FDA approves new targeted drug for leukemia

The U.S. 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.

microscopic image of acute myeloid leukemia cells

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.

Read more at Penn Medicine News.