REG - AstraZeneca PLC - Koselugo (selumetinib) approved in the EU

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RNS Number : 0166F  AstraZeneca PLC  28 October 2025

28 October 2025

 

Koselugo approved in the EU for plexiform neurofibromas in adults with
neurofibromatosis type 1

 

Approval based on KOMET Phase III trial results which showed 20% objective
response rate in tumour size reduction

 

Alexion, AstraZeneca Rare Disease's Koselugo (selumetinib), an oral, selective
MEK inhibitor, has been approved in the European Union (EU) for the treatment
of symptomatic, inoperable plexiform neurofibromas (PN) in adult patients with
neurofibromatosis type 1 (NF1).(1)

 

The approval by the European Commission follows the positive opinion
(https://www.astrazeneca.com/media-centre/press-releases/2025/koselugo-recommended-for-eu-approval.html)
of the Committee for Medicinal Products for Human Use (CHMP) and is based on
results from KOMET, the largest and only placebo-controlled global Phase III
trial in this patient population, which were presented at the 2025 American
Society of Clinical Oncology (ASCO) Annual Meeting and published in The Lancet
(https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(25)00986-9/abstract)
.(2)

 

NF1 is a rare, progressive, genetic condition usually diagnosed in early
childhood, but often progressing into adulthood, that can impact every organ
system.(3,4) 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.(4,5)

 

Prof. Pierre Wolkenstein, MD, PhD, Head of the Department of Dermatology at
Henri Mondor Hospital, APHP, Paris East University (UPEC), and National
Coordinating Investigator of the KOMET trial in Europe, 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, Alexion, said: "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 the trial, Koselugo showed a statistically
significant objective response rate (ORR) of 20% (n=14/71, 95% CI: 11.2, 30.9)
compared to 5% with placebo (n=4/74, 95% CI: 1.5, 13.3; p=0.01) by cycle 16.
After 12 cycles, patients on placebo were switched to Koselugo and patients on
Koselugo remained on treatment for an additional 12 cycles.(2)

 

The safety profile of Koselugo in the KOMET Phase III trial was consistent
with its known profile and established use in paediatric patients.(2)

 

Koselugo has been recently approved in Japan and other countries for the
treatment of adult patients with NF1 who have symptomatic, inoperable PN based
on data from the KOMET Phase III trial, and additional regulatory reviews are
ongoing.

 

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 (http://www.astrazeneca.com/)  and follow the
Company on Social Media @AstraZeneca
(https://www.linkedin.com/company/astrazeneca/) .

 

Contacts

For details on how to contact the Investor Relations Team, please click here
(https://www.astrazeneca.com/investor-relations.html#Contacts) . For Media
contacts, click here (https://www.astrazeneca.com/media-centre/contacts.html)
.

 

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
(https://clinicaltrials.gov/study/NCT04924608) . Accessed October 2025.

 

Matthew Bowden

Company Secretary

AstraZeneca PLC

 

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