- Accelerated approval follows priority review designation and inclusion in overseas new drugs urgently needed in clinical settings -

-­ XOSPATA® is the first and only FLT3 inhibitor approved by the NMPA 
for patients with relapsed or refractory AML -

TOKYO, February 4, 2021 – Astellas Pharma Inc. (TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D., “Astellas”) today announced that the China National Medical Products Administration (NMPA) has granted conditional approval to XOSPATA® (gilteritinib) for the treatment of adult patients who have relapsed (disease that has returned) or refractory (resistant to treatment) acute myeloid leukemia (AML) with a FLT3 mutation (FLT3mut+) detected by a fully validated test. Gilteritinib has been approved under an expedited pathway, following NMPA’s acceptance of gilteritinib for priority review in July 20201 and its inclusion in the third batch of overseas new drugs urgently needed in clinical settings in November 2020.2

“Patients with relapsed or refractory AML with a FLT3 mutation are in urgent need of new treatment options,” said Professor Ma Ju, Director of the Harbin Institute of Hematology, China. “As the first approved targeted therapy agent to treat relapsed or refractory AML with a FLT3 mutation in China, gilteritinib, which was approved under an expedited pathway, has enabled patients in China to have rapid access to a novel treatment option.” 

Gilteritinib has shown itself to be effective against two types of FLT3 mutation – FLT3 internal tandem duplication (FLT3-ITD) and FLT3 tyrosine kinase domain (FLT3-TKD). Impacting approximately 30% of AML patients,3 the FLT3-ITD mutation is associated with higher risk of relapse and shorter overall survival compared to wild-type FLT3.4,5 FLT3-TKD mutations impact approximately 7% of AML patients.3 The status of FLT3 mutation can change over the course of AML treatment, including after relapse. Confirming patients’ FLT3 mutation status at the time of relapse can help inform an appropriate and potentially targeted treatment approach.6

“Having a FLT3 mutation has a highly negative impact on prognosis for people living with AML,” said Professor Wang Jianxiang, Vice Director of Institute of Hematology, Chinese Academy of Medical Sciences. “The approval of gilteritinib provides an important new option for Chinese patients that have relapsed or refractory AML with a FLT3 mutation, backed by substantial safety and efficacy data.”

AML is a cancer that impacts the blood and bone marrow,7 and its incidence increases with age.8 It is one of the most common types of leukemia in adults.9 Every year, it is estimated that around 80,000 people in China are diagnosed with leukemia.10 

“There is an urgent unmet need among FLT3-mutated relapsed or refractory AML patients, whose median survival is currently less than six months with chemotherapy,” said Andrew Krivoshik, M.D., Ph.D., Senior Vice President and Global Therapeutic Area Head, Oncology Development. “The expedited approval of gilteritinib is an important step in offering a new treatment option for doctors and patients in China. We look forward to offering gilteritinib as part of our commitment to developing innovative solutions for patients with hard-to-treat cancers with limited treatment options.”

The approval was based on results from the Phase 3 ADMIRAL trial, published in the New England Journal of Medicine. Patients treated with gilteritinib had significantly longer overall survival (OS) than those who received salvage chemotherapy. Median OS for patients who received gilteritinib was 9.3 months, compared to 5.6 months for patients who received salvage chemotherapy (Hazard Ratio = 0.64 (95% CI 0.49, 0.83), P=0.0004).11 Additional Chinese patient pharmacokinetics data from the ongoing Phase 3 COMMODORE trial were also reviewed.

The safety of gilteritinib was evaluated in 319 patients with relapsed or refractory AML who have received at least one dose of 120 mg gilteritinib daily.11 The most frequent all-grade adverse reactions (frequency ≥ 10%) with gilteritinib were alanine aminotransferase (ALT) increased (25.4%), aspartate aminotransferase (AST) increased (24.5%), anemia (20.1%), thrombocytopenia (13.5%), febrile neutropenia (12.5%), platelet count decreased (12.2%), diarrhea (12.2%), nausea (11.3%), blood alkaline phosphatase increased (11%), fatigue (10.3%), white blood cell count decreased (10%), and blood creatine phosphokinase increased (10%). One fatal adverse reaction of differentiation syndrome occurred in patients receiving gilteritinib. The most frequent (frequency ≥3%) serious adverse reactions were febrile neutropenia (7.5%), ALT increased (3.4%), and AST increased (3.1%). Other clinically significant serious adverse reactions included electrocardiogram QT prolonged (0.9%) and posterior reversible encephalopathy syndrome (0.3%). 

Astellas has already reflected the impact from this approval in its financial forecast of the current fiscal year ending March 31, 2021.


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