Efficacy and Safety of Selumetinib in Adults With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas

Summary

A global study to demonstrate the effectiveness of selumetinib in participants with NF1 who have symptomatic, inoperable plexiform neurofibromas.

Timeline

Start

2021-11-19

Primary completion

2024-08-05

Completion

2029-02-15

Outcome

Met primary endpoint

paper Selumetinib achieved a significant objective response rate versus placebo. PMID 40473450 ↗

registry analysis (superiority test); Selumetinib 25 mg/m2 vs Placebo; p = 0.0112; Fisher Exact NCT04924608 ↗

release “met its primary endpoint, demonstrating a statistically significant and clinically meaningful objective response rate (ORR) versus placebo in these adult patients.” astrazeneca.com ↗

release “In the primary analysis of the KOMET Phase III trial, Koselugo showed a statistically significant and clinically meaningful overall response rate (ORR) of 20%” astrazeneca.com ↗

Publications

• Chen AP, Coyne GO, Wolters PL, Martin S, Farschtschi S, Blanco I, Chen Z, Darrigo LG Jr, Eoli M, Whittle JR, Nishida Y, Lamarca R, de la Rosa Rodriguez R, Adeyemi A, Herrero I, Llorente N, Diede SJ, Dombi E, Wolkenstein P; KOMET study investigators. Efficacy and safety of selumetinib in adults with neurofibromatosis type 1 and symptomatic, inoperable plexiform neurofibromas (KOMET): a multicentre, international, randomised, placebo-controlled, parallel, double-blind, phase 3 study. Lancet. 2025 Jun 21;405(10496):2217-2230. doi: 10.1016/S0140-6736(25)00986-9. Epub 2025 Jun 2.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Selumetinib	Small molecule	25 mg/m2	Oral

Indications