PKC412 (midostaurin), an investigational, oral, multi-targeted kinase inhibitor manufactured by pharmaceutical company Novartis, has been granted Breakthrough Designation by the U.S. Food and Drug Administration (FDA).
The therapy is indicated for treatment of patients living with acute myelogenous leukemia (AML).
AML is an aggressive cancer of the blood and bone marrow marked by decreased maturation of white blood cells, creating an accumulation of "blasts" that do not allow room for normal blood cells.
Although AML is the most common acute leukemia diagnosed in adults, it has the lowest survival rate.
Breakthrough Designation is given by the FDA for medications that have, through tests and trials, proven to be effective in treating life-threatening conditions.
PKC412 is indicated for patients whose AML is newly diagnosed and who have been proven by an FDA-approved test to be FLT3 mutation-positive. Additionally, candidates must have been approved to receive standard induction and consolidation chemotherapy.
"For more than 25 years, medical developments have been limited for AML patients, and the chemotherapy treatment strategy has essentially remained unchanged," Alessandro Riva, global head of Novartis' Oncology Development and Medical Affairs, said. "We look forward to working closely with the FDA to bring PKC412 -- the first potential AML-targeted therapy -- to patients as quickly as possible."
