The US FDA has approved mirdametinib, a kinase inhibitor, for treating adult and pediatric patients aged two years and older with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection. Clinical evidence In a clinical trial involving 114 patients, mirdametinib achieved an overall response rate (ORR) of 41% in adults and 52% in pediatric patients. In addition, 88% of adult and 90% of pediatric patients demonstrated a duration of response of 12 months or more.
Moreover, 50% of adult and 48% of pediatric patients showed a duration of response of 24 months or more. Dosage and administration Available as capsules and tablets 2 mg/m2 orally twice daily, with or without food Warnings and precautions Ocular toxicity Left ventricular dysfunction Dermatologic adverse reactions Embryo-fetal toxicity Adverse reactions In adult patients:…
