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FDA grants breakthrough therapy designation to midostaurin for newly diagnosed AML
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The FDA today granted breakthrough therapy designation to midostaurin to treat patients newly diagnosed with FLT3-positive acute myeloid leukemia.
The designation is intended for patients who are eligible to receive standard induction and consolidation chemotherapy.
The FDA based its decision in part on positive results from the phase 3 RATIFY clinical trial (CALGB 10603), which evaluated the safety and efficacy of midostaurin (PKC412, Novartis), an oral, multi-targeted kinase inhibitor.
The addition of midostaurin to standard induction and consolidation chemotherapy significantly improved OS (HR = 0.77; P = 0.0074). Patients who received midostaurin achieved a median OS of 74.7 months (95% CI, 31.7-not attained), whereas patients who received standard induction and consolidation chemotherapy plus placebo achieved a median OS of 25.6 months (95% CI, 18.6-42.9).
Researchers observed no statistically significant difference in the overall rate of grade 3 or higher hematologic and nonhematologic adverse events in the midostaurin treatment group compared with the placebo group. In total, 37 deaths were reported, with no difference in treatment-related deaths between groups.
“For more than 25 years, medical developments have been limited for AML patients and the chemotherapy treatment strategy has essentially remained unchanged,” Alessandro Riva, MD, global head of Novartis oncology development and medical affairs, said in a press release. “We look forward to working closely with the FDA to bring PKC412, the first potential AML targeted therapy, to patients as quickly as possible.”
Midostaurin also is being investigated for the treatment of aggressive systemic mastocytosis/mast cell leukemia.