What did the FDA just approve for neurofibromatosis type 1?

The FDA approved mirdametinib (brand name Gomekli) for adults and children aged 2 and older with neurofibromatosis type 1 who have symptomatic plexiform neurofibromas that cannot be completely removed by surgery.

The FDA approved mirdametinib (brand name Gomekli) for adults and children aged 2 and older with neurofibromatosis type 1 who have symptomatic plexiform neurofibromas that cannot be completely removed by surgery. It's a kinase inhibitor — it blocks the signals that tell tumor cells to grow — and the approval rested on the ReNeu trial of 114 patients (58 adults, 56 children), measuring how many saw meaningful tumor shrinkage. Be clear about what this is and isn't: it's a targeted therapy for a specific, hard-to-treat complication of NF1 — not a cure for NF1 itself, and not a general "tumor drug." For families who've had no options beyond impossible surgeries, it's a genuine new tool.