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The US Food and Drug Administration (FDA) has approved Novartis Rydapt (midostaurin, formerly PKC412) for two indications.

Available in 25mg capsules, Rydaptis an oral, multi-targeted inhibitor of several kinases that help in regulating multiple essential cell processes, preventing cancer cells' ability to grow and multiply.

The drug can be used to treat acute myeloid leukaemia (AML) in newly diagnosed patients who are FMS-like tyrosine kinase 3 mutation-positive (FLT3+) along with chemotherapy.

The FDA has also approved Rydapt for the treatment of adults affected with advanced systemic mastocytosis (SM) that includes aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated haematological neoplasm (SM-AHN) and mast cell leukaemia.

Novartis oncology CEO Bruno Strigini said: “Rydapt represents a remarkable advance as the first and only targeted therapy approved for patients who had limited options for many years.

“We are proud to continue our leadership in haematology as we work diligently to bring innovative medicines to patients worldwide.”

The FDA approval follows a prior breakthrough therapy designation in FLT3-mutated AML, orphan drug designation and priority review for both indications.

The FDA has approved Rydapt in newly diagnosed FLT3-mutated AML, a rare and aggressive cancer of blood and bone marrow, on the basis of Phase III RATIFY (CALGB 10603 / Alliance) clinical trial.

In the US alone, nearly 21,000 people are estimated to be diagnosed with AML this year, 7,000 of which will have an FLT3 gene mutation.

"Rydapt represents a remarkable advance as the first and only targeted therapy approved for patients who had limited options for many years."

FLT3 is a type of cell-surface receptor that helps in specific blood cells production.

The FLT3 gene mutation accelerates disease progression and increases the chances of relapse rates than other forms of AML.

On the other hand, Rydapt received approval to treat three types of SM on the basis of two single-arm, open-label multicentre trials, including the Phase II study (CPKC412D2201).

Advanced SM is a rare blood disorder that exhibits uncontrolled growth and accumulation of mast cells in one or multiple organs, thereby damaging them.


Image: Novartis headquarters in Basel, Switzerland. Photo: courtesy of Novartis.