Novartis wins EU approval for Rydapt in rare forms of cancer

Switzerland’s Novartis has been approved to market Rydapt (midostaurin) in two indications in rare, hard-to-treat cancers.

The therapy is approved in combination with chemotherapy for certain patients with newly diagnosed acute myeloid leukemia (AML), and as monotherapy to treat certain kinds of systemic mastocytosis (SM) or mast cell leukemia.

Rydapt is the first and only targeted therapy for this kind of AML and the only treatment for three subtypes of SM, collectively known as advanced SM, in Europe.

The application was supported by data from the RATIFY trial, which showed a 23% reduction in the risk of death for AML patients treated with Rydapt plus standard chemotherapy compared with placebo plus standard chemotherapy.

For advanced SM, the approval is based on two trials, including a Phase II study (CPKC412D2201), the largest prospective trial ever conducted in this rare disorder.