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midostaurin（商品名：Rydapt Capsules）参比制剂，抗癌新药，美国产品，一次性进口(二)
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FDA approval for Rydapt® in newly diagnosed FLT3-mutated acute myeloid leukemia (AML) and three types of systemic mastocytosis (SM)
US Food and Drug Administration (FDA) has approved Rydapt® (midostaurin, formerly PKC412) for two indications. The first indication is for the treatment of acute myeloid leukemia (AML) in newly diagnosed patients who are FMS-like tyrosine kinase 3mutation-positive (FLT3+), 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[3]. This approval follows a prior Breakthrough Therapy designation in FLT3-mutated AML, as well as Orphan Drug designation and Priority Review in both indications by the FDA. Worldwide filings for Rydapt are currently underway.
"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."
Rydapt is indicated for use in combination with standard cytarabine and daunorubicin induction and cytarabine consolidation chemotherapy, for the treatment of adult patients with newly diagnosed AML who are FLT3 mutation-positive, as detected by an FDA-approved test. Rydapt is not indicated as a single-agent induction therapy for the treatment of patients with AML.
The FDA approval is based on the Phase III RATIFY (CALGB 10603[Alliance]) clinical trial, which was conducted in collaboration with the Alliance for Clinical Trials in Oncology and its 13 contributing international cooperative groups.
In the trial, newly diagnosed FLT3+ patients who received Rydapt plus chemotherapy experienced significant improvement in overall survival with a 23% reduction in the risk of death compared with chemotherapy alone (hazard ratio [HR] = 0.77, 95%confidence interval[CI], 0.63, 0.95; 2 sided p=0.016).
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