Novartis’s Rydapt has been approved by the FDA for the treatment of acute myeloid leukemia, and for treating adults with advanced systemic mastocytosis.
The first treatment will be used for newly diagnosed patients whoa re FMS-like tyrosine kinase 3 mutation-positive, as detected by an FDA-approved test, in combination with chemotherapy. Rydapt is also approved to treat adults with advanced systemic mastocytosis (SM), which includes aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematological neoplasm (SM-AHN) and mast cell leukemia.
“Rydapt represents a remarkable advance as the first and only targeted therapy approved for patients who had limited options for many years,” said Bruno Strigini, CEO, Novartis Oncology. “We are proud to continue our leadership in hematology as we work diligently to bring innovative medicines to patients worldwide.”
Rydapt is the only approved targeted therapy for newly diagnosed FLT3-mutated AML
AML is a rare and aggressive cancer of the blood and bone marrow. In the US, about 21,000 people are estimated to be diagnosed with AML in 2017. Approximately one-third of these AML patients, or 7,000, will have a FLT3 gene mutation. FLT3 is a type of cell-surface receptor which plays a role in increasing the number of certain blood cells. The FLT3 gene mutation can result in faster disease progression, higher relapse rates and lower rates of survival than other forms of AML. Prior to the approval of Rydapt, the AML therapeutic strategy had remained relatively unchanged for more than 25 years.
“The overall survival advantage for midostaurin plus chemotherapy seen in the RATIFY trial was a significant advancement for newly diagnosed AML patients with the FLT3 mutation,” said Dr. Richard Stone, Chief of Staff and Director of the Adult Leukemia program at Dana-Farber Cancer Institute, and Alliance for Clinical Trials in Oncology study chair for the RATIFY trial. “The availability of midostaurin now helps to establish a new standard of care in this high-risk patient population.”