On November 19, 2025, the Food and Drug Administration approved selumetinib for adults with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN). FDA previously approved selumetinib capsules and granules for paediatric patients 1 year of age and older for this indication.
Full prescribing information for selumetinib will be posted on Drugs@FDA.
Efficacy was evaluated in KOMET (NCT04924608), a global, randomised, multicenter, double-blind, placebo-controlled trial. Eligible patients were required to be 18 years of age or older with NF1 and symptomatic, inoperable PN. Inoperable PN was defined as a PN that could not be completely removed without risk for substantial morbidity due to encasement or proximity to vital structures, invasiveness, or high vascularity. A total of 145 patients were randomized (1:1) to selumetinib or placebo twice daily for 12 cycles.
The major efficacy outcome measure was confirmed overall response rate (ORR) by the end of Cycle 16, determined by independent central review (Response Evaluation in Neurofibromatosis and Schwannomatosis criteria). Duration of response (DOR) was an additional efficacy outcome measure. Confirmed ORR was 20% (95% CI: 11, 31) in the selumetinib arm compared to 5% (95% CI: 2, 13) for those receiving placebo (p-value 0.011), with 86% in the selumetinib arm having an observed DOR of at least 6 months.
The prescribing information includes warnings and precautions for Left Ventricular Dysfunction, Ocular Toxicity, Gastrointestinal Toxicity, Skin Toxicity, Increased Creatine Phosphokinase, Increased Levels of Vitamin E, and Increased Risk of Bleeding (KOSELUGO capsules), and Embryo-Fetal Toxicity. Adverse reactions observed in adult patients receiving selumetinib were consistent with the known selumetinib safety profile.
The recommended selumetinib dose, based on body surface area, is 25 mg/m2 orally twice daily, until disease progression or unacceptable toxicity.
This review used the Assessment Aid, a voluntary submission from the applicant to facilitate the FDA’s assessment.
Selumetinib received orphan drug designation.
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Source: FDA
