Leukemia drug Vanflyta enters market

Demonstrates improvement in key efficacy endpoints … overall survival doubled vs. placebo, risk of death reduced by 22%

Expanding treatment options across the entire disease course, from induction and consolidation combination therapy to maintenance therapy

With the domestic approval of ‘Vanflyta,’ a targeted therapy for FLT3-ITD mutation-positive acute myeloid leukemia (AML), the possibility of a shift in treatment strategies is being raised.

Not only has a treatment option covering the full course from combination use in induction and consolidation therapy to maintenance therapy been added, but the drug is also expected to emerge as a new alternative for patients at high risk of relapse after demonstrating clinical benefits such as improved overall survival (OS).

On the 14th, Daiichi Sankyo Korea held a press conference at the Plaza Hotel in Jung-gu, Seoul, to commemorate the domestic approval of Vanflyta (quizartinib). Vanflyta was approved in Korea in January for the treatment of acute myeloid leukemia (AML).

Professor Byung Sik Cho, Department of Hematology, Seoul St. Mary’s Hospital

The specific indication includes its use for newly diagnosed adult AML patients who are positive for the FLT3-ITD mutation, in combination with standard cytarabine- and anthracycline-based induction therapy and cytarabine consolidation therapy, and as monotherapy maintenance treatment thereafter.

Its defining feature is its applicability to a full-cycle treatment strategy spanning induction, consolidation, and maintenance.

With this approval, a new FLT3-targeted therapy has been added to the AML treatment landscape, alongside Novartis’ ‘Rydapt (midostaurin)’ and Astellas’ ‘Xospata (gilteritinib).’

Experts agree that the arrival of Vanflyta is particularly significant as it offers a treatment option specifically targeting FLT3-ITD mutation-positive patients, who remain at high risk of relapse despite existing treatment.

FLT3 mutations are detected in approximately 37% of newly diagnosed AML patients, with about 80% of these cases involving the FLT3-ITD mutation. This mutation is known to promote cancer cell proliferation and increase the risk of relapse, and the 5-year survival rate for these patients is only about 20%.

FLT3 is a key receptor that regulates the survival, proliferation, and differentiation of hematopoietic stem cells; however, when a mutation occurs, abnormal signaling is activated, promoting the growth of leukemia cells.

Professor Byung Sik Cho of the Department of Hematology at Seoul St. Mary’s Hospital said, “Treatment outcomes have improved since the introduction of FLT3-targeted agents, but even when existing FLT3 inhibitors are combined with chemotherapy, relapse rates of around 40% are still reported. There has been a significant unmet need for new treatment options that can improve outcomes, especially in FLT3-ITD-positive patients.”

Vanflyta demonstrated efficacy in the Phase III QuANTUM-First study in patients with FLT3-ITD mutation-positive AML. In the study, patients were randomized 1:1 to the Vanflyta group or the placebo group, received combination treatment with induction and consolidation therapy, and then underwent maintenance therapy for up to 3 years.

Dong-Yeop Shin, Division of Hematology and Medical Oncology, Seoul National University Hospital

The results showed that the Vanflyta group’s risk of death was reduced by 22% compared to the placebo group. At a median follow-up of 39.2 months, the median overall survival (OS) was 31.9 months in the Vanflyta group, more than double the 15.1 months observed in the placebo group.

Additionally, the duration of complete remission (CR) was 38.6 months in the Vanflyta group, approximately 3 times longer than the 12.4 months in the placebo group, demonstrating meaningful improvement in disease control as well.

In terms of safety, febrile neutropenia, hypokalemia, and pneumonia were reported as major adverse events, and the overall pattern of adverse events was similar to that of the placebo group.

Professor Dong-Yeop Shin of the Division of Hematology and Oncology at Seoul National University Hospital said, “Vanflyta demonstrated consistent benefits not only in improving overall survival but also in prolonging the duration of complete remission and reducing the cumulative relapse rate. It has the potential to change the treatment paradigm for FLT3-ITD mutation-positive AML.”