On April 28, 2017, Rydapt (midostaurin; from Novartis) became the first targeted therapy, and the first new drug in more than 20 years, to be approved by the FDA, in combination with chemotherapy, for the treatment of acute myeloid leukemia (AML) plus the FLT3 genetic mutation in adults who have not received any treatment before. Rydapt is a kinase inhibitor and comes in capsules to be taken orally. Before patients can receive Rydapt, they need to be tested by a companion diagnostic, the LeukoStrat CDx FLT3 Mutation Assay, to verify that they have the FLT3 mutation.
“Rydapt is the first targeted therapy to treat patients with AML, in combination with chemotherapy. The ability to detect the gene mutation with a diagnostic test means doctors can identify specific patients who may benefit from this treatment,” said Richard Pazdur, MD, Director of the FDA’s Oncology Center of Excellence.
On the same day, the FDA also approved Rydapt for the treatment of adults with other types of rare blood disorders, including aggressive systemic mastocytosis, systemic mastocytosis with associated hematologic neoplasm, and mast-cell leukemia.
In the clinical trial that led to the approval of Rydapt, patients who received this drug had better outcomes compared with those who received a placebo, with longer survival and longer periods without any undesirable events.
The most common side effects reported in patients with AML who received Rydapt were febrile neutropenia, nausea, mucositis, vomiting, headache, several types of infection, and pain. The most common side effects reported in patients with mastocytosis or mast-cell leukemia were nausea, vomiting, diarrhea, edema, pain, fatigue, respiratory infection, constipation, fever, and headache.
