GOMEKLI mirdametinib
Drug Class
MEK1/2 kinase inhibitorMEK1/2 选择性激酶抑制剂
Mechanism
Selectively inhibits MEK1 and MEK2 proteins, blocking downstream mitogen-activated protein kinase (MAPK) signaling cascade involved in neurofibroma cell growth.选择性结合并抑制 MEK1 和 MEK2 蛋白的激活，从而阻断神经纤维瘤细胞生长所需的下游丝裂原活化蛋白激酶 (MAPK) 级联反应。
Approved Indication
FDA-approved for the treatment of adult and pediatric patients (2 years and older) with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete surgery.FDA批准用于治疗伴有症状性、且无法通过手术完整切除的丛状神经纤维瘤 (PN) 的 1 型神经纤维瘤病 (NF1) 成年及 2 岁及以上儿童患者。
What This Means For Patients
GOMEKLI is a targeted MEK inhibitor designed for neurofibromatosis type 1 with plexiform neurofibromas. It is approved by the FDA and accessible for clinical use in Hainan Boao Lecheng.GOMEKLI 是一种针对 1 型神经纤维瘤病伴丛状神经纤维瘤的口服选择性 MEK 抑制剂。其已获 FDA 批准，可在海南博鳌先行区内申请临床特许使用。
Regulatory Approvals
- FDA — approved (2025) — source
Pivotal Clinical Evidence
- ReNeu — SpringWorks, J Clin Oncol 2024 (NCT03962543) — DOI
Sources
This page provides regulatory and mechanism-of-action information for reference only. It is not medical advice, a treatment recommendation, or a guarantee of outcome. Availability is subject to individual clinical review.