Regulatory Affairs

FDA approves Novartis’ Rydapt for AML and rare blood disorders

PBR Staff Writer Published 01 May 2017

Novartis’ Rydapt (midostaurin) has secured approval from the US Food and Drug Administration (FDA) in newly diagnosed FLT3-mutated acute myeloid leukemia (AML) and three types of systemic mastocytosis (SM).

Rydapt is indicated for use in combination with standard cytarabine and daunorubicin induction and cytarabine consolidation chemotherapy to treat adult patients with newly diagnosed AML who are FLT3 mutation-positive, as detected by an FDA-approved test.

It is not indicated as a single-agent induction therapy for the treatment of patients with AML.

The drug has to be used along with a companion diagnostic known as the LeukoStrat CDx FLT3 Mutation Assay which detects the FLT3 mutation in AML patients.

Rydapt has also been approved for certain rare blood disorders that include aggressive systemic mastocytosis, mast cell leukemia and systemic mastocytosis with associated hematological neoplasm.

FDA Oncology Center of Excellence director Richard Pazdur said: “Rydapt is the first targeted therapy to treat patients with AML, in combination with chemotherapy.

“The ability to detect the gene mutation with a diagnostic test means doctors can identify specific patients who may benefit from this treatment.”

The oral, multi-targeted inhibitor of multiple kinases like FLT3 and KIT, Rydapt enables control of several essential cell processes and breaks the ability of cancer cells to grow and multiply.

During a phase 3 study named RATIFY in FLT3-mutated AML patients, the Rydapt treatment regimen was shown to considerably improve overall survival with a 23% reduction in the risk of death.

Rydapt’s approval comes after the FDA granted a Breakthrough Therapy designation previously in AML with FLT3 mutations along with an Orphan Drug designation and Priority Review in both indications.

Novartis is currently filing for Rydapt in various countries for approvals for the treatment of AML, a rare and aggressive cancer of the blood and bone marrow.

The FDA approval means that Rydapt has become the first and only targeted therapy till date for newly diagnosed AML with FLT3 mutations.

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 hematology as we work diligently to bring innovative medicines to patients worldwide."

Image: Rydapt Capsules. Photo: courtesy of Novartis AG.