Work

Mirdametinib approval for neurofibromatosis type 1 with inoperable plexiform neurofibromas: the journey of MEK inhibitors

Year: 2025

Type: article

Abstract: Neurofibromatosis type I (NF-1) is an autosomal dominant genetic disorder, regularly accompanied by plexiform neurofibroma. Surgical intervention for plexiform neurofibromas is reserved for high-risk cases, while recent studies have explored various treatments, including mitogen-activated protein kinase kinase (MEK) inhibitors, which target the RAS/mitogen-activated protein kinase (MAPK) pathway, a key driver of NF 1 -associated tumorigenesis. The United States Food and Drug Administration (FDA) has recently approved mirdametinib for adult and pediatric NF-1 patients aged 2 years or older with symptomatic and inoperable plexiform neurofibroma in view of its significant impact on tumor volume reduction. In this article, we review the current evidence on mirdametinib's mechanisms of action and effectiveness in the management of NF-1.

Source: ESMO rare cancers.

DOI: 10.1016/j.esmorc.2025.100015

Authors: M.A. Akbarzadeh, Y. Vaez-Gharamaleki, M.-S. Hosseini

Institution: Tabriz University of Medical Sciences

Language: English

Topic: Neurofibromatosis and Schwannoma Cases

Subfield: Neurology

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: hybrid

Estimated APC paid: $3,170