Single-drug first-line treatment AML has significant efficacy The first FLT3 targeted inhibitor applied for market

Daiichi Sankyo Pharmaceuticals, a Japanese pharmaceutical company, recently announced that it has submitted a new drug application (NDA) to the Japanese Ministry of Health, Labour and Welfare (MHLW) for the treatment of adult patients with the targeted anti-cancer drug quizartinib for relapse/refractic FLT3-ITD acute myeloid leukemia (AML). The application is based on data from QuANTUM-R, a key randomized Phase III clinical study conducted in the United States, the European Union, Asia (excluding Japan), and an open label single-group Phase II study of adult patients treating relapse/recurring FLT3-ITD AML conducted in Japan. It is worth mentioning that quizartinib is the first FLT3 inhibitor to demonstrate survival benefits compared to chemotherapy in the treatment of recurring/refractic FLT3-ITD AML Random Phase III studies.
in the QuANTUM-R study, quizartinib oral monotherapy significantly reduced the risk of death by 24% (HR=0.76, p=0.0177,95% CI:0.58-0.98) and significantly longer total lifetime compared to salvage chemotherapy (Middle OS: 6.2 months (bilateral test 95% CI: 5.3-7.2) vs 4.7 months (bilateral test 95% CI: 4.0-5.5)); quizartinib treatment group estimated a one-year survival rate of 27% and rescue chemotherapy group 20%. Based on the study's data, First Three Pharmaceuticals has plans to file quizartinib regulatory filings with the U.S. and European Union later this year.
in the Open Label Single Group II study, the data on the complete remission rate of quizartinib therapy reached the main endpoint of the study during the medium-term analysis, and the study was terminated early because of the remarkable efficacy. The safety observed in this study is consistent with that observed in the Phase III QuantUM-R study.
AML is an invasive blood and bone marrow cancer that causes abnormally functioning cancerous white blood cells to multiply and accumulate uncontrolled and affect the production of normal blood cells. FLT3 gene mutations are one of the most common genetic abnormalities in AML, where FLT3-ITD is the most common FLT3 mutation, affecting about one-t4 of AML patients. FLT3-ITD is a driving mutation with high leukemia burden, poor prognosticity, and significant impact on disease management in AML patients. The overall prognosis of FLT3-ITD AML patients was worse than in AML patients who did not carry FLT3-ITD mutations, including an increased recurrence rate, an increased risk of death after recurrence, and a higher likelihood of recurrence after hematopoietic stem cell transplantation.
quizartinib is a leading candidate drug in the AML franchise of the first three cancer businesses, is an oral small molecule-specific tyrosine kinase inhibitor, selectively targeting FLT3. Currently, the drug is being treated in the United States and the European Union with relapse/resoicative FLT3-ITD AML in Phase III clinical development (QuANTUM-R);
the United States, quizartinib has been awarded the FDA's breakthrough drug qualification for adult patients treating relapse/refragsive FLT3-ITD AML, and fast-track status for treating recurring/refragsive AML. In addition, quizartinib has been granted the treatment of AML orphan drugs in the United States and the European Union, and in Japan has been granted the treatment of FLT3 mutation AML orphan drug qualification.
It's worth noting that at the end of April 2017, Novartis' targeted cancer drug Rydapt (midostaurin) was also approved by the U.S. FDA for the first-line chemotherapy treatment of FLT3 mutation-positive AML patients, specifically: standard agaminosine and erythromycin induction and adcomycin consolidation chemotherapy, used in an FDA-approved companion diagnostic kit tested positive for FLT3 mutation adult AML patients.
Rydapt is an oral multi-kinase inhibitor that inhibits a variety of kinases that help regulate many important cellular processes, including FLT3 and KIT, disrupting the ability of cancer cells to grow and multiply. The approval makes Rydapt the world's first targeted drug for FLT3 mutation AML and the first new drug approved for AML treatment in the U.S. market in the past 25 years. However, Rydapt is not suitable as a single-drug induction therapy for AML patients. (Bio Valley)