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<p>Mirdametinib (GOMEKLI<sup>TM</sup>) is an oral small molecule inhibitor of mitogen-activated protein kinase kinases 1&#xa0;and 2&#xa0;(MEK1/2) developed by SpringWorks Therapeutics for the treatment of neurofibromatosis type 1 (NF1)-associated plexiform neurofibromas (PN). It is the first therapy to be approved for the treatment of both adults and children with NF1-associated PN in the USA, where it is indicated for use in adult and paediatric patients 2&#xa0;years of age and older with NF1 who have symptomatic PN not amenable to complete resection. A marketing authorisation application for mirdametinib in NF1-PN is currently under review in the EU. Clinical studies of mirdametinib for the treatment of paediatric low-grade glioma are ongoing. This article summarizes the milestones in the development of mirdametinib leading to this first approval.</p>

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Mirdametinib: First Approval

  • 期刊论文

  • 出版模式：

    开放获取

  • 发表日期：

    2025年5月27日

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  • Sheridan M. Hoy

摘要

Mirdametinib (GOMEKLITM) is an oral small molecule inhibitor of mitogen-activated protein kinase kinases 1 and 2 (MEK1/2) developed by SpringWorks Therapeutics for the treatment of neurofibromatosis type 1 (NF1)-associated plexiform neurofibromas (PN). It is the first therapy to be approved for the treatment of both adults and children with NF1-associated PN in the USA, where it is indicated for use in adult and paediatric patients 2 years of age and older with NF1 who have symptomatic PN not amenable to complete resection. A marketing authorisation application for mirdametinib in NF1-PN is currently under review in the EU. Clinical studies of mirdametinib for the treatment of paediatric low-grade glioma are ongoing. This article summarizes the milestones in the development of mirdametinib leading to this first approval.