A Novel Drug
Midostaurin, PKC412, developed by the Swiss pharmaceutical company Novartis, has been granted a novel drug status by FDA.
Preliminary clinical data specify the ability of midostaurin to provide significant increase in efficacy of treatment of myeloid leukemia versus current drugs. However, the drug has not yet been sufficiently studied. Positive results of clinical trials performed in sufficient quantity, midostaurin may become a good treatment for adult patients with newly diagnosed acute myeloid leukemia with FLT3 mutation.
Currently it may only be confirmed that in phase III clinical trial Ratify in patients receiving midostaurin and chemotherapy increase of mean overall survival versus those who only had a course of chemotherapy. No significant difference was observed in adverse events of grade 3 and above. However, the full safety and efficacy profile of the new drug has not been established. The status of novel drug will help accelerate the work over it and conduct all the needed trials to make the drug available to patients as soon as possible, as the first target therapy for acute myeloid leukemia.