FDA approves mirdametinib for symptomatic, inoperable NF1 tumors

The FDA approved mirdametinib (Gomekli) on February 11, 2025, for adults and children 2 years of age and older with neurofibromatosis type 1 (NF1) and symptomatic plexiform neurofibromas that cannot be completely removed. The approval covers tumors causing significant morbidity when surgery cannot remove them without risk of substantial harm.

The FDA evaluated the drug in ReNeu, a multicenter, single-arm trial of 114 patients, including 58 adults and 56 pediatric patients. The confirmed overall response rate, based on tumor disappearance or a reduction in tumor volume of at least 20%, was 41% for adults and 52% for children. Common adverse reactions included rash, diarrhea, musculoskeletal pain and vomiting. The FDA also said mirdametinib can cause left ventricular dysfunction and ocular toxicity. Dosage may be reduced or the drug withheld or discontinued depending on the severity of adverse reactions.