January 2026

Below is in response to the inquiry: A 6-year-old patient with neurofibromatosis type 1 has inoperable plexiform neurofibromas causing significant morbidity. It covers a significant portion of their body with BSA 1.60 m^2. I've come across Gomekli (mirdametinib) as a treatment option. Would Gomekli be an appropriate systemic option for managing their condition? What should be done if they have adverse reactions?

Gomekli (mirdametinib) would be an appropriate systemic treatment option for this 6-year-old child with neurofibromatosis type 1 and symptomatic, inoperable plexiform neurofibromas (PN). Mirdametinib is FDA-approved for adults and children 2 years of age and older whose PN are not amenable to complete resection. Because this patient’s BSA is 1.60 m², they qualify for the full recommended pediatric dose of 4 mg twice daily on days 1 to 21 of a 28 day cycle.

Clinical evidence from the ReNeu Phase IIb trial supports its use in this population. In children aged 2 to 17 years (n=56), 52 percent achieved a confirmed 20 percent or greater reduction in PN volume, with a median best reduction of 42 percent and durable responses beyond 12 to 24 months (Moertel et al., 2024). Morbidities such as pain, functional limitation, and disfigurement often improved on therapy, which aligns with this patient’s presentation. Before treatment, baseline echocardiogram and a comprehensive ophthalmologic exam are required because of risks of left ventricular dysfunction and ocular toxicity.

If adverse reactions develop, management is based on severity. For intolerable Grade 2 or Grade 3 reactions including rash, diarrhea, or abdominal pain, Gomekli should be withheld until toxicity improves to Grade 1 or baseline, then resumed at a reduced dose of 3 mg twice daily for this BSA category. More serious toxicities require stronger actions. Retinal vein occlusion requires permanent discontinuation, symptomatic retinal pigment epithelium detachment requires treatment interruption, and an LVEF decline of 20 percent or greater from baseline also requires permanent discontinuation.

Overall, sufficient evidence supports the use of Gomekli as a systemic option for this patient, with clear guidance available for monitoring and managing adverse reactions.

Reference:

1. Gomekli®. (n.d.). GOMEKLI® (mirdametinib). GOMEKLI DTC. https://www.gomekli.com/?utm_medium=ppc&utm_source=google&utm_term=gomekli&utm_id=PS250908150301&gad_source=1

2. Moertel CL, Hirbe AC, Shuhaiber HH, et al. ReNeu: A Pivotal, Phase IIb Trial of Mirdametinib in Adults and Children With Symptomatic Neurofibromatosis Type 1-Associated Plexiform Neurofibroma. J Clin Oncol. 2025;43(6):716-729. doi:10.1200/JCO.24.01034