GOMEKLI mirdametinib

Drug Class

MEK1/2 kinase inhibitor

Mechanism

Selectively inhibits MEK1 and MEK2 proteins, blocking downstream mitogen-activated protein kinase (MAPK) signaling cascade involved in neurofibroma cell growth.

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.

What This Means For Patients

GOMEKLI is not distributed through mainland China's retail pharmacy network. Uwell Cloud can help arrange a purchase channel for this medicine, sourced and dispatched from Hong Kong; clinical administration support in China can be coordinated on request. Availability and eligibility for any specific patient are determined by clinical review.

Regulatory Approvals

• FDA — Approved (2025) — Sources

Pivotal Clinical Evidence

• ReNeu — SpringWorks, J Clin Oncol 2024 (NCT03962543) — Sources

Sources

• https://www.fda.gov/drugs/resources-information-approved-drugs
• https://www.springworkstx.com/media/