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- 11/07/2018

EMA Validates and Grants Accelerated Assessment of Marketing Authorization Application for Daiichi Sankyo’s FLT3 Inhibitor Quizartinib

TKS News

Daiichi Sankyo Company, Limited (hereafter, Daiichi Sankyo) announced that the European Medicines Agency (EMA) validated for review and granted accelerated assessment to the Marketing Authorization Application (MAA) for quizartinib for the treatment of adults with relapsed or refractory acute myeloid leukemia (AML) which is FLT3-ITD positive.

Validation confirms that the application is complete and commences the scientific review process by the EMA’s Committee for Medicinal Products for Human Use (CHMP). Accelerated assessment is given to products expected to be of major interest for public health and therapeutic innovation and can significantly reduce the review timelines.
The EU MAA is based on results of the pivotal phase 3 QuANTUM-R study of quizartinib, which was the first randomized phase 3 study to show that a FLT3 inhibitor prolonged overall survival as an oral, single agent compared to chemotherapy in patients with relapsed/refractory FLT3-ITD AML. Topline results of the phase 3 QuANTUM-R study were presented during the plenary program at the 23rd Congress of the European Hematology Association in June 2018. “The accelerated assessment of the quizartinib MAA underscores the significant unmet need for patients with relapsed/refractory FLT3-ITD AML, a very aggressive form of the disease with no approved targeted treatment options in Europe,” said Arnaud Lesegretain, Vice President, Oncology Research and Development and Head, AML Franchise, Daiichi Sankyo. “Achieving both these milestones are significant next steps and we look forward to working with the EMA to bring this important potential new targeted treatment option to patients in the EU.”
Quizartinib is currently under accelerated regulatory review with the Japan Ministry of Health, Labour and Welfare (MHLW) for the treatment of adult patients with relapsed/refractory FLT3-ITD AML. Submission in the U.S. remains on track for the second half of fiscal year 2018. In the QuANTUM-R study, the median treatment duration with quizartinib was 4 cycles of 28 days each versus 1 cycle in the salvage chemotherapy arm. Incidence of treatment-emergent adverse events was comparable between patients who received single agent quizartinib and those who received salvage chemotherapy. The most common adverse drug reactions (>30 percent, any Grade) in patients treated with
quizartinib included infections, bleeding, nausea, asthenic conditions, pyrexia, febrile neutropenia and vomiting, and the most common Grade ≥ 3 adverse drug reactions (>20 percent) were infection and febrile neutropenia. The most common laboratory adverse reactions (incidence >50 percent) were decreased white blood cell count, decreased lymphocyte count, decreased hemoglobin, decreased neutrophil count and decreased platelet count. The safety profile observed in QuANTUM-R appears consistent with that observed at similar doses in the quizartinib clinical development program. About FLT3-ITD Acute Myeloid Leukemia AML is an aggressive blood and bone marrow cancer that causes uncontrolled growth and accumulation of malignant white blood cells that fail to function normally and interfere with the production of normal blood cells.1 FLT3 gene mutations are one of the most common genetic abnormalities in AML.2 FLT3-ITD is the most common FLT3 mutation, affecting approximately one in four patients with AML.
3,4,5,6 FLT3-ITD is a driver mutation that presents with high leukemic burden and has poor prognosis and a significant impact on disease management for patients with AML.4,7
Patients with FLT3-ITD AML have a worse overall prognosis, including an increased incidence of relapse, an increased risk of death following relapse and a higher likelihood of relapse following hematopoietic stem cell transplantation as compared to those without this mutation.8,9

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