China Approves Vanflyta for Newly Diagnosed FLT3-ITD Positive AML

China’s National Medical Products Administration (NMPA) has approved Daiichi Sankyo’s Vanflyta® (quizartinib) in combination with standard chemotherapy and subsequent maintenance monotherapy for adults with newly diagnosed FLT3-internal tandem duplication (FLT3-ITD) positive acute myeloid leukemia (AML).

With this decision, Vanflyta becomes the first and only FLT3 inhibitor approved in China for newly diagnosed FLT3-ITD positive AML. AML accounts for nearly half of the approximately 82,000 leukemia cases diagnosed annually in China. FLT3 mutations occur in up to 37% of patients, with approximately 80% being FLT3-ITD, a subtype associated with rapid disease progression, increased relapse risk, and poor outcomes. Historically, the five-year survival rate for patients with FLT3-ITD positive AML has been estimated at around 20%.

QuANTUM-First Trial Results

Approval was supported by the global Phase 3 QuANTUM-First trial (NCT02668653), published in The Lancet. The randomized, double-blind study evaluated quizartinib in combination with standard induction and consolidation chemotherapy, followed by maintenance monotherapy for up to three years.

The trial met its primary endpoint, reducing the risk of death by 22% compared with chemotherapy alone (HR=0.78; 95% CI: 0.62–0.98; p=0.032). Median overall survival was 31.9 months with quizartinib versus 15.1 months in the control arm at a median follow-up of 39.2 months.

Notably, the study incorporated maintenance therapy for up to three years following consolidation chemotherapy, supporting sustained FLT3 inhibition beyond initial treatment. Patients were also permitted to undergo allogeneic hematopoietic stem cell transplantation (HSCT), reflecting current AML treatment practice.

Clinical Implications

Professor Wang Jianxiang of the Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences, noted that Vanflyta significantly extended survival in a patient population that has historically faced poor outcomes despite intensive chemotherapy.

Michio Hayashi, China President of Daiichi Sankyo, said the approval underscores the company’s commitment to delivering innovative oncology medicines to patients in China and represents an important milestone for individuals with FLT3-ITD positive AML.

Safety Profile

Among 268 patients treated with quizartinib, the most common Grade 3 or 4 adverse events were thrombocytopenia (40%), anemia (35.5%), neutropenia (21.5%), and increased alanine aminotransferase (ALT) levels (12.1%).

QT prolongation greater than 500 milliseconds occurred in 2.3% of patients, while 0.8% discontinued treatment because of QT prolongation. Two cardiac arrest events associated with ventricular fibrillation occurred in the setting of severe hypokalemia, including one fatal case.

Overall, the safety profile was consistent with previous clinical experience, and no new safety signals were identified.

About Vanflyta

Vanflyta is an oral, highly potent type II FLT3 inhibitor designed to selectively target FLT3-ITD mutations. The medicine is approved in more than 35 countries and regions, including the United States, European Union, Japan, and China. In Japan, Vanflyta is also approved as monotherapy for patients with relapsed or refractory FLT3-ITD positive AML based on findings from the QuANTUM-R study.

Ongoing Development Program

Daiichi Sankyo continues to advance quizartinib through multiple clinical studies, including the Phase 3 QuANTUM-Wild trial in newly diagnosed FLT3-wild-type AML, a Phase 1/2 pediatric study in relapsed or refractory FLT3-ITD positive AML, and several combination trials being conducted in collaboration with The University of Texas MD Anderson Cancer Center.

Reference

“Vanflyta® Approved in China as First and Only FLT3 Inhibitor for Patients with Newly Diagnosed FLT3-ITD Positive AML”