FDA Grants Breakthrough Therapy Designation to Daiichi Sankyo's FLT3 Inhibitor Quizartinib for Relapsed/Refractory FLT3-ITD AML
TOKYO and MUNICH and BASKING RIDGE, N.J., Aug. 1, 2018 /PRNewswire/ -- Daiichi Sankyo Company, Ltd. (hereafter, Daiichi Sankyo) announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to quizartinib, an investigational FLT3 inhibitor, for the treatment of adult patients with relapsed/refractory FLT3-ITD acute myeloid leukemia (AML).
"There have been limited advances over the past several decades for the treatment of relapsed/refractory FLT3-ITD AML, a very aggressive form of the disease associated with poor prognosis. Quizartinib is the first FLT3 inhibitor to significantly improve overall survival as an oral, single agent compared to chemotherapy in patients with relapsed/refractory AML with FLT3-ITD, an underlying driver of this subtype of AML," said Arnaud Lesegretain, Vice President, Oncology Research and Development and Head, AML Franchise, Daiichi Sankyo. "We are excited that quizartinib has received Breakthrough Therapy designation and we look forward to working closely with the FDA to bring this potential new treatment option to patients as quickly as possible."
Breakthrough Therapy designation is designed to expedite the development and regulatory review of medicines that may demonstrate substantial benefit over currently approved treatments, in order to more quickly bring new treatment options to patients with serious diseases. Significant unmet medical need exists in relapsed/refractory AML, as available treatment options are limited and there are no approved targeted therapies for patients with relapsed/refractory FLT3-ITD AML.
The designation was granted based on the results of the pivotal phase 3 QuANTUM-R study of quizartinib, which were presented during the plenary program at the 23rd Congress of the European Hematology Association in June 2018. QuANTUM-R is the first randomized phase 3 study to show that a FLT3 inhibitor, quizartinib, prolongs overall survival as an oral, single agent compared to chemotherapy in patients with relapsed/refractory FLT3-ITD AML.
The safety profile observed in QuANTUM-R appears consistent with that observed at similar doses in the quizartinib clinical development program. Incidence of treatment-emergent adverse events was comparable between patients who received single agent quizartinib (n=241) and those who received salvage chemotherapy (n=94). The most common adverse events (>30 percent, any Grade) in patients treated with quizartinib included nausea, thrombocytopenia, fatigue, musculoskeletal pain, pyrexia, anemia, neutropenia, febrile neutropenia, vomiting and hypokalemia.
In addition to Breakthrough Therapy designation, quizartinib has been granted Fast Track designation by the FDA for the treatment of relapsed/refractory AML. Quizartinib also has been granted Orphan Drug designation by both the FDA and the European Medicines Agency (EMA) for the treatment of AML. Quizartinib is an investigational agent that has not been approved for any indication in any country. Safety and efficacy have not been established.
About FLT3-ITD Acute Myeloid Leukemia
FLT3 gene mutations are one of the most common genetic abnormalities in AML.3 FLT3-ITD is the most common FLT3 mutation, affecting approximately one in four patients with AML.4,5,6,7 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
About Daiichi Sankyo Cancer Enterprise
About Daiichi Sankyo
References 1. Leukemia & Lymphoma Society. Facts 2015-2016. 2016. 2. American Cancer Society. Key Statistics for AML. 2018. 3. Small D. Am Soc Hematol Educ Program. 2006;178-84. 4. Schneider F, et al. Ann Hematol. 2012;91:9-18. 5. Santos FPS, et al. Cancer. 2011;117(10):2145-2155. 6. Kainz B, et al. Hematol J. 2002;3:283-289. 7. Kottaridis PD, et al. Blood. 2001;98(6):1752-1759. 8. Wagner K, et al. Haematol. 2011;96(5):681-686. 9. Brunet S, et al. J Clin Onc. 2012;30(7):735-741.
Date of prep: July 2018 QZT/18/0004
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