The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended approval of AstraZeneca’s LON:AZN Koselugo (selumetinib) for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in adults with neurofibromatosis type 1 (NF1). This marks a significant expansion of the drug’s reach into an underserved patient population.
The decision, announced Friday, is based on results from the KOMET trial, the largest and only placebo-controlled, double-blind global Phase III study in adults with NF1. Data presented at the American Society of Clinical Oncology’s annual meeting earlier this year and published in The Lancet showed Koselugo achieved an objective response rate (ORR) of 20 per cent compared with 5 per cent for placebo, a statistically significant improvement.
Why this drug could be a game changer
NF1 is a rare, progressive genetic disorder typically diagnosed in childhood but often continuing to cause complications into adulthood. Between 30 and 50 per cent of patients develop plexiform neurofibromas, non-malignant tumours of the nerve sheath that can grow large and cause pain, disfigurement, muscle weakness and, in some cases, impaired function of critical organs.
With no approved treatments for adults in the EU, therapeutic options have been limited to surgery, which is often not feasible given the complexity and location of these tumours.
- Anglo American and Teck merger to create copper powerhouse
- Phoenix Group delivers strong performance ahead of rebrand
- St James’s Place shares: More to come from this FTSE 100 wealth manager?
Pierre Wolkenstein, head of dermatology at Henri Mondor Hospital in Paris and European coordinator of the KOMET trial, said the CHMP’s recommendation underscored the urgent need for therapies addressing tumour progression beyond childhood. “For adults with NF1, tumour growth doesn’t stop at childhood and can rapidly progress or develop into adulthood, impacting physical, emotional and social well-being,” he said. “When approved, Koselugo could offer continuity of care across age groups, supported by established clinical experience in paediatrics.”
Koselugo is an oral, selective inhibitor of MEK1 and MEK2, enzymes that are overactive in NF1 and drive the uncontrolled growth of PN tumours. The drug has already been approved in multiple regions, including the US, EU, Japan and China, for use in children with NF1. Regulatory agencies in Japan and several other countries have also cleared its use in adults based on the KOMET findings, with further reviews under way.
Marc Dunoyer, chief executive of Alexion, AstraZeneca’s rare disease unit, described the CHMP opinion as an extension of Koselugo’s “pioneering legacy”. “This builds on more than a decade of global clinical and real-world experience, reflecting the unmatched body of evidence supporting the safety profile and efficacy of Koselugo,” he said.
Why this is important for AstraZeneca
The KOMET trial enrolled 145 adult patients across 13 countries. Participants receiving Koselugo showed not only reduced tumour size but also improvements in PN-related pain and quality of life. Safety results were consistent with the drug’s established profile in paediatric use, the company said.
Koselugo was granted orphan drug designation in the US, EU, Japan and other countries. It is co-developed and co-commercialised by AstraZeneca and Merck & Co (known as MSD outside North America), under a strategic collaboration signed in 2017.
If approved by the European Commission, Koselugo will become the first targeted therapy available for adults with NF1-associated plexiform neurofibromas in the EU, potentially reshaping treatment standards for this rare and debilitating condition.
