US approves Novartis' Rydapt for acute myeloid leukaemia, systemic mastocytosis

Novartis announced Friday that the FDA cleared its drug Rydapt (midostaurin), in combination with chemotherapy, to treat adults newly diagnosed with acute myeloid leukaemia (AML) who carry the FLT3 genetic mutation. Invivoscribe Technologies' LeukoStrat companion diagnostic test was simultaneously approved to detect the mutation. The FDA also cleared Rydapt for adults with advanced systemic mastocytosis, which includes aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated haematological neoplasm and mast cell leukaemia. 

Richard Pazdur, acting director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research, stated "Rydapt is the first targeted therapy to treat patients with AML, in combination with chemotherapy."

The safety and efficacy of the drug as an AML treatment were supported by data from the 717-patient Phase III RATIFY study. Results, which were unveiled in 2015, demonstrated that the addition of Rydapt, formerly known as PKC412, to chemotherapy in patients with newly diagnosed FLT3-positve AML reduced the risk of death by 23 percent versus chemotherapy alone. 

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Meanwhile, the therapy's approval for systemic mastocytosis was backed by safety and efficacy data from two single-arm clinical trials, including the Phase II CPKC412D2201 study. In results released last year, the median overall survival was 28.7 months, while the median duration of response in the primary efficacy population was 24.1 months. 

Company spokeswoman Julie Masow said Rydapt will be available as of the start of next week, with the US list price for AML patients being $7495 for a 14-day treatment duration and $14 990 for a 28-day duration. She noted that the median duration of therapy in a late-stage trial of patients with AML was 42 days, translating to a list price of $22 485. Meanwhile, the US list price for patients with ASM is $32 121 for a 30-day treatment duration, with Masow indicating that the median duration of therapy in clinical trials was 11.4 months.

The FDA previously awarded Rydapt a breakthrough therapy designation for AML and priority review status for both AML and systemic mastocytosis.  

For related analysis, see Analyst Notes: FLT3 inhibitors are gearing up to join the AML treatment algorithm.

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