Rare Disease Report

New AML Therapy Receives Breakthrough Designation

FEBRUARY 18, 2016
Rebekah Harrison
The FDA granted breakthrough therapy designation to Novaris’ PKC412 (midostaurin), an investigational treatment for adults with newly-diagnosed acute myeloid leukemia (AML) who are FMS-like tyrosine kinase-3 (FLT3) mutation-positive and who are eligible to receive standard induction and consolidation chemotherapy.  

What is Acute Myeloid Leukemia?

Acute myeloid leukemia is a type of cancer in which the bone marrow makes abnormal myeloblasts. Symptoms include fatigue, recurrent infections and easy bruising. Acute myeloid leukemia is generally a disease of older people and is uncommon before the age of 45. The average age of a patient with AML is about 67 years. The number of new cases of acute myeloid leukemia was 4.0 per 100,000 men and women per year.
Mutations in specific genes are found in many cases of AML, and biomarker testing is considered standard of care for newly-diagnosed patients to help determine the best possible treatment option. FLT3 is a receptor tyrosine kinase, a type of cell-surface receptor, which plays a role in the proliferation, or increase, in the number of certain blood cells
AML is typically treated with chemotherapy and the intensity of chemotherapy depends on a person's age and health.
The breakthrough therapy designation for PKC412 is primarily based upon the positive results from the phase 3 RATIFY (CALGB 10603) clinical trial.

The Study

Patients who received PKC412 and standard induction and consolidation chemotherapy experienced a significant improvement in overall survival (OS) (hazard ratio = 0.77, p = 0.0074) 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 (95% confidence interval [CI]: 31.7, not attained), versus 25.6 months (95% CI: 18.6, 42.9) for patients in the placebo group. No statistically significant differences were observed in the overall rate of grade 3 or higher hematologic and non-hematologic 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.
"For more than 25 years, medical developments have been limited for AML patients and the chemotherapy treatment strategy has essentially remained unchanged," said Alessandro Riva MD, global head of Novartis Oncology Development and Medical Affairs. "We look forward to working closely with the FDA to bring PKC412, the first potential AML targeted therapy, to patients as quickly as possible."


Novartis drug PKC412 (midostaurin) receives Breakthrough Therapy designation from the FDA for newly-diagnosed FLT3-mutated acute myeloid leukemia (AML) [news release]. Basil, Switzerland; Novartis: February 19, 2016. https://www.novartis.com/news/media-releases/novartis-drug-pkc412-midostaurin-receives-breakthrough-therapy-designation-fda

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