NICE recommends Koselugo for the treatment of neurofibromatosis type 1

NICE recommends Koselugo for the treatment of neurofibromatosis type 1

First medicine approved for treating tumours growing on nerve sheaths in children with rare and genetic condition will now be available in England

Uxbridge, UK, April 1st, 2022 – Alexion Pharma UK, AstraZeneca’s Rare Disease group, has announced that the National Institute for Health and Care Excellence (NICE) has recommended AstraZeneca and MSD's Koselugo®▼ (selumetinib) for use by the NHS in England, within its marketing authorisation, for treating symptomatic and inoperable plexiform neurofibromas (PN) associated with type 1 neurofibromatosis (NF1) in children aged 3 and over.1

Vanessa Martin, Chair of the Childhood Tumour Trust, said, “Children with NF1 deal with many complications of their condition. Some may develop benign tumours called PNs that can occur all over the body, sometimes close to vital organs and structures. These tumours can lead to severe morbidity, including pain and disfigurement, motor dysfunction, and in some cases, life-threatening organ impairment. Depending on their location, PNs may be extremely difficult or impossible to remove surgically, and as a result are often left untreated. Any of these factors have a serious impact on the patient’s quality of life. Being able to treat PNs medically has given hope and excitement to our community and marks a significant step forward in addressing the debilitating impact of these tumours.”

NF1 is a debilitating genetic condition affecting one in 4,000 individuals worldwide.2 In 30-50% of people with NF1, plexiform neurofibromas tumours develop on the nerve sheaths and can cause clinical issues such as disfigurement, motor dysfunction, pain, airway dysfunction, visual impairment, and bladder or bowel dysfunction.3,4

Dr. Grace Vassallo said, "For some children with NF1, PNs can grow and develop so significantly that they can become very debilitating. Until now, children with PNs have had limited treatment options and this approval is a big step forward for managing and treating this complication offering improved outcomes and quality of life for the affected children and their families."

Selumetinib blocks specific enzymes (mitogen-activated protein kinase kinases 1 and 2, also known as MEK 1 and MEK2) which are involved in stimulating cells to grow.5 In NF1, these enzymes are overactive, causing tumour cells to grow in an unregulated way. By blocking these enzymes, selumetinib slows down the growth of tumour cells.5

“We understand the difficulties caused by the symptoms of NF1 and the implications it can have on a child’s overall wellbeing, so we are extremely pleased to see NICE welcome selumetinib for the treatment of PNs in children with NF1,” said Sean Richardson, Alexion General Manager, UK & Ireland. “At Alexion, we are relentless in our ambition for innovation, and it brings us great pleasure knowing that patients in England will benefit from the approval of this new therapeutic option.”

The NICE approval of selumetinib is based on a comprehensive clinical and health economic submission by AstraZeneca and Alexion as well as evidence provided by patient advocacy organisations and clinical experts.

************************************************************************************************************
Notes

NF1

NF1 is a debilitating genetic condition that is caused by a spontaneous or inherited mutation in the NF1 gene.6 NF1 is associated with a variety of symptoms, including soft lumps on and under the skin (cutaneous neurofibromas) and skin pigmentation (so-called ‘café au lait’ spots) and, in 30-50% of patients, tumours develop on the nerve sheaths (PNs).3,7 These PNs can cause clinical issues such as disfigurement, motor dysfunction, pain, airway dysfunction, visual impairment, and bladder/bowel dysfunction.4 PNs begin during early childhood, with varying degrees of severity, and can reduce life expectancy by up to 15 years.8

Koselugo
Koselugo (selumetinib) is the first and only therapy approved by the European Commission for the treatment of symptomatic, inoperable PN in paediatric patients with NF1 aged three years and above.9

Selumetinib blocks specific enzymes (MEK1 and MEK2) which are involved in stimulating cells to grow.5 In NF1, these enzymes are overactive, causing tumour cell to grow in an unregulated way. By blocking these enzymes, selumetinib slows down the growth of tumour cells.5

Koselugo is also approved for use in the United States, has received Orphan Drug Designation in Japan, Russia, Switzerland, South Korea, Taiwan and Australia and health authorities worldwide are reviewing regulatory submissions.

Alexion
Alexion, AstraZeneca Rare Disease, is the group within AstraZeneca focused on rare diseases, created following the 2021 acquisition of Alexion Pharmaceuticals, Inc. As a leader in rare diseases for nearly 30 years, Alexion is focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development, and commercialisation of life-changing medicines. Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on haematology, nephrology, neurology, metabolic disorders, cardiology and ophthalmology. Headquartered in Boston, Massachusetts, Alexion has offices around the globe and serves patients in more than 50 countries. Please visit alexion.com/worldwide/UK.

AstraZeneca
AstraZeneca 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. AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide.
AstraZeneca is based in six different locations across the UK, with its global headquarters in Cambridge. In the UK, around 8,300 employees work in research and development, manufacturing, supply, sales, and marketing. We supply 34 different medicines to the NHS, which treat more than one million UK patients every year. For more information, please visit
www.astrazeneca.co.uk and follow us on Twitter @AstraZenecaUK.

Media Inquiries
Lilianna Husseini
lilianna.husseini-consultant@alexion.com
+33 (0) 647641720

1 NICE. Final appraisal document. Selumetinib for treating symptomatic and inoperable plexiform neurofibromas associated with type 1 neurofibromatosis in children ages 3 years and over. Available at: https://www.nice.org.uk/guidance/gid-hst10045/documents/html-content-3. Last accessed: April 2022.
2 About Neurofibromatosis. National Human Genome Research Institute. Available at: https://www.genome.gov/Genetic-Disorders/Neurofibromatosis. Last accessed: March 2022.
3 National Institute of Neurological Disorders and Stroke. Neurofibromatosis Fact Sheet. Available at: “What is NF1?”. Available at: www.ninds.nih.gov/disorders/patient-caregiver-education/fact-sheets/neurofibromatosis-fact-sheet #3162_2. Last accessed: March 2022.
4 Dombi E, Baldwin A, Marcus LJ, et al. Activity of selumetinib in neurofibromatosis type 1-related plexiform neurofibromas. N Engl J Med. 2016;375:2550-2560.
5 European Medicines Agency. Koselugo (selumetinib). Available at: https://www.ema.europa.eu/en/documents/overview/koselugo-epar-medicine-overview_en.pdf. Last accessed March 2022.
6 Rasmussen SA, Yang Q, Friedman JM. Mortality in neurofibromatosis 1: an analysis using U.S. death certificates. Am J Hum Genet. 2001;68:1110-1118. Last accessed March 2022
7 Hirbe AC, Gutmann DH. Neurofibromatosis type 1: a multidisciplinary approach to care. Lancet Neurol. 2014;13:834-43. doi: 10.1016/S1474-4422(14)70063-8. Last accessed March 2022.
8 Evans DGR, Ingham SL. Reduced Life Expectancy Seen in Hereditary Diseases Which Predispose to Early Onset Tumors. Appl Clin Genet. 2013;6:53-61.
9 Koselugo (selumetinib) [prescribing information]. Wilmington, DE: AstraZeneca Pharmaceuticals LP; 2020. Accessed March 2022.

Job Code: UK/KOS-NF1/0014
Last Updated: 03/22