FDA Approves Selumetinib for Adults With Neurofibromatosis Type 1

The FDA today approved selumetinib (Koselugo; Alexion, AstraZeneca Rare Disease) for the treatment of adults with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN), AstraZeneca announced in a press release.¹

The approval was based on positive results from the randomized, placebo-controlled, phase 3 KOMET trial (NCT04924608), which evaluated the safety and efficacy of selumetinib in adults with NF1 and symptomatic, inoperable PN. Patients who received selumetinib showed a 20% (95% CI, 11.2%-30.9%) objective response rate by cycle 16 vs 5% (95% CI, 1.5%-13.3%) in the placebo group (P = .011).²

Baseline chronic pain intensity scores also improved in the selumetinib group compared with the placebo group. Participants with baseline chronic pain scores of at least 3 experienced greater reductions in score by cycle 12 (least-squares mean [SE], −2.0 [0.30]; 95% CI, −2.6 to −1.4) vs patients in the placebo group (−1.3 [0.29]; 95% CI, −1.8 to −0.7), but this difference did not reach statistical significance (P = .070).

Regarding safety, adverse events (AEs) in the KOMET study were consistent with known AEs with selumetinib in pediatric patients.

“The KOMET phase 3 trial, which builds on the established clinical profile of [selumetinib] and its real-world use in pediatric patients, underscores its potential to address the substantial and oftentimes progressive clinical burdens associated with PN in adulthood," KOMET trial investigator Pierre Wolkenstein, MD, PhD, head of the Department of Dermatology at Henri Mondor Hospital, APHP, Paris East University, said in a statement.¹ "This approval reaffirms the role of [selumetinib] as a strong option for the treatment of adult and pediatric patients with NF1 PN.”

Notably, KOMET is the first international, randomized, placebo-controlled trial in adults with NF1-plexiform neurofibromas.²

Selumetinib was approved in September 2025 for pediatric patients as young as 1 year old with NF1 and symptomatic, inoperable PN, adding to its initial 2020 approval for pediatric patients aged 2 years and older with the condition.³

“We celebrate this FDA approval of [selumetinib] for adults with NF1 plexiform neurofibromas—a major step forward for NF patients everywhere," Annette Bakker, PhD, CEO of the Children’s Tumor Foundation, said.¹ "[Selumetinib] has already changed what is possible for children with NF1, and now adults will benefit from that same progress. It is proof that NF research is delivering real results and opening the door to even more treatment options. This milestone shows what can be achieved when scientists, clinicians, industry, and the NF community work together with one focus: getting effective treatments to patients faster.”

References

1. Koselugo approved in the US for adults with neurofibromatosis type 1. News release. AstraZeneca. November 20, 2025. Accessed November 20, 2025. https://www.astrazeneca.com/media-centre/press-releases/2025/koselugo-selumetinib-approved-in-the-us.html

2. Chen AP, Coyne GO, Wolters PL, et al. KOMET: a phase 3, multicentre, international, randomised, placebo-controlled study to assess the efficacy and safety of selumetinib in adults with neurofibromatosis type 1 and symptomatic, inoperable plexiform neurofibromas. Lancet. 2025;405(10496):2217-2230. doi:10.1016/S0140-6736(25)00986-9

3. Steinzor P. FDA approves selumetinib for children with NF1 and inoperable tumors. AJMC^®. September 10, 2025. Accessed November 20, 2025. https://www.ajmc.com/view/fda-approves-selumetinib-for-children-with-nf1-and-inoperable-tumors