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mirdemetinib
- Development stage
- Unknown
- Lead developer
- SpringWorks Therapeutics
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
Mirdemetinib is an investigational, potent, and highly selective allosteric small molecule inhibitor of mitogen-activated protein kinase kinase 1 and 2 (MEK1/2). Originally discovered by Pfizer and now being developed by SpringWorks Therapeutics, mirdemetinib is designed to inhibit the MAPK/ERK pathway, which is frequently dysregulated in various cancers and genetic disorders. Its primary clinical focus is the treatment of neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN) in both pediatric and adult patients. By binding to an allosteric site on MEK, it prevents the activation of downstream extracellular signal-regulated kinase (ERK), thereby inhibiting tumor growth and survival. The drug has received Orphan Drug, Fast Track, and Breakthrough Therapy designations from the FDA for NF1-PN, and an NDA was submitted in 2024.
- Other names
- mirdemetinib
02
Targets
MEK1 (Dual specificity mitogen-activated protein kinase kinase 1)MEK2 (Dual specificity mitogen-activated protein kinase kinase 2)
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