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Daiichi Sankyo’s FLT3 Inhibitor Quizartinib Receives Orphan Drug Designation from Japanese MHLW for FLT3-Mutated AML
September 11, 2018
- Orphan drug designation in Japan for quizartinib follows recent Breakthrough Therapy designation received from the U.S. Food and Drug Administration (FDA)
- Significant unmet medical need exists for AML in Japan with no approved targeted therapies for patients with FLT3-ITD AML, a very aggressive form of the disease associated with poor prognosis
- Quizartinib is the first FLT3 inhibitor to demonstrate a survival benefit in a randomized, phase 3 study in patients with relapsed/refractory FLT3-ITD AML
Tokyo, Munich and Basking Ridge, NJ – (September 11, 2018) – Daiichi Sankyo Company, Limited (hereafter, Daiichi Sankyo) announced 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).
“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 Kouichi 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.
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
About Daiichi Sankyo Cancer Enterprise
The mission of Daiichi Sankyo Cancer Enterprise is to leverage our world-class, innovative science and push beyond traditional thinking to create meaningful treatments for patients with cancer. We are dedicated to transforming science into value for patients, and this sense of obligation informs everything we do. Anchored by three pillars including our investigational Antibody Drug Conjugate Franchise, Acute Myeloid Leukemia Franchise and Breakthrough Science, we aim to deliver seven distinct new molecular entities over eight years during 2018 to 2025. Our powerful research engines include two laboratories for biologic/immuno-oncology and small molecules in Japan, and Plexxikon Inc., our small molecule structure-guided R&D center in Berkeley, CA. Compounds in pivotal stage development include: DS-8201, an antibody drug conjugate (ADC) for HER2-expressing breast, gastric and other cancers; quizartinib, an oral selective FLT3 inhibitor, for newly-diagnosed and relapsed/refractory FLT3-ITD acute myeloid leukemia (AML); and pexidartinib, an oral CSF1R inhibitor, for tenosynovial giant cell tumor (TGCT). For more information, please visit: www.DSCancerEnterprise.com.
About Daiichi Sankyo
Daiichi Sankyo Group is dedicated to the creation and supply of innovative pharmaceutical products to address diversified, unmet medical needs of patients in both mature and emerging markets. With over 100 years of scientific expertise and a presence in more than 20 countries, Daiichi Sankyo and its 15,000 employees around the world draw upon a rich legacy of innovation and a robust pipeline of promising new medicines to help people. In addition to a strong portfolio of medicines for hypertension and thrombotic disorders, under the Group’s 2025 Vision to become a “Global Pharma Innovator with Competitive Advantage in Oncology,” Daiichi Sankyo research and development is primarily focused on bringing forth novel therapies in oncology, including immuno-oncology, with additional focus on new horizon areas, such as pain management, neurodegenerative diseases, heart and kidney diseases, and other rare diseases. For more information, please visit: www.daiichisankyo.com. Daiichi Sankyo, Inc., headquartered in Basking Ridge, New Jersey, is a member of the Daiichi Sankyo Group. For more information on Daiichi Sankyo, Inc., please visit: www.dsi.com.
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- 8. Brunet S, et al. J Clin Onc. 2012;30(7):735-741.
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