Novartis receives positive CHMP opinion for Rydapt

If approved, Rydapt would represent the first targeted treatment for newly diagnosed FLT3-mutated AML in the EU.

Tthe European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending approval of Novartis's Rydapt (midostaurin) for the treatment of adults with newly diagnosed acute myeloid leukemia (AML) who are FLT3 mutation-positive. If approved by the European Commission (EC), Rydapt will be indicated 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 adult patients with newly diagnosed AML who are FLT3 mutation-positive. Rydapt was also recommended for approval as monotherapy for the treatment of adult patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematological neoplasm (SM-AHN) or mast cell leukemia.

If approved, Rydapt will be the first targeted treatment available in the European Union (EU) for newly diagnosed FLT3 mutation-positive AML patients and advanced systemic mastocytosis (SM) patients. The opinion follows the recent FDA 2017 approval of Rydapt for FLT3-mutated AML and advanced SM on 28 April and the Swissmedic approval on 4 May.

"Novartis is dedicated to bringing new treatment options to patients with rare diseases, including AML and advanced SM, which have seen limited treatment developments in the past 25 years," said Bruno Strigini, CEO, Novartis Oncology. "We are pleased with the positive recommendation from the CHMP and excited to move a step closer to bringing this much-needed treatment to these patients across Europe."

FMS-like tyrosine kinase 3 (FLT3) is a type of cell-surface receptor which plays a role in increasing the number of certain blood cells and the FLT3 gene mutation can result in faster disease progression, higher relapse rates and lower rates of survival than other forms of AML. Prior to the approval of Rydapt in the US, the AML therapeutic strategy had remained relatively unchanged for more than 25 years.

Advanced SM is a rare blood disorder characterized by uncontrolled growth and accumulation of mast cells - mediators of allergic responses - in one or more organs. In advanced SM, mast cells accumulate in such high quantities that they begin to cause organ damage. Median overall survival is currently less than 6 months for mast cell leukemia, 2 years for SM-AHN and 3.5 years for ASM.

The EC typically adheres to the recommendation of the CHMP and delivers its final decision within approximately 2 to 3 months. The decision will be applicable to all 28 EU member states, plus Iceland, Liechtenstein and Norway.