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Mirdametinib (Gomekli in the US, Ezmekly in Europe) is a MEK-blocking capsule or dispersible tablet for adults and children with neurofibromatosis type 1 whose plexiform neurofibromas cannot be removed surgically; it is the first such drug approved for adults.
Mirdametinib was approved by the FDA in February 2025 for adults and children aged 2 and over with NF1 and symptomatic plexiform neurofibromas not amenable to complete resection, on the ReNeu phase 2b trial in which 41% of adults and 52% of children had a confirmed objective response by central review, with deep and durable responses and improvement in pain. The European Commission granted a conditional marketing authorisation for Ezmekly in July 2025 for the same population. It is the first MEK inhibitor approved for adults with this benign but disabling tumour (selumetinib covered children first). Rash, diarrhoea, nausea, musculoskeletal pain, reduced ejection fraction and ocular toxicity are class effects. Developed by SpringWorks Therapeutics, acquired by Merck KGaA in 2025.
Non-ATP-competitive MEK1/2 inhibitor that penetrates the CNS; lowers ERK signalling in NF1-deficient Schwann-lineage cells driving plexiform neurofibromas. Connects to MEK1/2.
1.Mirdametinib slips into a pocket on MEK1/2.
Sources: NICE TA1192. Funding decisions are indication-specific and change monthly; verify with NICE and your treating team.
Compare all products across the US, EU, UK, Japan, China and Australia →
| Region | Year | Indication |
|---|---|---|
| US | 2025 | NF1 with symptomatic plexiform neurofibromas not amenable to complete resection, adults and children 2 and over (Gomekli) |
| EU | 2025 | Symptomatic, inoperable plexiform neurofibromas in NF1, age 2 and over (Ezmekly, conditional) |
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Query for this drug: (TITLE:"Mirdametinib" OR ABSTRACT:"Mirdametinib" OR TITLE:"Gomekli" OR ABSTRACT:"Gomekli" OR TITLE:"Ezmekly" OR ABSTRACT:"Ezmekly" OR TITLE:"PD0325901" OR ABSTRACT:"PD0325901" OR TITLE:"PD 0325901" OR ABSTRACT:"PD 0325901" OR TITLE:"PD-0325901" OR ABSTRACT:"PD-0325901") AND (cancer OR tumor OR tumour OR oncology OR carcinoma OR lymphoma OR leukemia OR leukaemia OR myeloma OR sarcoma OR melanoma OR glioma). Results are unfiltered search hits about Mirdametinib, not a curated reading list.
Shares NF1 (neurofibromin), Selumetinib.
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Shares Mirdametinib in Histiocytic Disorders, RAS / RAF / MEK / ERK (MAPK), Small-molecule kinase inhibitors.
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Shares Mirdametinib in Histiocytic Disorders, RAS / RAF / MEK / ERK (MAPK), Small-molecule kinase inhibitors.