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FDA grants Breakthrough Therapy status to PKC412 (midostaurin)

19 February 2016  •  Author: Victoria White

The US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to Novartis’ PKC412 (midostaurin).


PKC412 (midostaurin) is an investigational treatment for adults with newly-diagnosed acute myeloid leukaemia (AML) who are FLT3 mutation-positive, as detected by an FDA-approved test, and who are eligible to receive standard induction and consolidation chemotherapy.

The Breakthrough Therapy designation for PKC412 is primarily based upon the positive results from the Phase III RATIFY (CALGB 10603) clinical trial. Patients who received PKC412 and standard induction and consolidation chemotherapy experienced a significant improvement in overall survival (OS) compared to those who received standard induction and consolidation chemotherapy alone. The median OS for patients in the PKC412 treatment group was 74.7 months, versus 25.6 months for patients in the placebo group. No statistically significant differences were observed in the overall rate of grade 3 or higher haematologic and non-haematologic adverse events in the PKC412 treatment group versus the placebo group. A total of 37 deaths were reported, with no difference in treatment-related deaths observed between groups.

PKC412 is the first potential AML targeted therapy

Commenting on the announcement, Alessandro Riva, MD, Global Head, Novartis Oncology Development and Medical Affairs, 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.” 

Since the therapy is investigational at this time and is expected to be submitted for FDA approval, Novartis opened a Global Individual Patient Programme and a US Expanded Treatment Protocol (ETP) to enable PKC412 access. Patients 18 years of age and older with newly-diagnosed FLT3-mutated AML and able to receive standard induction and consolidation therapy will be considered.

In order to help identify patients who may have a FLT3 mutation and potentially benefit from treatment with the therapy, Novartis is collaborating with Invivoscribe Technologies, who is leading regulatory submissions for a companion diagnostic.

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