[Correspondence] Clinical benefits of selumetinib in adults with neurofibromatosis type 1 – Authors' reply

Back to news list

Source: The Lancet

Original: https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(25)02248-2/fullt...

Published: 2025-12-13

The KOMET trial is the first global, randomized, placebo-controlled trial to evaluate the efficacy and safety of selumetinib in adult patients with neurofibromatosis type 1 (NF1) and symptomatic, inoperable plexiform neurofibromas[1]. Selumetinib was administered at a dose of 25 mg/m² twice daily in 28-day cycles. The results showed a significant reduction in the volume of plexiform neurofibromas already up to the 16th cycle of treatment compared to placebo[1]. In addition to shrinking tumors, there was also a reduction in chronic and acute pain, a reduction in the need for analgesics, and an improvement in patients' quality of life[1]. The study did not reveal any new safety risks associated with selumetinib treatment[1]. Selumetinib is a selective MEK1/2 kinase inhibitor that blocks pathological signals leading to the growth of neurofibromas[6]. This drug is already approved for the treatment of pediatric patients with NF1 and inoperable plexiform neurofibromas, while the KOMET study extended the evidence for its efficacy in adults[4][5]. Overall, the study confirmed the clinical benefit of selumetinib in the treatment of adults with NF1 and inoperable plexiform neurofibromas[1].