Mirdametinib para Neurofibromas plexiformes inoperables
FDA Approves Mirdametinib for Patients With NF1 Who Have Symptomatic Plexiform Neurofibromas
The US Food and Drug Administration (FDA) approved mirdametinib (Gomekli), a kinase inhibitor, for adult and paediatric patients aged 2 years and older with neurofibromatosis type 1(NF1) who have symptomatic plexiform neurofibromas not amenable to complete resection.
Efficacy of mirdametinib was
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An inoperable plexiform neurofibromas was defined as a plexiform neurofibroma that could not be completely surgically removed without risk for substantial morbidity due to encasement or close proximity to vital structures, invasiveness, or high vascularity.
The major efficacy outcome measure was confirmed overall response rate (ORR), defined as the percentage of patients with complete response (disappearance of the target plexiform neurofibroma) or partial response (≥20% reduction in plexiform neurofibroma volume). Responses were assessed by blinded independent central review using volumetric MRI analysis per Response Evaluation in Neurofibromatosis and Schwannomatosis criteria, modified to require confirmation of responses within 2 to 6 months during the 24-cycle treatment phase.
Confirmed ORR was 41% for adults and 52% in the paediatric cohort.
The most common adverse reactions in adult patients were rash, diarrhoea, nausea, musculoskeletal pain, vomiting, and fatigue. The most common grade 3 or 4 laboratory abnormality was increased creatine phosphokinase.
The most common adverse reactions in paediatric patients were rash, diarrhoea, musculoskeletal pain, abdominal pain, vomiting, headache, paronychia, left ventricular dysfunction, and nausea. The most common grade 3 or 4 laboratory abnormalities were decreased neutrophil count and increased creatine phosphokinase.
Mirdametinib can also cause left ventricular dysfunction and ocular toxicity including retinal vein occlusion, retinal pigment epithelial detachment, and blurred vision. Mirdametinib should be withheld, dosage reduced or permanently discontinued based on the severity of adverse reactions.
Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA'sMedWatch Reporting System or by calling 1-800-FDA-1088.
SOURCE: US Food and Drug Administration
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posted by dermatica at
June 12, 2025
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