Alexion, AstraZeneca Rare Disease, announced that the European Commission has approved Koselugo (selumetinib) for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in adult patients with neurofibromatosis type 1 (NF1). This marks the first EU approval of a targeted therapy for adults living with this rare genetic disorder.
The decision follows a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) and is based on findings from KOMET, the largest and only placebo-controlled Phase III trial in this patient population. Results from the study, presented at the 2025 ASCO Annual Meeting and published in The Lancet, demonstrated significant tumour reduction and a favorable safety profile consistent with prior use in paediatric patients.
NF1 is a rare, lifelong condition that often begins in childhood and can affect multiple organ systems. Up to half of patients develop plexiform neurofibromas—non-cancerous tumours that may cause pain, disfigurement, and muscle weakness.
“The approval of Koselugo for adults with NF1 PN in Europe offers a meaningful new option for patients beyond childhood,” said Prof. Pierre Wolkenstein, Head of Dermatology at Henri Mondor Hospital, Paris East University. “In the KOMET trial, Koselugo achieved significant tumour shrinkage with a consistent safety profile, validating its clinical benefit in adults.”
In the KOMET study, Koselugo achieved a 20% objective response rate compared with 5% for placebo by cycle 16. The treatment was well tolerated, and safety outcomes mirrored previous data from paediatric use.
Marc Dunoyer, CEO of Alexion, stated, “This approval extends the life-changing potential of Koselugo to adults with NF1 PN, ensuring continuity of care into adulthood. It reinforces our commitment to meeting the unmet needs of people living with rare diseases.”
Koselugo has also received recent approvals in Japan and other countries for adult NF1 PN, with additional regulatory reviews underway globally.