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FDA Approves Koselugo for Adults With NF1-Related Plexiform Neurofibromas

The decision rests on phase 3 evidence showing clinically meaningful tumor responses versus placebo.

Overview

  • The FDA authorized selumetinib (Koselugo) for adult patients with neurofibromatosis type 1 who have symptomatic, inoperable plexiform neurofibromas.
  • Approval on November 19, 2025, was supported by the phase 3 KOMET trial, which reported a confirmed overall response rate of 20% for selumetinib versus 5% for placebo.
  • Among responders, 86% maintained a duration of response of at least six months in the selumetinib arm.
  • Trial safety findings were consistent with the drug’s established profile from pediatric use.
  • The adult authorization follows a September 2025 U.S. approval of selumetinib granules and capsules for pediatric patients aged one year and older.