European Commission Authorises AstraZeneca’s Koselugo for Adult Neurofibromatosis Type 1 Patients After Landmark KOMET Trial

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Photo by Anthony Devlin/Getty Images for AstraZeneca

(IN BRIEF) AstraZeneca’s Alexion has secured European Commission approval for Koselugo (selumetinib), the first targeted therapy available to adults with neurofibromatosis type 1 (NF1) and symptomatic, inoperable plexiform neurofibromas (PN). The approval is based on the KOMET Phase III trial, which showed a 20% tumour response rate compared with 5% on placebo, alongside a favourable safety profile consistent with paediatric use. Representing a major step in continuity of care for adults with NF1 PN, the approval extends Koselugo’s reach across Europe following similar authorisations in Japan and other regions. AstraZeneca and Alexion continue to lead efforts in addressing rare disease challenges through innovation and global collaboration.

(PRESS RELEASE) CAMBRIDGE, 29-Oct-2025 — /EuropaWire/ — AstraZeneca’s rare disease unit, Alexion, has achieved European Commission approval for Koselugo (selumetinib), an oral, selective MEK inhibitor designed for adults with symptomatic, inoperable plexiform neurofibromas (PN) associated with neurofibromatosis type 1 (NF1). The decision follows compelling data from the global Phase III KOMET trial — the largest and only placebo-controlled study in this patient population — which showed a 20% objective tumour response rate compared with 5% for placebo. The results were presented at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting and published in The Lancet.

NF1 is a rare, lifelong genetic disorder that often manifests in early childhood and can affect multiple organ systems. Up to 50% of individuals with NF1 develop plexiform neurofibromas, benign nerve sheath tumours that may cause disfigurement, chronic pain, and muscle weakness. Until now, treatment options for adults with NF1 PN have been scarce, creating a significant unmet need in adult care.

In the KOMET Phase III study, Koselugo demonstrated a statistically significant 20% objective response rate in reducing tumour size (n=14/71, 95% CI: 11.2, 30.9) compared with 5% in the placebo group (n=4/74, 95% CI: 1.5, 13.3; p=0.01) by cycle 16. After 12 cycles, patients receiving placebo were switched to Koselugo, while those on active treatment continued for another 12 cycles. The therapy’s safety profile remained consistent with its established use in paediatric populations, confirming its suitability for adults transitioning from childhood care.

Professor Pierre Wolkenstein, MD, PhD, Head of Dermatology at Henri Mondor Hospital, APHP, Paris East University (UPEC), and European Coordinating Investigator of the KOMET trial, commented: “The approval of Koselugo for adults with NF1 PN in Europe represents a meaningful step in closing the treatment gap beyond childhood. The KOMET trial, the most comprehensive late-stage study in this field, confirmed significant tumour reduction and a consistent safety profile, validating Koselugo’s clinical value for both newly diagnosed adults and those continuing care into adulthood.”

Marc Dunoyer, Chief Executive Officer of Alexion, added: “This European Commission approval broadens the reach of Koselugo to adult patients with NF1 PN, ensuring continuity of care into later life. It reinforces our commitment to addressing unmet needs within the rare disease community, and we are dedicated to delivering Koselugo to adults across Europe as swiftly as possible.”

Koselugo has already received approval in Japan and other countries for the same indication, with additional regulatory reviews underway globally. These milestones underline AstraZeneca and Alexion’s leadership in advancing targeted therapies for rare and complex diseases, expanding treatment horizons for NF1 PN patients worldwide.

Notes

NF1
NF1 is a rare, progressive, genetic condition that is caused by a spontaneous or inherited mutation in the NF1 gene.3,4 NF1 is associated with a variety of symptoms, including soft lumps on and under the skin (cutaneous neurofibromas) and, in up to 50% of patients, tumours called plexiform neurofibromas (PN) may develop on the nerve sheaths.4,5 These PN can cause clinical issues such as disfigurement, motor dysfunction, pain, airway dysfunction, visual impairment and bladder or bowel dysfunction.4,5

