FDA News

FDA grants FLT3 inhibitor breakthrough therapy designation for acute myeloid leukemia

The FDA granted breakthrough therapy designation to quizartinib for the treatment of patients with relapsed/refractory acute myeloid leukemia with a FLT3 internal tandem duplication mutation.

Quizartinib (Daiichi Sankyo) is an oral selective FLT3 inhibitor.

The FDA based the designation on results from the phase 3 QuANTUM-R study. The analysis included 335 patients with relapsed/refractory FLT3-internal tandem duplication [ITD] AML who received single-agent quizartinib (n = 241) or salvage chemotherapy (n = 94).

Results showed quizartinib prolonged OS compared with chemotherapy.

“There have been limited advances over the past several decades for the treatment of relapsed/refractory FLT3-ITD AML, a very aggressive form of the disease associated with poor prognosis. Quizartinib is the first FLT3 inhibitor to significantly improve overall survival as an oral, single agent compared to chemotherapy in patients with relapsed/refractory AML with FLT3 ITD, an underlying driver of this subtype of AML,” Arnaud Lesegretain, vice president of oncology research and development and head of the AML franchise at Daiichi Sankyo, said in a press release. “We are excited that quizartinib has received breakthrough therapy designation and we look forward to working closely with the FDA to bring this potential new treatment option to patients as quickly as possible.”

Researchers observed a similar safety profile among patients who received quizartinib compared with those who received salvage chemotherapy. The most common adverse events among patients treated with quizartinib included nausea, thrombocytopenia, fatigue, musculoskeletal pain, pyrexia, anemia, neutropenia, febrile neutropenia, vomiting and hypokalemia.

The FDA granted breakthrough therapy designation to quizartinib for the treatment of patients with relapsed/refractory acute myeloid leukemia with a FLT3 internal tandem duplication mutation.

Quizartinib (Daiichi Sankyo) is an oral selective FLT3 inhibitor.

The FDA based the designation on results from the phase 3 QuANTUM-R study. The analysis included 335 patients with relapsed/refractory FLT3-internal tandem duplication [ITD] AML who received single-agent quizartinib (n = 241) or salvage chemotherapy (n = 94).

Results showed quizartinib prolonged OS compared with chemotherapy.

“There have been limited advances over the past several decades for the treatment of relapsed/refractory FLT3-ITD AML, a very aggressive form of the disease associated with poor prognosis. Quizartinib is the first FLT3 inhibitor to significantly improve overall survival as an oral, single agent compared to chemotherapy in patients with relapsed/refractory AML with FLT3 ITD, an underlying driver of this subtype of AML,” Arnaud Lesegretain, vice president of oncology research and development and head of the AML franchise at Daiichi Sankyo, said in a press release. “We are excited that quizartinib has received breakthrough therapy designation and we look forward to working closely with the FDA to bring this potential new treatment option to patients as quickly as possible.”

Researchers observed a similar safety profile among patients who received quizartinib compared with those who received salvage chemotherapy. The most common adverse events among patients treated with quizartinib included nausea, thrombocytopenia, fatigue, musculoskeletal pain, pyrexia, anemia, neutropenia, febrile neutropenia, vomiting and hypokalemia.