New Products

Novartis bags FDA OK for Rydapt, adding a new chemo combo drug for AML

Novartis has picked up a new drug approval, this time for Rydapt (midostaurin, PKC412) for newly diagnosed cases of acute myeloid leukemia among patients with a specific FLT3 genetic mutation. The drug will be used in combination with a chemotherapy and there’s a diagnostic available to ID the right patients for this drug.

Vasant Narasimhan, Novartis

The drug, designated as a breakthrough therapy at the FDA, is a kinase inhibitor that tackles enzymes associated with the advance of the blood cancer.

The agency’s statement on the approval — the 16th to date during what is proving to be a much more productive year than last year — notes that investigators never did nail down a reliable median survival benefit in their studies. But Novartis has cited data indicating that the drug delivered a 23% reduction in death risk as well as a median OS rate in one study of 74.7 months compared to 25.6 months for the placebo arm.

The FDA also noted today that the drug combo went 8.2 months longer than the chemo-alone control arm without a set of complications. The review process was facilitated by a priority review designation for Novartis.

“Rydapt is the first targeted therapy to treat patients with AML, in combination with chemotherapy,” said Richard Pazdur, acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research and director of the FDA’s Oncology Center of Excellence. “The ability to detect the gene mutation with a diagnostic test means doctors can identify specific patients who may benefit from this treatment.”


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RAPS Regulatory Convergence 2017