KOMET
KOMET is a global Phase III randomised, double-blind, placebo-controlled, multicentre trial designed to evaluate the efficacy and safety of Koselugo in adults with NF1 who have symptomatic, inoperable PN. The trial enrolled 145 adults from 13 countries across North America, South America, Europe, Asia and Australia, with participants’ baseline characteristics, including gender and distribution of PN, reflective of the global adult NF1 patient population. Patients were enrolled and randomised to receive Koselugo or placebo (1:1) for 12 28-day cycles. Participants were required to have diagnosis of NF1, at least one symptomatic, inoperable PN measurable by volumetric MRI analysis, chronic PN pain score documented during screening, adequate organ and marrow function and stable chronic PN pain medication use at enrolment.2,6

The primary endpoint is confirmed objective response rate (ORR) by cycle 16 as assessed by ICR. ORR is defined as the percentage of patients with confirmed complete response (disappearance of PNs) or partial response (at least 20% reduction in tumour volume). Secondary endpoints include improved PN-related pain and health-related quality of life (HRQoL) at cycle 12.2,6

After 12 cycles, patients on placebo were switched to Koselugo and patients on Koselugo remained on treatment for an additional 12 cycles. Patients who had the opportunity to complete 24 cycles of treatment have the option to participate in a long-term extension period and continue to receive Koselugo.2,6

Koselugo 

Koselugo (selumetinib) is a kinase inhibitor that blocks specific enzymes (MEK1 and MEK2), which are involved in stimulating cells to grow. In NF1, these enzymes are overactive, causing tumour cells to grow in an unregulated way creating so-called plexiform neurofibromas (PN). By blocking these enzymes, Koselugo slows down the growth of tumour cells and, therefore, the PN growth.

Koselugo is approved in the US, EU, Japan, China and other countries for the treatment of certain paediatric patients with NF1 who have symptomatic, inoperable PN.

Koselugo is approved in the EU, Japan and other countries for the treatment of adult patients with NF1 who have symptomatic, inoperable PN, and additional regulatory reviews are ongoing.

Koselugo has been granted Orphan Drug Designation in the US, EU, Japan and other countries for the treatment of NF1.

AstraZeneca and MSD Strategic Collaboration
In July 2017, AstraZeneca and Merck & Co., Inc., Rahway, NJ, US, known as MSD outside the US and Canada, announced a global strategic collaboration to co-develop and co-commercialise Lynparza (olaparib), a first-in-class PARP inhibitor, and Koselugo. The companies may develop Lynparza and Koselugo in combination with other potential new medicines and as monotherapies.

Alexion
Alexion, AstraZeneca Rare Disease, is focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and delivery of life-changing medicines. A pioneering leader in rare disease for more than three decades, Alexion was the first to translate the complex biology of the complement system into transformative medicines, and today it continues to build a diversified pipeline across disease areas with significant unmet need, using an array of innovative modalities. As part of AstraZeneca, Alexion is continually expanding its global geographic footprint to serve more rare disease patients around the world. It is headquartered in Boston, US.

AstraZeneca
AstraZeneca (LSE/STO/Nasdaq: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development, and commercialisation of prescription medicines in Oncology, Rare Diseases, and BioPharmaceuticals, including Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca’s innovative medicines are sold in more than 125 countries and used by millions of patients worldwide. Please visit astrazeneca.com and follow the Company on Social Media @AstraZeneca.

Contacts
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References

  1. Koselugo (selumetinib) SmPC; October 2025.
  2. Chen, AP, et al. KOMET: a phase 3, multicentre, international, randomised, placebo-controlled study to assess the efficacy and safety of selumetinib in adults with neurofibromatosis type 1 and symptomatic, inoperable plexiform neurofibromas. The Lancet. 2025;405(10496):2217-2230.
  3. Tamura R. Current understanding of neurofibromatosis type 1, 2, and schwannomatosis. Int J Mol Sci. 2021;22(11):5850.
  4. Hirbe AC, et al. Neurofibromatosis type 1: a multidisciplinary approach to care. Lancet Neurol. 2014;13:834-843.
  5. Bergqvist C, et al. Neurofibromatosis 1 French national guidelines based on an extensive literature review since 1966. Orphanet J Rare Dis. 2020;15(1):37.
  6. ClinicalTrials.gov. Efficacy and safety of selumetinib in adults with NF1 who have symptomatic, inoperable plexiform neurofibromas (KOMET). NCT Identifier: NCT04924608. Available here. Accessed October 2025.

SOURCE: AstraZeneca

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