FDA to Prioritize Review of Oral Drug to Treat Common Type of Leukemia

The Food and Drug Administration (FDA) granted quizartinib, an oral drug, a priority review for the treatment of adults with newly diagnosed acute myeloid leukemia that is FLT3-ITD positive, according to a press release from the drug’s manufacturer Daiichi Sankyo.

A priority review is given to drugs that, if approved, would be a significant improvement over existing treatment options.

In this instance, the priority review was granted to quizartinib plus the chemotherapies cytarabine and anthracycline as either induction or consolidation treatment. Induction chemotherapy is given to patients as the initial treatment to decrease signs or symptoms of cancer, according to the National Cancer Institute. Consolidation chemotherapy is administered after the cancer has disappeared following induction chemotherapy.

Moreover, the priority review is indicated to continue treatment with single-agent quizartinib following the completion of consolidation chemotherapy.

The FDA based its decision on data from the phase 3 QuANTUM-First trial, which demonstrated that this regimen led to a meaningful overall survival (time from start of treatment until death from any cause) improvement versus chemotherapy alone in patients with FLT3-ITD positive acute myeloid leukemia.

According to the press release, the five-year overall survival rate of acute myeloid leukemia is 30.5%. Moreover, of the estimated 20,050 new cases of acute myeloid leukemia, approximately 25% have the FLT3-ITD mutation. This mutation, according to the release, is associated with an unfavorable prognosis.

“There is a need for new targeted therapy options for patients with acute myeloid leukemia and the results of the QuANTUM-First trial showed that quizartinib in combination with standard chemotherapy has potential to change the current standard of care for newly diagnosed patients with the historically difficult-to-treat FLT3-ITD subtype,” Dr. Ken Takeshita, global head of research and development at Daiichi Sankyo, said in the release. “The FDA’s prioritization of this application reflects the importance of the data, and we will continue to work with the FDA and other global regulatory authorities to support the review of quizartinib for the treatment of patients with newly diagnosed FLT3-ITD positive acute myeloid leukemia.”

Approximately 539 patients across 193 cancer centers around the world were enrolled onto the QuANTUM-First trial with the aim of assessing overall survival outcomes. Additional goals included reviewing the event-free survival (time following treatment that people have not had cancer return or get worse) rates, as well as post-induction rates of complete remission and composite complete remission following treatment with study drug.

The FDA plans to make its decision on quizartinib by April 2023.

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