Mirdametinib: FDA approved MEK inhibitor for neurofibromatosis type 1.

Mirdametinib is an MEK1/2 inhibitor having therapeutic potential for the treatment of neurofibromatosis type 1 (NF1)-associated plexiform neurofibromas (PNs). NF1 is a rare and progressive genetic disorder. As a selective MAPK pathway modulator, it targets the dysregulated RAS (Rat Sarcoma viral oncogene homolog)/RAF (Rapidly Accelerated Fibrosarcoma)/MEK (Mitogen-activated protein kinase)/ (ERK) Extracellular signal-regulated kinase) signaling cascade, which plays a crucial role in NF1 tumor growth. PN, often progressive and disfiguring, significantly impairs quality of life (QOL), and it can transform into malignant peripheral nerve sheath tumors (MPNST). In February 2025, mirdametinib was FDA-approved under the brand name GOMEKLI for pediatric and adult patients with inoperable PNs.