The Food and Drug Administration (FDA) has approved Rydapt (midostaurin; Novartis) capsules for the treatment of adults newly diagnosed with acute myeloid leukemia (AML) who have a FLT3 mutation, in combination with chemotherapy. Rydapt is approved for use with CDx FLT3 Mutation Assay (Invivoscribe Technologies), a companion diagnostic used to detect the mutation in patients with AML.

Rydapt also gained FDA approval for the treatment of aggressive systemic mastocytosis, systemic mastocytosis with associated hematological neoplasm, or mast cell leukemia. 

Rydapt was evaluated in a randomized study (n=717) in patients who were not previously treated for AML. Study patients who received Rydapt with chemotherapy had longer survival vs. patients who received chemotherapy alone; a specific median survival rate could not be reliably estimated. Also, the time to complications—failure to achieve complete remission within 60 days of treatment start, disease progression, or death—was longer among patients who received Rydapt with chemotherapy vs. chemotherapy alone (median 8.2 months vs. 3 months). 

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Rydapt works by inhibiting multiple receptor tyrosine kinases. The drug demonstrated the ability to inhibit FLT3 receptor signaling and cell proliferation, and it induced apoptosis in leukemic cells expressing ITD and TKD mutant FLT3 receptors or overexpressing wild type FLT3 and PDGF receptors. It also demonstrated the ability to inhibit KIT signaling, cell proliferation and histamine release and induce apoptosis in mast cells.

Common adverse events reported for patients with AML included febrile neutropenia, nausea, mucositis, vomiting, headache, petechiae, among others. Common adverse events reported for patients with mastocyotsis included nausea, vomiting, diarrhea, edema, musculoskeletal pain, abdominal pain, and others.

“Rydapt is the first targeted therapy to treat patients with AML, in combination with chemotherapy,” said Richard Pazdur, MD, 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.” 

Rydapt will be available as 25mg strength capsules in 56- and 112-count cartons. 

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