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Mirdametinib is an oral, small molecule MEK inhibitor approved in the United States for the treatment of adults and pediatric patients 2 years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete surgical resection[2][3][5]. It acts by selectively inhibiting MEK1 and MEK2, key kinases in the MAPK pathway that regulate cell growth and survival. By blocking these proteins, mirdametinib helps control abnormal cell proliferation associated with NF1-PN[3][6][8]. The drug was developed by SpringWorks Therapeutics and received FDA approval based on results from the Phase 2b ReNeu trial. Mirdametinib is also being investigated for pediatric low-grade glioma (LGG)[3][6].
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