Selumetinib (KOSELUGOTM; AZD6244, ARRY-142886) is a mitogen-activated protein kinase 1 and 2 (MEK1/2) inhibitor being developed by AstraZeneca for the treatment of tumours associated with neurofibromatosis and various cancers. Selumetinib has been granted orphan drug status as adjuvant treatment for thyroid cancer (in the USA) and as treatment for neurofibromatosis type 1 (in the USA and the EU) and, based on the results of the phase II SPRINT trial, was recently approved in the USA in paediatric patients with neurofibromatosis type 1 and symptomatic, inoperable plexiform neurofibromas. This article summarizes the milestones in the development of selumetinib leading to this first approval for the treatment of paediatric patients aged ≥ 2 years with neurofibromatosis type 1 who have symptomatic, inoperable plexiform neurofibromas.
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The preparation of this review was not supported by any external funding.
Conflict of interest
During the peer review process the manufacturer of the agent under review was offered an opportunity to comment on the article. Changes resulting from any comments received were made by the authors on the basis of scientific completeness and accuracy. Anthony Markham, a contracted employee of Adis International Ltd/Springer Nature and Susan Keam, a salaried employee of Adis International Ltd/Springer Nature are responsible for the article content and declare no relevant conflicts of interest.
Enhanced material for this AdisInsight Report can be found at https://doi.org/10.6084/m9.figshare.12363758.
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Markham, A., Keam, S.J. Selumetinib: First Approval. Drugs 80, 931–937 (2020). https://doi.org/10.1007/s40265-020-01331-x