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The US Food and Drug Administration (FDA) has granted breakthrough therapy designation for Novartis’ investigational drug, PKC412 (midostaurin), to treat FLT3-mutated acute myeloid leukaemia (AML) in adults.

Novartis said that PKC412 is an investigational, multi-targeted kinase inhibitor being developed for the treatment of patients with AML with a FLT3 mutation, which is associated with worse outcomes and shorter survival than in those without the mutation.

The breakthrough status was based on the results of Phase III RATIFY (CALGB 10603) clinical trial, which was conducted in partnership with the Alliance for Clinical Trials in Oncology.

PKC412 has significantly improved the overall survival of adult patients eligible to receive standard induction and consolidation chemotherapy.

AML is said to have the lowest survival rate of all adult leukaemia and the treatment strategy with chemotherapy has remained unchanged for more than 25 years.

"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."

Novartis oncology development and medical affairs global head 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.

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"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."

More than 20,000 people were diagnosed with AML in the US last year and the majority of them were adults.

PKC412 is also being investigated for the treatment of aggressive systemic mastocytosis / mast cell leukaemia.


Image: Bone marrow aspirate showing acute myeloid leukaemia. Photo: courtesy of VashiDonsk / Wikipedia.