# Selumetinib

Source: https://onco.cc/drugs/selumetinib/  
OnCo record `selumetinib` (Treatment). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

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.

## Fields

- Kind: Treatment
- Status: approved
- Last checked: 2026-09-10
- Tags: nci-list
- 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: 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)

## Notes

- NF1 plexiform neurofibroma is a benign tumour with no cancer record; not linked to a cancer id.

## Sources

- Wikipedia: https://en.wikipedia.org/wiki/Selumetinib
- US label (DailyMed): https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=selumetinib
- NCI drug page: https://www.cancer.gov/about-cancer/treatment/drugs/selumetinibsulfate
- EPAR: https://www.ema.europa.eu/en/medicines/human/EPAR/koselugo

## Connected records

- drugs: [Mirdametinib](https://onco.cc/drugs/mirdametinib/), [Trametinib](https://onco.cc/drugs/trametinib/)
- technologies: [Small-molecule kinase inhibitors](https://onco.cc/technologies/kinase-inhibitors/)
- targets: [MEK1/2](https://onco.cc/targets/mek/), [NF1 (neurofibromin)](https://onco.cc/targets/nf1/)
- companies: [AstraZeneca](https://onco.cc/companies/astrazeneca/), [Merck & Co. (MSD)](https://onco.cc/companies/merck/)
- pathways: [Glioma (KEGG map)](https://onco.cc/pathways/glioma-signalling/), [RAS / RAF / MEK / ERK (MAPK)](https://onco.cc/pathways/ras-mapk/), [Thyroid cancer (KEGG map)](https://onco.cc/pathways/thyroid-cancer-signalling/)
- trials: [A Study of Different Dosing Schedules of Selumetinib With Cisplatin/Gemcitabine (CIS/GEM) Versus CIS/GEM Alone in Biliary Cancer](https://onco.cc/trials/nct02151084/), [Assess Efficacy & Safety of Selumetinib in Combination With Docetaxel in Patients Receiving 2nd Line Treatment for v-Ki-ras2 Kirsten Rat Sarcoma Viral](https://onco.cc/trials/nct01933932/), [National Lung Matrix Trial](https://onco.cc/trials/national-lung-matrix-trial/), [Testing the Combination of Anti-Cancer Drugs, Selumetinib and DS-8201a (Trastuzumab Deruxtecan), for Advanced Pancreatic Ductal Adenocarcinoma](https://onco.cc/trials/nct07619521/)
- people: [D. Wade Clapp](https://onco.cc/people/d-wade-clapp/)
- roadmaps: [Paediatric oncology roadmap: cooperative-group cures → engineered immunity → drugs developed for children first](https://onco.cc/roadmaps/paediatric-oncology-roadmap/)
- cancers: [Malignant peripheral nerve sheath tumour (MPNST)](https://onco.cc/cancers/malignant-peripheral-nerve-sheath-tumour/), [Optic pathway glioma](https://onco.cc/cancers/optic-pathway-glioma/)

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