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Midostaurin has received Breakthrough Therapy designation from the FDA for newly-diagnosed FLT3-mutated acute myeloid leukemia

The FDA ( Food and Drug Administration ) has granted Breakthrough Therapy designation to PKC412 ( Midostaurin ).
Midostaurin is an investigational treatment for adults with newly-diagnosed acute myeloid leukemia ( 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 Midostaurin is primarily based upon the positive results from the phase III RATIFY clinical trial.
This study was presented during a plenary session at the 57th American Society of Hematology ( ASH ) Annual Meeting.

Patients who received Midostaurin and standard induction and consolidation chemotherapy experienced a significant improvement in overall survival ( hazard ratio, HR=0.77, P = 0.0074 ) compared to those who received standard induction and consolidation chemotherapy alone.

The median overall survival for patients in the Midostaurin 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 Midostaurin treatment group versus the placebo group.

A total of 37 deaths were reported, with no difference in treatment-related deaths observed between groups.

According to the FDA, Breakthrough Therapy designation is intended to expedite the development and review of new medicines that treat serious or life-threatening conditions, if the therapy has demonstrated substantial improvement over an available therapy on at least one clinically significant endpoint.
The designation includes all of the Fast Track program features, as well as more intensive FDA guidance on an efficient drug development program.

In the US, about 20,000 people were diagnosed with acute myeloid leukemia in 2015, the majority of whom were adults.
According to the latest research, approximately one-third of AML patients also harbor a FLT3 gene mutation, which is associated with worse outcomes and shorter survival than in those without the mutation.
FMS-like tyrosine kinase-3 ( 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.
Midostaurin is the first drug targeting FLT3 to demonstrate an overall survival benefit in acute myeloid leukemia.

Midostaurin is also being investigated for the treatment of aggressive systemic mastocytosis / mast cell leukemia. ( Xagena )

Source: Novartis, 2016