{"entity":{"id":"selumetinib","kind":"drug","name":"Selumetinib","aka":[],"tldr":"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.","summary":"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.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Selumetinib","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=selumetinib"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/selumetinibsulfate"},{"label":"EPAR","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/koselugo"}],"tags":["nci-list"],"related":["trametinib"],"cancers":[],"sections":[],"technologies":["kinase-inhibitors"],"targets":["mek"],"drugs":[],"companies":["astrazeneca","merck"],"institutions":[],"pathways":["ras-mapk"],"terms":[],"trials":["nct01933932"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["NF1 plexiform neurofibroma is a benign tumour with no cancer record; not linked to a cancer id."],"brand":"Koselugo","modality":"Small-molecule allosteric MEK1/2 inhibitor","mechanism":"Non-ATP-competitive inhibitor of MEK1 and MEK2; in NF1-deficient cells with unrestrained RAS activity it lowers ERK signalling and shrinks plexiform neurofibromas.","approvals":[{"region":"US","year":2020,"indication":"NF1 with symptomatic, inoperable plexiform neurofibromas, children 2 and over (extended to adults and to age 1 and over in 2025)"},{"region":"EU","year":2021,"indication":"Symptomatic, inoperable plexiform neurofibromas in NF1, children 3 and over (conditional)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},"route":"/drugs/selumetinib/","neighbours":{"drug":[{"id":"mirdametinib","kind":"drug","name":"Mirdametinib","route":"/drugs/mirdametinib/"},{"id":"trametinib","kind":"drug","name":"Trametinib","route":"/drugs/trametinib/"}],"technology":[{"id":"kinase-inhibitors","kind":"technology","name":"Small-molecule kinase inhibitors","route":"/technologies/kinase-inhibitors/"}],"target":[{"id":"mek","kind":"target","name":"MEK1/2","route":"/targets/mek/"},{"id":"nf1","kind":"target","name":"NF1 (neurofibromin)","route":"/targets/nf1/"}],"company":[{"id":"astrazeneca","kind":"company","name":"AstraZeneca","route":"/companies/astrazeneca/"},{"id":"merck","kind":"company","name":"Merck & Co. (MSD)","route":"/companies/merck/"}],"pathway":[{"id":"glioma-signalling","kind":"pathway","name":"Glioma (KEGG map)","route":"/pathways/glioma-signalling/"},{"id":"ras-mapk","kind":"pathway","name":"RAS / RAF / MEK / ERK (MAPK)","route":"/pathways/ras-mapk/"},{"id":"thyroid-cancer-signalling","kind":"pathway","name":"Thyroid cancer (KEGG map)","route":"/pathways/thyroid-cancer-signalling/"}],"trial":[{"id":"nct02151084","kind":"trial","name":"A Study of Different Dosing Schedules of Selumetinib With Cisplatin/Gemcitabine (CIS/GEM) Versus CIS/GEM Alone in Biliary Cancer","route":"/trials/nct02151084/"},{"id":"nct01933932","kind":"trial","name":"Assess Efficacy & Safety of Selumetinib in Combination With Docetaxel in Patients Receiving 2nd Line Treatment for v-Ki-ras2 Kirsten Rat Sarcoma Viral","route":"/trials/nct01933932/"},{"id":"national-lung-matrix-trial","kind":"trial","name":"National Lung Matrix Trial","route":"/trials/national-lung-matrix-trial/"},{"id":"nct07619521","kind":"trial","name":"Testing the Combination of Anti-Cancer Drugs, Selumetinib and DS-8201a (Trastuzumab Deruxtecan), for Advanced Pancreatic Ductal Adenocarcinoma","route":"/trials/nct07619521/"}],"person":[{"id":"d-wade-clapp","kind":"person","name":"D. Wade Clapp","route":"/people/d-wade-clapp/"}],"roadmap":[{"id":"paediatric-oncology-roadmap","kind":"roadmap","name":"Paediatric oncology roadmap: cooperative-group cures → engineered immunity → drugs developed for children first","route":"/roadmaps/paediatric-oncology-roadmap/"}],"cancer":[{"id":"malignant-peripheral-nerve-sheath-tumour","kind":"cancer","name":"Malignant peripheral nerve sheath tumour (MPNST)","route":"/cancers/malignant-peripheral-nerve-sheath-tumour/"},{"id":"optic-pathway-glioma","kind":"cancer","name":"Optic pathway glioma","route":"/cancers/optic-pathway-glioma/"}]}}