Novartis today announced that the European Commission (EC) approved Rydapt® (midostaurin) for two indications in rare, hard-to-treat cancers.
Rydapt is approved for use in combination with standard daunorubicin and cytarabine induction and high-dose cytarabine consolidation chemotherapy, and for patients in complete response followed by Rydapt single agent maintenance therapy, for adults with newly diagnosed acute myeloid leukemia (AML) who are FLT3 mutation-positive. 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.
The approval follows a positive opinion issued by the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) on July 20, 2017 and applies to all 28 EU member states, plus Iceland, Liechtenstein and Norway. 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.
“Novartis is proud that we can deliver Rydapt, a breakthrough medicine, to patients with serious and hard-to-treat diseases where there are few treatment options,” said Bruno Strigini, CEO, Novartis Oncology. “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.”