FDA OKs Selumetinib in NF1 Symptomatic, Inoperable Plexiform Neurofibromas

Results from the KOMET trial led to the approval of selumetinib in adults with 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