Selumetinib
Selumetinib (Koselugo) is the first medicine for children and adults with neurofibromatosis type 1 whose plexiform neurofibromas, benign but disfiguring and painful nerve tumours, cannot be removed by surgery.
Overview
Selumetinib was approved by the FDA in April 2020 for children aged 2 and over with NF1 and symptomatic, inoperable plexiform neurofibromas, on the SPRINT phase 2 stratum 1 trial in which two-thirds of 50 children had a confirmed partial response with improvements in pain and function; approval was extended to adults (KOMET trial) and, with a granule formulation, to children from 1 year in 2025. The EU granted conditional authorisation in 2021. Plexiform neurofibromas are benign tumours, but the drug is an oncology-class MEK inhibitor developed with Merck & Co. Rash, paronychia, diarrhoea, raised creatine kinase, reduced ejection fraction and ocular toxicity are class effects and require monitoring. Mirdametinib (Gomekli, 2025) is the second MEK inhibitor for the same condition.
Approvals
| Region | Year | Indication |
|---|---|---|
| US | 2020 | NF1 with symptomatic, inoperable plexiform neurofibromas, children 2 and over (extended to adults and to age 1 and over in 2025) |
| EU | 2021 | Symptomatic, inoperable plexiform neurofibromas in NF1, children 3 and over (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:"Selumetinib" OR ABSTRACT:"Selumetinib" OR TITLE:"Koselugo" OR ABSTRACT:"Koselugo") 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 Selumetinib, not a curated reading list.
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