FDA New Drug and Biologic Approvals -- 2017 Year-in-Review

Mary L Windle, PharmD


January 11, 2018

In This Article

Rydapt (midostaurin)


Adults with newly diagnosed acute myeloid leukemia (AML) who are FLT3 mutation-positive. It is also approved for aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematological neoplasm (SM-AHN), or mast cell leukemia (MCL).


Inhibits FLT3 receptor signaling and cell proliferation. Also induces apoptosis in leukemic cells expressing ITD and TKD mutant FLT3 receptors or overexpressing wild type FLT3 and PDGF receptors. Midostaurin also demonstrated the ability to inhibit KIT signaling, cell proliferation, and histamine release and induce apoptosis in mast cells.


AML: 50 mg PO every 12 hr on days 8-21 of each cycle of induction with cytarabine and daunorubicin and on days 8-21 of each cycle of consolidation with high-dose cytarabine.

Systemic mastocytosis: 100 mg PO every 12 hours. Continue treatment until disease progression or unacceptable toxicity.


Approval for AML was based on the CALGB 10603 (RATIFY) study. Patients in the midostaurin plus standard induction and consolidation chemotherapy group experienced a 23% improvement in overall survival (OS) (P=0.0074) compared to those treated with standard induction and consolidation chemotherapy alone. Approval for mastocytosis was from an open-label study in 89 patients with mastocytosis-related organ damage. The overall response rate (ORR) was 60%, and 45% of the patients had a major response, which was defined as complete resolution of at least one type of mastocytosis-related organ damage.


Stone RM, et al. The multi-kinase inhibitor midostaurin prolongs survival compared with placebo in combination with daunorubicin/cytarabine induction, high-dose consolidation, and as maintenance therapy in newly diagnosed acute myeloid leukemia (AML) patients age 18-60 with FLT3 mutations: an international prospective randomized P-controlled double-blind trial (CALGB 10603/RATIFY [Alliance]). Abstract 6, Presented at the American Society of Hematology 57th Annual Meeting; Orlando, FL. 2015 Dec 06.

Gotlib J, et al. Efficacy and Safety of Midostaurin in Advanced Systemic Mastocytosis. N Engl J Med. 2016 Jun 30;374(26):2530-41.


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