UK NICE approves use of Rydapt in acute myeloid leukaemia.- Novartis.

NICE has announced that it has approved the use of Rydapt (midostaurin), from Novartis, for patients with acute myeloid leukaemia who have mutations to the FLT3 gene, after an initial negative decision by NICE in December 2017. In trials, Rydapt was found to significantly improve survival and reduce relapse rates, and can be taken as maintenance treatment for a year after chemotherapy treatment has finished. The drug was linked with a 22 percent reduction in the risk of death compared with chemotherapy alone.

Comment: Approximately 30% of patients with AML have a genetic mutation within the FLT3 gene. A mutation within the FLT3 gene results in continuous signals to the cancer cells, causing them to grow in an uncontrolled manner, and preventing them from natural death. These patients have a poorer survival than other patients with AML treated with standard therapeutic approaches. Rydapt is an agent that blocks the growth signal caused by the FLT3 mutation, resulting in cellular death.