The European Commission has approved Novartis’s Rydapt for two indications in rare cancers, in combination with standard daunorubicin and cytarabine induction and high-dose cytarabine consolidation chemotherapy. The drug has also been approved for adults with newly diagnosed acute myeloid leukemia (AML) who are FLT3 mutation-positive.
In addition, Novartis said Wednesday that it was also cleared for use as monotherapy for the treatment of adults with aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematological neoplasm (SM-AHN) or mast cell leukemia.
Rydapt represents the first and only targeted therapy for FMS-like tyrosine kinase 3 (FLT3)-mutated AML and the only treatment for three subtypes of SM, collectively known as advanced SM, in the EU, all of which have limited life expectancy and few treatment options. Rydapt represents the first major advancement for the treatment of patients with newly diagnosed FLT3-mutated AML in more than 25 years.
Bruno Strigini, CEO, Novartis Oncology, said that 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.
In order to further investigate the potential of Rydapt in AML, Novartis is planning a Phase III study in newly diagnosed AML patients without a FLT3 mutation (wildtype).
About AML
AML is the most common acute leukemia, or blood cancer, in adults; it accounts for approximately 25% of all adult leukemias worldwide, with the highest incidence rates occurring in the US, Europe and Australia. It also has the lowest survival rate of all adult leukemias.
In AML, white blood cells are not able to mature and instead build up an accumulation of “blasts,” blocking room for normal blood cells. Mutations in specific genes, such as FLT3, are found in many cases of the disease. Genetic testing for mutations in newly diagnosed AML patients can help to determine prognosis and potential treatment strategies.
In the EU, there are more than 18,000 estimated new cases of AML diagnosed each year[10]. Approximately one-third of AML patients have a FLT3 gene mutation. FLT3 is a type of cell-surface receptor, which plays a role in increasing the number of certain blood cells. The FLT3 gene mutation can result in faster disease progression, higher relapse rates and lower rates of survival than other forms of AML
Image: A sign marks a building on Novartis’ campus in Cambridge, Massachusetts, U.S., February 28, 2017. REUTERS/Brian Snyder