Alexion, an AstraZeneca rare disease, has received EU approval for Koselugo (selumetinib), its oral, selective MEK inhibitor, to treat symptomatic, inoperable plexiform neurofibromas (PN) in adult patients diagnosed with neurofibromatosis type 1 (NF1). The European Commission’s decision follows a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) and is supported by results from KOMET, the largest and only placebo-controlled global Phase III trial in this patient group.
NF1 is a rare genetic disorder that usually appears in early childhood and often carries on into adulthood, affecting several organ systems along the way. As many as half of people with the condition can develop PN, a non-cancerous tumour that forms in the brain, spinal cord, or peripheral nerves. Over time, these growths can enlarge and cause pain, disfigurement, or muscle weakness, along with other complications.
Prof. Pierre Wolkenstein, MD, PhD, Head of Dermatology at Henri Mondor Hospital, APHP, Paris East University (UPEC), and European National Coordinating Investigator of the KOMET trial, said: “The approval of Koselugo for adults with NF1 PN in Europe offers patients and physicians a meaningful approach to close treatment gaps beyond childhood. As demonstrated in the KOMET Phase III trial, the most robust late-stage clinical trial conducted in this patient group to-date, adults administered Koselugo saw significant tumour volume reduction with a safety profile consistent with its established use in paediatric patients, validating the clinical benefits of Koselugo for newly diagnosed adults and those transitioning to adult care.”
Marc Dunoyer, Chief Executive Officer at Alexion, commented: “The European Commission approval extends the life-changing potential of Koselugo to adults with NF1 PN in the region, including continuity of care into adulthood. This milestone, along with our pioneering leadership in NF1 PN treatment landscape, embodies Alexion’s unwavering commitment to addressing the unmet needs in the rare disease community. We look forward to bringing Koselugo to those adults in need across Europe as soon as possible.”
In the primary analysis of KOMET, Koselugo demonstrated a statistically significant objective response rate (ORR) of 20% (n=14/71, 95% CI: 11.2, 30.9) compared with 5% for placebo (n=4/74, 95% CI: 1.5, 13.3; p=0.01) by cycle 16. Following 12 cycles, patients receiving placebo were switched to Koselugo, while those already on Koselugo continued therapy for another 12 cycles. The drug’s safety profile in adults aligned with its established performance in paediatric use.
In addition to EU approval for Koselugo, the drug has also secured approval in Japan and several other markets for the treatment of adult NF1 patients with symptomatic, inoperable PN, based on data from the KOMET Phase III trial, with additional regulatory assessments currently underway.
