Novartis’ Rydapt receives approval for two cancer treatments in the EU

The European Commission (EC) has approved Novartis’ Rydapt (midostaurin) for two indications in rare, hard-to-treat cancers.

Rydapt is an oral, targeted treatment that interferes with certain pathways involved in the growth and progression of cancer.

The Novartis therapy is approved for use in combination with standard daunorubicin and cytarabine induction and high-dose cytarabine consolidation chemotherapy.

The drug can also be used for people in complete response followed by Rydapt single agent maintenance therapy, for adults suffering from newly diagnosed acute myeloid leukaemia (AML) and are FMS-like tyrosine kinase 3 (FLT3) mutation-positive.

Additionally, it has been approved for use a monotherapy to treat adult patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated haematological neoplasm (SM-AHN) or mast cell leukaemia.

AML is the most common type of acute leukaemia or blood cancer in adults.

ASM leads to the uncontrolled growth of neoplastic mast cells resulting in organ damage, low blood counts and weight loss.

Novartis oncology CEO Bruno Strigini said: "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.

"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."

Rydapt is the first and only targeted therapy for FLT3-mutated AML and the only treatment for three subtypes of SM in the European Union (EU).

The EC approval for Rydapt to treat FLT3-mutated AML is based on data from the RATIFY trial, while the approval for ASM is based on two single-arm open-label multicentre trials, including the Phase II study.

The approval is applicable for all 28 EU member states in addition to Iceland, Liechtenstein and Norway.