Regulatory

As NDA talks loom, FDA hands Daiichi Sankyo a ‘breakthrough’ on its $410M AML drug quizartinib

Daiichi Sankyo execs just gained an added edge for their upcoming discussions with the FDA on an application to start selling quizartinib, a new drug for treatment-resistant acute myeloid leukemia with FLT3-ITD mutations.

Arnaud Lesegretain

The FDA handed the Japanese company a breakthrough drug designation for quizartinib, putting the company on its inside track and likely heralding a rapid-fire approval.

Daiichi bought this drug in a $410 million deal — $315 million in cash — to acquire Ambit Biosciences in San Diego 4 years ago. And just weeks ago the company outlined positive, though far from impressive, survival data from its pivotal study.

AML patients in the drug arm had a 27% survival rate at 52 weeks, compared to 20% in the control arm. That’s statistically significant and good enough for an OK — barring a nasty surprise — but nothing that will wow physicians in the field.

Jorge Cortes

“For 50 years, since the standard of therapy was introduced, nothing has changed since then,” lead investigator Jorge Cortes told me 6 weeks ago. “You could say a few weeks isn’t much,” but he sees this as a landmark success that will change that long-running standard of care.

Daiichi is studying their drug in combination with chemo in ongoing studies. Meanwhile, Novartis’ Rydapt (midostaurin) was approved a year ago in combination with chemo for frontline cases of AML with FLT3 mutations (including ITD) while Nexavar recently demonstrated efficacy with chemo in the same specific patient population.

“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,” said Arnaud Lesegretain, head of the AML franchise at Daiichi Sankyo. “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.”


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