Mirdametinib
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.
Overview
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.
Approvals
| 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) |
Trials
topRecruiting now (live from ClinicalTrials.gov)
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Notes
top- NF1 plexiform neurofibroma is a benign tumour with no cancer record; not linked to a cancer id.
Latest papers
topQuery for this drug: (TITLE:"Mirdametinib" OR ABSTRACT:"Mirdametinib" OR TITLE:"Gomekli" OR ABSTRACT:"Gomekli" OR TITLE:"Ezmekly" OR ABSTRACT:"Ezmekly" 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.
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