Alexion, AstraZeneca Rare Disease reaches an agreement with the pan-Canadian Pharmaceutical Alliance (pCPA) for Koselugo (selumetinib) for the treatment of paediatric patients with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN)

The agreement is a critical step in ensuring paediatric patients living with NF1 PN have public access to Koselugo, the only approved therapy in Canada for this rare disease

MISSISSAUGA, ON, Dec. 18, 2024 /CNW/ - Alexion Pharma Canada Corp., AstraZeneca's Rare Disease group, has entered into a Letter of Intent (LOI) with the pan-Canadian Pharmaceutical Alliance (pCPA) for Koselugo for the treatment of paediatric patients aged two years and above with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN). With the agreement in place with the pCPA, individual provinces and territories may now initiate the process to list Koselugo on their formularies, the timing of which will vary by province and territory. Following the agreement, the Province of Quebec was the first to list Koselugo and provide public reimbursement of the only approved therapy for eligible children living with NF1 PN.

NF1 is a rare, progressive genetic condition affecting one in 3,000 individuals worldwide, most commonly diagnosed in children under the age of 10.^1,2 In 30-50% of patients, tumours develop on the nerve sheaths (plexiform neurofibromas) and cause clinical issues such as disfigurement, motor dysfunction, pain, airway dysfunction, visual impairment and bladder or bowel dysfunction.^3-7

Véronique Maheux, President of L' Association de la Neurofibromatose du Québec (ANFQ) said: "The positive conclusion of this agreement between Alexion and pCPA is very exciting news for Canadian children and their families living with NF1 PN. Living with NF1 PN impacts day-to-day life for these children and can be very challenging for the entire family. Having access to Koselugo will be key to helping ease the painful symptoms and shrink the inoperable tumours caused by the disease. I would also like to recognize the Province of Quebec for their leadership in being the first to list Koselugo and call on the other provinces and territories to make Koselugo accessible to children living with NF1 PN as quickly as possible."

Dr. Vijay Ramaswamy, MD, PhD, FRCPC, Neuro-Oncologist at The Hospital for Sick Children said: "This agreement is an exciting development for children and families with NF1 PN in Canada. Access to selumetinib, the first and only approved treatment for children with NF1 PN, represents a major shift in the treatment paradigm and will help improve the quality of life for a significant number of Canadian children with this rare tumour."

Karen Heim, General Manager of Alexion Canada, said: "We would like to thank the pCPA for its partnership in recognizing the need for this life-changing innovation for Canadian children and adolescents living with NF1 PN. We look forward to our ongoing collaboration with provincial and territorial jurisdictions to finalize listing agreements to bring these patients and their families access to this important therapy."

Notes

NF1
NF1 is a rare, progressive, genetic condition that is caused by a spontaneous or inherited mutation in the NF1 gene.^8-9 NF1 is associated with a variety of symptoms, including soft lumps on and under the skin (cutaneous neurofibromas) and, in 30-50% of patients, tumours develop on the nerve sheaths (PNs).^3,9 These PNs can cause clinical issues such as disfigurement, motor dysfunction, pain, airway dysfunction, visual impairment and bladder or bowel dysfunction.^3-7 PNs begin during early childhood, with varying degrees of severity, and can reduce life expectancy by up to 15 years.^4,7,9,10

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 and has been granted Orphan Drug Designation in the US, EU, Japan and other countries for the treatment of certain paediatric patients with NF1 who have symptomatic, inoperable PN.

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. For more information, please visit https://alexion.com/worldwide/canada. 

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 www.astrazeneca.ca and follow the Company on social media  @AstraZeneca.

References

1.	Mindrup R, Idoate R. Neurofibromatosis and a Portrait of 1 in 3000. AMA J Ethics. 2020 Jun 1;22(6):E513-524.
2.	Mayo Clinic. Neurofibromatosis Type 1. Available at: https://www.mayoclinic.org/diseases-conditions/neurofibromatosis-type-1/symptoms-causes/syc-20350490#:~:text=Neurofibromatosis%20type%201%20(NF1)%20usually,vary%20from%20person%20to%20person. Accessed December 2024.
3.	Gross AM, et al. Selumetinib in children with inoperable plexiform neurofibromas. N Engl J Med. 2020 Apr 9;382(15):1430-1442. doi: 10.1056/NEJMoa1912735.
4.	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.
5.	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. doi: 10.1056/NEJMoa1605943.
6.	Mayo Clinic. Neurofibromatosis. Available at: https://www.mayoclinic.org/diseases-conditions/neurofibromatosis/symptoms-causes/syc-20350490. Accessed July 2022.
7.	NHS. Neurofibromatosis Type 1, Symptoms. Available at https://www.nhs.uk/conditions/neurofibromatosis-type-1/symptoms/#:~:text=The%20most%20common%20symptom%20of,child%20is%203%20years%20old. Accessed December 2024.
8.	Tamura R. Current understanding of neurofibromatosis type 1, 2, and schwannomatosis. Int J Mol Sci. 2021;22(11):5850.
9.	National Institute of Neurological Disorders and Stroke. Neurofibromatosis. Available here. Accessed November 2024.
10.	Evans DGR, et al. Reduced life expectancy seen in hereditary diseases which predispose to early-onset tumors. Appl Clin Genet. 2013;6:53-61.

SOURCE AstraZeneca

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