On September 11, 2018 Daiichi Sankyo Company, Limited (hereafter, Daiichi Sankyo) reported that quizartinib, an investigational FLT3 inhibitor, has been granted Orphan Drug designation by the Japan Ministry of Health, Labour and Welfare (MHLW) for the treatment of FLT3-mutated acute myeloid leukemia (AML) (Press release, Daiichi Sankyo, SEP 11, 2018, View Source [SID1234529382]).

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"There is a critical need for new treatment options for patients with FLT3-ITD AML, especially given the poor prognosis associated with this subtype of AML," said Koichi Akahane, PhD, MBA, Executive Officer, Head of Oncology Function, R&D Division, Daiichi Sankyo. "Following the recent U.S. FDA Breakthrough Therapy designation for quizartinib, receiving Orphan Drug designation is another important regulatory milestone that will help accelerate the development of quizartinib in Japan. We look forward to working closely with the Japan MHLW to bring quizartinib to patients as quickly as possible."

The Japan MHLW Orphan Drug designation system is designed to promote research activities and support the development of orphan drugs for serious, difficult-to-treat diseases that affect fewer than 50,000 patients in Japan, and for which significant unmet medical need exists. An investigational compound can qualify for Orphan Drug designation if there is no approved alternative treatment option or if high efficacy or safety compared to existing treatment options is expected. Compounds receiving Orphan Drug designation qualify for several measures intended to support development, including, but not limited to, guidance and subsidies for research and development activities, priority consultation for clinical development and priority review of applications.

Quizartinib is the first FLT3 inhibitor to prolong overall survival as an oral, single agent compared to chemotherapy in a randomized, phase 3 trial (QuANTUM-R) in patients with relapsed/refractory FLT3-ITD

AML. Results of QuANTUM-R were presented during the plenary program at the 23rd Congress of the

European Hematology Association in June 2018.

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 and those who received salvage chemotherapy. 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.

About Quizartinib

Quizartinib, the lead investigational agent in the AML Franchise of the Daiichi Sankyo Cancer Enterprise, is an oral selective FLT3 inhibitor currently in phase 3 development for relapsed/refractory FLT3-ITD AML (QuANTUM-R) in the U.S. and EU and newly-diagnosed FLT3-ITD AML (QuANTUM-First) in the U.S., EU and Japan; and phase 2 development for relapsed/refractory FLT3-ITD AML in Japan.

In addition to Orphan Drug designation in Japan, quizartinib has been granted Breakthrough Therapy designation for the treatment of adult patients with relapsed/refractory FLT3-ITD AML and Fast Track designation for the treatment of relapsed/refractory AML by the U.S. Food and Drug Administration (FDA). Quizartinib also has received 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
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 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.7,8