FDA OKs Selumetinib in NF1 Symptomatic, Inoperable Plexiform Neurofibromas

Selumetinib (Koselugo) has been approved by the FDA for patients with neurofibromatosis type 1 (NF1) with symptomatic, inoperable plexiform neurofibromas, according to a press release from the FDA.¹

The approval was based on results from the phase 3 KOMET (NCT04924608) trial, which assessed selumetinib vs placebo in patients with NF1 with symptomatic, inoperable plexiform neurofibromas. Overall, 145 patients were randomly assigned to one of the 2 arms and received either selumetinib or placebo twice daily for 12 cycles. The recommended selumetinib dose is based on body surface area, or 25 mg/m², given orally twice a day until disease progression or unacceptable toxicity.

The confirmed overall response rate was 20% (95% CI, 11%-31%) in the selumetinib arm vs 5% (95% CI, 2%-13%) in the placebo arm (P = .011). In the selumetinib arm, 86% of patients had an observed duration of response of at least 6 months.

Warnings in the prescribing information include left ventricular dysfunction, ocular toxicity, gastrointestinal toxicity, and skin toxicity.

Previously, the FDA approved selumetinib capsules and granules for pediatric patients who were 1 year or older for the aforementioned indication.²

References

1. FDA approves selumetinib for adults with neurofibromatosis type 1 with symptomatic, inoperable plexiform neurofibromas. News release. FDA. November 19, 2025. Accessed November 19, 2025. https://tinyurl.com/y3bbf8pa
2. FDA approves selumetinib for pediatric patients 1 year of age and older with neurofibromatosis type 1 with symptomatic, inoperable plexiform neurofibromas. News release. FDA. September 10, 2025. Accessed November 19, 2025. https://tinyurl.com/bdd8yvjf