The European Commission has approved Novartis’ Rydapt to treat a certain form of acute myeloid leukaemia (AML) as well as three other rare diseases.
The decision allows doctors to prescribe the drug in combination with standard daunorubicin and cytarabine induction and high-dose cytarabine consolidation chemotherapy, and for patients in complete response followed by Rydapt (midostaurin) single agent maintenance therapy, for adults with newly diagnosed AML who are FLT3 mutation-positive.
The drug was also cleared for use as monotherapy for the treatment of adults with aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated haematological neoplasm (SM-AHN) or mast cell leukaemia.
Rydapt represents the first and only targeted therapy for FLT3-mutated AML and the only treatment for three subtypes of systemic mastocytosis, collectively known as advanced SM, in the EU, “all of which have limited life expectancy and few treatment options,” Novartis noted.
“For patients with FLT3-mutated AML, there have been no meaningful advancements in more than 25 years and with Rydapt they now have a targeted medicine that could significantly extend their lives,” said Bruno Strigini, chief executive of the Swiss drug giant’s Oncology division.
For newly diagnosed FLT3-mutated AML, the approval rides on data from the RATIFY trial, which showed a 23 percent reduction in the risk of death with Rydapt plus standard chemotherapy compared with placebo plus standard chemotherapy. Median overall survival was 74.7 months and 25.6 months, respectively.
For advanced SM, clearance is based on two single-arm open-label multicenter trials, in which patients demonstrated an overall response rate, defined as a major or partial response, of 59.6 percent.
Novartis is now planning a Phase III study in newly diagnosed AML patients without a FLT3 mutation (wildtype).