Selumetinib Shrinks NF1-Plexiform Neurofibromas in Adults, Too

CHICAGO -- Selumetinib (Koselugo) significantly improved response rates in adults with neurofibromatosis type 1 (NF1) and symptomatic, inoperable plexiform neurofibromas, the phase III KOMET trial showed.

After 16 cycles, responses occurred in 20% of patients assigned to the MEK inhibitor versus 5% of those randomized to placebo ( P =0.011), meeting the study's primary endpoint, reported Alice Chen, MD, of the National Cancer Institute in Bethesda, Maryland.

The drug led to rapid responses (median 3.7 months) and a 37% reduction in tumor volume on average among the responder population, according to findings presented at the American Society of Clinical Oncology annual meeting here and published in The Lancet .

"Selumetinib shows statistically significant and clinically meaningf