Alexion, AstraZeneca (NYSE:AZN) Rare Disease's Koselugo (selumetinib), an oral, selective MEK inhibitor, has been approved in Canada for the treatment of adult patients with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN).
The approval by Health Canada was based on positive results from KOMET, the largest and only placebo-controlled global Phase III trial in this patient population. Data were presented at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting and published in The Lancet.2
NF1 is a rare, progressive, genetic condition usually diagnosed in early childhood, but often progressing into adulthood, that can impact every organ system.
Up to 50% of people living with NF1 may develop a type of non-malignant tumour called PN that may affect the brain, spinal cord and nerves.4,5 PN may appear later in a person's life and can grow and become large, leading to pain, disfigurement and muscle weakness, among other debilitating symptoms.
Ryan Thomas, MD, Family Physician, Scarborough Academic Family Health Centre, and Clinical Associate at the Elisabeth Raab Neurofibromatosis Clinic, University Health Network, Toronto, said: "Adults in Canada living with NF1 who have symptomatic, inoperable PN now have a meaningful treatment option beyond childhood. The approval of selumetinib addresses a significant care gap and offers new hope in this space."
AZN shares began Monday lower by $3.61, or 1.9%, to $190.61.
