Mirdametinib: First Approval
Declarations
Funding The preparation of this review was not supported by any external funding.
Authorship and 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. Sheridan M. Hoy is a salaried employee of Adis International Ltd/Springer Nature, and declares no relevant conflicts of interest. All authors contributed to this article and are responsible for its content.
Ethics approval, Consent to participate, Consent to publish, Availability of data and material, Code availability Not applicable.
Additional information about this Adis Drug Review can be found here.
Abstract
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.
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