Mirdametinib

DB07101ApprovedInvestigationalSmall moleculeKinase Inhibitor

Mirdametinib is a kinase inhibitor used to treat neurofibromatosis type 1 in children and adults. Mirdametinib is a mitogen-activated protein kinase (MAP2K, MEK, MAPKK) inhibitor.

Mechanism: NF1 is caused by the loss-of-function variants in the NF1 tumour suppressor gene, which encodes neurofibromin: Neurofibromin loss in tumour cells leads to dysregulated Ras signalling with hyperactivation of downstream Ras effectors, including mitogen-activated protein kinase (MAPK, MEK) and its downstream effector extracellular signal–regulated protein kinase (ERK). As the MAPK pathway regulates cellular functions such as cellular growth and senescence, dysregulation of this pathway aberrant glioblastoma cell growth.
Mirdametinib is a highly selective, non-competitive inhibitor of MEK1/2. In vitro, mirdametinib inhibited kinase activity of MEK1 and MEK2 and downstream phosphorylation of ERK.
Curator reviewed · 6 references
Primary indication: Mirdametinib is indicated for the treatment of neurofibromatosis type 1 (NF1) in patients two years and older with symptomatic plexiform neurofibromas (PN) that are not amenable to complete resection.Curator reviewed · 1 structured indication
Formula / weight: C₁₆H₁₄F₃IN₂O₄ · 482.193 g/mol (avg)
First approval: United States, 2025 · European Union, 2025
Also known as: (-)-N-(((R)-2,3-DIHYDROXYPROPYL)OXY)-3,4-DIFLUORO-2-((2-FLUORO-4-IODOPHENYL)AMINO)BENZAMIDE · Benzamide, N-((2R)-2,3-dihydroxypropoxy)-3,4-difluoro-2-((2-fluoro-4-iodophenyl)amino)- · N-(((R)-2,3-DIHYDROXYPROPYL)OXY)-3,4-DIFLUORO-2-((2-FLUORO-4-IODOPHENYL)AMINO)BENZAMIDE · N-(((R)-2,3-DIHYDROXYPROPYL)OXY)-3,4-DIFLUORO-2-((2-FLUORO-4-IODOPHENYL)AMINO)BENZAMIDE N-((2R)-2,3-DIHYDROXYPROPOXY)-3,4-DIFLUORO-2-(2-FLUORO-4-IODOANILINO)BENZAMIDE · N-[(2R)-2,3 Dihydroxypropoxy]-3,4 Difluro-2 -[(2-Fluoro-4-Iodophenyl)Amino] Benzamide
Code names: PD 0325901 · PD-325901 · PD 901
Brand names: Gomekli