FDA approves Koselugo as first U.S. drug to treat type 1 neurofibromatosis in children
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Last Update: 2020-05-30
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Source: Internet
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Author: User
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The FDA announced Friday (April 10, 2020) that it has approved AstraZeneca and Merck and Merck's Koselugo for the treatment of children with type 1 neurofibromatoma (NF1) ages 2 and older, the first FDA-approved treatment for NF1Specifically, Koselugo is suitable for children with symptomatic, surgically invasive neurofibromatosis"For the first time, pediatric patients have FDA-approved drugs to treat clumps of neurofibromatoma, a rare tumor associated with NF1," said Richard Pazdur, director of the FDA's Center for Oncology Researchthe approval is based on a clinical trial by the U.SNational Cancer Institute in children with NF1 and unsurgical clumps of neurofibromatomaPatients took oral selumetinib twice a day during the 28-day cycle and regularly assessed tumor size and tumor changesThe results showed that the overall remission rate (ORR) was 66% in patients and that all patients had partial remission (PR), of which 82% were for 12 months or more
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