Regulatory Affairs

Novartis’ Rydapt approved in Europe for AML and rare blood disorders

PBR Staff Writer Published 21 September 2017

Novartis’ cancer drug Rydapt (midostaurin) has been approved by the European Commission (EC) for a mutated form of acute myeloid leukemia (AML) and certain rare blood disorders.

Rydapt has been approved to be used for the hard-to-treat cancers in combination with standard daunorubicin and cytarabine induction and high-dose cytarabine consolidation chemotherapy.

The drug has been approved 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.

Rydapt has also been approved as a monotherapy for three forms of advanced systemic mastocytosis (ASM), related to hematological neoplasm (SM-AHN) or mast cell leukemia.

According to Novartis, Rydapt interferes with certain pathways that are responsible for growth, progression and spread of cancer. The drug inhibits various kinases like FLT3, which play a role in controlling several essential cell processes by stopping the growth and multiplication of cancer cells.

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’s approval for newly diagnosed FLT3-mutated AML was based on the findings of the phase 3 RATIFY trial which established the regimen to significantly improve overall survival by reducing chances of death by 23%.

On the other hand, its approval for advanced SM was based on the positive results of a couple of single-arm trials including a phase 2 study.

In May, Rydapt was approved in the US for the treatment of newly diagnosed FLT3-mutated AML and three types of systemic mastocytosis (SM).

Image: Rydapt Capsules. Photo: courtesy of Novartis AG.