Rare Disease Report

FDA Fast Tracks Astellas' AML Treatment

OCTOBER 11, 2017
Mathew Shanley
Astellas Oncology released data at the 2017 American Society of Clinical Oncology (ASCO) annual meeting that revealed the improvement of overall survival of patients with FLT3 mutation-positive (FLT3mut+) relapsed/refractory (R/R) acute myeloid leukemia (AML) when given gilteritinib, and a month later, the drug was granted orphan drug designation by the U.S. Food and Drug Administration (FDA).

This morning, the company announced that it was granted Fast Track designation for the development of the drug in adult patients.

Gilteritinib is a FLT3/AXL tyrosine kinase inhibitor. Data has shown correlation between the administration of the investigational compound and antileukemic activity in patients, and gilteritinib has exhibited inhibitory action against FLT3 internal tandem duplication (ITD), as well as FLT3 tyrosine kinase domain (TKD) – two of the most common types of FLT3 mutations.

In AML, the bone marrow generates abnormal myeloblasts, or unipotent stem cells, affecting both a patient’s bone marrow and blood. The condition typically presents via fatigue, recurrent infections and easy bruising.

Per the American Cancer Society, there were an estimated 21,000 new cases of AML diagnosed in the United States in 2016, and nearly half resulted in death.

“Mutations of FLT3 in AML are associated with a poor prognosis and we are committed to working with the FDA to meet the requirements of the expedited review process,” said Steven Benner, M.D., senior vice president and global therapeutic area head, oncology development, Astellas. “We are pleased that the FDA has acknowledged the urgent need for new therapies for FLT3+ AML patients, which may allow for an expedited review process for gilteritinib.”

Ongoing Phase 3 trials are evaluating the safety and efficacy of the drug in a variety of AML patient populations. They include:
  • A Trial of the FMS-like Tyrosine Kinase 3 (FLT3) Inhibitor Gilteritinib Administered as Maintenance Therapy Following Allogeneic Transplant for Patients With FLT3/Internal Tandem Duplication (ITD) Acute Myeloid Leukemia (AML) (NCT02997202)
  • A Study of ASP2215 (Gilteritinib), Administered as Maintenance Therapy Following Induction/Consolidation Therapy for Subjects With FMS-like Tyrosine Kinase 3 (FLT3/ITD) Acute Myeloid Leukemia (AML) in First Complete Remission (NCT02927262)
  • A Study of ASP2215 (Gilteritinib), Combination of ASP2215 Plus Azacitidine and Azacitidine Alone in the Treatment of Newly Diagnosed Acute Myeloid Leukemia With FMS-like Tyrosine Kinase (FLT3) Mutation in Patients Not Eligible for Intensive Induction Chemotherapy (NCT02752035)
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