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Quizartinib receives Orphan Drug designation for FLT3-mutated AML in Japan
By: News Feed | Last updated: 12th September 2018 | In: Haematology, Medical News Asia, Oncology, Targeted Therapies
AC220, AML, Daiichi Sankyo, FLT3, HSCT, leukaemia, quizartinib
TOKYO and MUNICH and BASKING RIDGE, N.J., September 11, 2018 — The Japan Ministry of Health, Labour and Welfare (MHLW) has granted quizartinib (AC220, Daiichi Sankyo), an investigational inhibitor of FLT3, the Orphan Drug designation for the treatment of acute myeloid leukaemia (AML) harbouring FLT3-mutations.
AML is an aggressive haematological malignancy leading to uncontrolled growth and accumulation of malignant, non-functional white blood cells thus interfering with the production of normal blood cells.1Leukemia & Lymphoma Society. Facts 2015-2016. Mutations in the FLT3 gene are a common genetic abnormality in AML.2Small D. Am Soc Hematol Educ Program. 2006;178-184.. One in four AML patients are reported to harbour FLT3-ITD mutations and have a poor overall prognosis with an increased incidence of relapse and risk of death following relapse, as well as a higher chance of relapse following haematopoietic stem cell transplantation (HSCT).3 Schneider F, et al. Ann Hematol. 2012;91:9-18.,4Santos FPS, et al. Cancer. 2011;117(10):2145-2155.,5Kainz B, et al. Hematol J. 2002;3:283-289.,6Kottaridis PD, et al. Blood. 2001;98(6):1752-1759.,7Wagner K, et al. Haematol. 2011;96(5):681-686.,8Brunet S, et al. J Clin Onc. 2012;30(7):735-741.
In the randomised Phase III QuANTUM-R trial single agent, oral quizartinib prolonged the overall survival (OS) compared to chemotherapy in patients with relapsed or refractory FLT3-ITD AML. The most common any-grade adverse events (>30%) in patients treated with quizartinib included nausea, thrombocytopenia, fatigue, musculoskeletal pain, pyrexia, anaemia, neutropenia, febrile neutropenia, vomiting and hypokalemia.
A study with quizartinib in newly diagnosed patients with FLT3-ITD AML, the QuANTUM-First trial, is ongoing in the United States (US), Europe, and Japan.
The Orphan Drug designation system by the Japan MHLW is designed to promote research and support the development of therapies for severe and difficult-to-treat diseases affecting less than 50,000 Japanese patients, and for which a major unmet medical need.
Quizartinib received the “Orphan Drug” designation for the treatment of AML by both the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA). Moreover, the US FDA granted quizartinib “Breakthrough Therapy” designation for the treatment of adult relapsed or refractory FLT3-ITD AML patients and “Fast Track” designation for the treatment of relapsed or refractory AML.
- Leukemia & Lymphoma Society. Facts 2015-2016.
- Small D. Am Soc Hematol Educ Program. 2006;178-184.
- Schneider F, et al. Ann Hematol. 2012;91:9-18.
- Santos FPS, et al. Cancer. 2011;117(10):2145-2155.
- Kainz B, et al. Hematol J. 2002;3:283-289.
- Kottaridis PD, et al. Blood. 2001;98(6):1752-1759.
- Wagner K, et al. Haematol. 2011;96(5):681-686.
- Brunet S, et al. J Clin Onc. 2012;30(7):735-741.
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This article is not medical advice. Patients should seek personal assessment by a licenced specialist. Physicians are recommended to read the full publication(s) as cited in the article before making medical decisions. This article does not supersede nor replace the published article(s).
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