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Mirdametinib keeps getting better with time. Updated data from the phase 2b ReNeu trial show that adults and children with NF1-associated plexiform neurofibromas who stayed on the drug longer saw deeper tumor shrinkage — with nearly half of adults and over half of kids achieving confirmed responses. Even better, treatment-related side effects actually declined over the years.
Mirdametinib (Gomekli) just got even more impressive. Long-term data from the phase 2b ReNeu trial, presented at the 2026 Society for Neuro-Oncology Annual Meeting, show that patients with neurofibromatosis type 1 (NF1)–associated plexiform neurofibromas who stayed on the drug longer achieved deeper and more frequent tumor responses — with no new safety concerns emerging over time.
After a median treatment exposure of nearly two years, confirmed objective response rates reached 46.6% in adults and 55.4% in children. Among those who responded, roughly two-thirds achieved a "deep response" — meaning their tumors shrank by more than 50%. Crucially, side effects that were most common in year one became less frequent with continued treatment, suggesting the drug gets easier to tolerate over time.
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Why it matters: NF1-associated plexiform neurofibromas are notoriously difficult to treat, often causing significant pain and disability. These updated findings reinforce that mirdametinib — FDA-approved since February 2025 — delivers compounding benefits the longer patients stay on it, offering real hope for both kids and adults living with this rare condition.
