2025 Approvals

GOMEKLI(mirdametinib): A New Treatment for Neurofibromatosis Type 1 Approved by the FDA on February 11 2025

Jasmine C

21 Nov 2025 — 1 min read

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What is it prescribed for?

GOMEKLI is prescribed for adults and children 2 years and older with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) that cannot be completely removed by surgery.

What is the name of the drug and what does it do?

The brand name is GOMEKLI (pronounced "go-MEK-lee"), and the generic name is mirdametinib. GOMEKLI treats plexiform neurofibromas—benign (non-cancerous) tumors that form on nerve tissue—in patients with NF1. It is meant for patients whose tumors are causing symptoms and cannot be safely removed with surgery.

How does it work?

GOMEKLI works by blocking proteins called MEK1 and MEK2, which are part of a signaling pathway (the ERK pathway) involved in cell growth. By inhibiting this pathway, GOMEKLI helps slow down the growth of the tumors.

What did the research discover?

In the ReNeu study which involved 114 patients aged 2 and older. These patients had neurofibromatosis type 1 (NF1) with plexiform neurofibromas (PN) that are inoperable and causing serious health issues. The study showed:

Among adults, 41% of patients had significant tumor shrinkage.
Among pediatric patients, 52% showed significant shrinkage.
Most patients who responded maintained their improvement for over a year, and nearly half maintained it for over two years.
The typical time to see a response was about 8 months.

What are some of the side effects?

Common side effects (occurred in over 25% of patients):

Rash
Diarrhea
Nausea
Vomiting
Musculoskeletal pain (pain in muscles, joints, or bones)
Fatigue

What are the dosage recommendations and how is it prescribed?

GOMEKLI is taken by mouth and comes in two forms: capsules or tablets for oral suspension (that can be mixed with water for easier swallowing).

The recommended dose is 2 mg/m² of body surface area, twice daily for the first 21 days of a 28-day cycle.
Treatment continues until disease worsens or unacceptable side effects occur.
Dosage is adjusted based on patient body size and whether side effects develop.

Source:

SpringWorks Therapeutics. GOMEKLI (mirdametinib) capsules and tablets for oral suspension. Prescribing Information. 2025. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2025/219379Orig1s000lbl.pdf

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