Drug intelligence / Profile preview

mirdametinib

Development stage
Approved
Lead developer
SpringWorks Therapeutics
Modality
Small Molecules
Administration
Oral
01

Overview

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].

Brand names
Gomekli
Other names
mirdametinibPD-0325901PD0325901PD 0325901PD325901PD-325901PD 325901
02

Targets

MEK1 (Dual specificity mitogen-activated protein kinase kinase 1)MEK2 (Dual specificity mitogen-activated protein kinase kinase 2)

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