policy Research, Research Project on Blood Coagulation Disorders, etc., FY2021 Summary/Supplemental Research Report (see November 24, 2022)  

https://ketsuekigyoko.org/wp-content/uploads/2022/06/b2af8a3aa9cb503f1ac6ab42b966b509.pdf

2 Reutter JC, Sanders KF, Brecher ME, Jones HG, Bandarenko N. Incidence of allergic reactions with fresh frozen plasma or cryo-supernatant plasma in the treatment of thrombotic thrombocytopenic purpura. J Clin Apher. 2001;16(3):134-8

3 Pandey S, Vyas G. Adverse effects of plasma transfusion. Transfusion. 2012;52 Suppl 1:65S-79S.

4 Hughes C, McEwan JR, Longair I, Hughes S, Cohen H, Machin S, et al. Cardiac involvement in acute thrombotic thrombocytopenic purpura: association with troponin T and IgG antibodies to ADAMTS 13. J Thromb Haemost. 2009;7(4):529-36.

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