FDA approves 1st targeted drug for certain leukemia patients

Endpoints News

Endpoints News

Midostaurin (Rydapt) was approved by the FDA for the treatment of adults with newly diagnosed acute myeloid leukemia (AML), who are FLT3 mutation-positive in combination with cytarabine and daunorubicin induction and cytarabine consolidation.

The FDA has approved midostaurin (Rydapt, Novartis) for the treatment of adult patients with newly diagnosed acute myeloid leukemia (AML), in combination with chemotherapy.

Rydapt is a kinase inhibitor that blocks several enzymes that promote cell growth.

Data has shown that using Rydapt as part of the treatment regimen for FLT3-mutated AML patients resulted in a 23 percent reduction in the risk of death, Novartis said.

Researchers randomly assigned patients to placebo or 50 mg oral midostaurin twice daily on days 8 to 21 of each cycle of induction and consolidation chemotherapy, followed by continuous daily midostaurin for up to 12 cycles.

Separately, the FDA also approved midostaurin for a group of related rare blood disorders (aggressive systemic mastocytosis, systemic mastocytosis with associated hematologic neoplasm, and mast cell leukemia). The FDA also approved LeukoStrat CDx FLT3 Mutations Assay as a companion diagnostic for use with midostaurin to test patients with AML for the FLT3 mutation.

The drug, which bagged a breakthrough tag from the FDA past year, charted a median overall survival of 74.7 months in a Phase 3 trial, beating out the 25.6 months seen on placebo alone. The 5-year EFS rate with midostaurin was 27.5% versus 19.3% with placebo.

Rydapt was evaluated in a randomized study (n=717) in patients who were not previously treated for AML. The median duration of therapy (100 mg administered twice a day) in clinical trials was 11.4 months, Masow said.

Common side effects included low white cell count, fever, nausea, headache and muscular/bone pain. The most frequent Grade 3 or greater adverse reactions (incidence greater than or equal to 5%), excluding laboratory terms, were fatigue, sepsis, gastrointestinal hemorrhage, pneumonia, diarrhea, febrile neutropenia, edema, dyspnea, nausea, vomiting, abdominal pain and renal insufficiency.

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