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news.The US Food and Drug Administration (FDA) has granted breakthrough therapy designation to Novartis' PKC412 (midostaurin) as a potential treatment for adult patients with newly diagnosed FLT3-mutated acute myeloid leukemia (AML).
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In the phase III Ratify trial, patients who received PKC412 and chemotherapy experienced an increase in median overall survival compared to those receiving chemo alone (74.7 months versus 25.6 months) with no significant difference in grade 3 (severe) or higher adverse events.
The safety and efficacy profile of PKC412 has not been fully established. Novartis said there is no guarantee that oral, multi-targeted kinase inhibitor will become commercially available.
The breakthrough therapy designation is given to expedite the development and review of new medicines that treat severe or life-threatening conditions, if the therapy showed substantial improvement on an available therapy over at least one clinically significant endpoint.
The designation includes the entire fast track program features and more intensive FDA guidance on an efficient drug development program.
Novartis global head of oncology development and medical affairs Alessandro Riva said: "For more than 25 years, medical developments have been limited for AML patients and the chemotherapy treatment strategy has essentially remained unchanged.
"We look forward to working closely with the FDA to bring PKC412 (midostaurin), the first potential AML targeted therapy, to patients as quickly as possible."
In the US, about 20,000 new cases of AML are diagnosed each year, the majority in adults. About one third are positive for the FLT3 mutation.