The US Food and Drug Administration has approved the first ever inhibitor drug specifically approved for treating patients with relapsed or refractory acute myeloid leukemia (AML) with a mutation in the Fms-like tyrosine kinase 3 (FLT3) gene. Patients with these mutations who have relapsed or refractory AML have very low response rates to chemotherapy at the time of relapse, and their survival is poor as a result.

The approval was granted for the drug gilteritinib and was based on clinical trials led by Alexander Perl, an associate professor of hematology-oncology in the Perelman School of Medicine and the Abramson Cancer Center. Gilteritinib is sold under the name XOSPATA® and manufactured by Astellas Pharma, Inc.

“Today’s approval brings a new, highly-effective, and well-tolerated treatment option to the clinic for a group of truly high-risk patients who, until today, had no specific therapies available beyond chemotherapy to treat their disease,” Perl says.

Gilteritinib is a pill patients take once a day, and studies have shown it can inhibit activity in AML patients with the two most common types of FLT3 mutations.