AstraZeneca Launches Koselugo® (Selumetinib) in China for Pediatric Patients with Symptomatic, Inoperable Plexiform Neurofibromas Associated with Neurofibromatosis Type 1
AstraZeneca
Biopharmaceutical Manufacturer
ShanghaiSeptember 1, 2023 PR Newswire -- Today, AstraZeneca's first innovative drug in the neurofibromatosis field, Koselugo®(English Product Name:Koselugo (Generic Name: Selumetinib Sulfate Capsules) has been officially launched in China, benefiting pediatric patients aged 3 years and older with symptomatic, inoperable plexiform neurofibromas (PN) associated with Neurofibromatosis Type 1 (NF1).
Neurofibromatosis (NF) is a group of autosomal dominant genetic disorders, among which NF1 accounts for approximately 98% of the three main subtypes.[1]. There is no epidemiological data of NF1 in China, and the estimated incidence rate is 5/1,000,000.[2]Among them, approximately 30%-50% of NF1 patients will develop plexiform neurofibromas (NF1-PN).[3-5]As a chronic syndrome that affects multiple systems throughout the body, NF1-PN can cause disfigurement, pain, functional impairment, and lifelong cognitive deficits.[6-10], the risk of malignant tumors and heart disease in pediatric patients is up to 13% higher than in the general population, with an average reduction in life expectancy of 15 years.[11-14]。
Before the advent of Selumetinib,The main treatment options for NF1 include surgical resection, plastic surgery, etc.[15], but 90% of NF1-PN cannot be treated surgically or completely resected, and the recurrence rate is high.[1]。
Li Qingfeng, Vice President of the Ninth People's Hospital affiliated to Shanghai Jiaotong University School of Medicine and Director of the Plastic Surgery DepartmentSaid: "Type I neurofibromatosis affects multiple systems throughout the body and can lead to pain, disability, cognitive dysfunction, and malignant transformation. We are very pleased to see that selumetinib has been officially applied in clinical practice. As the first treatment drug approved in China for the NF1 field, selumetinib is expected to help more children achieve a better quality of life."
Wang Fang, Director of Shenzhen Paopao Home Neurofibromatosis Care CenterIndicates:"As a rare, progressive genetic disorder, NF1 imposes a heavy physical and mental burden on patients and their families due to recurrent episodes and severe complications. It is hoped that with the help of innovative drugs, NF1 patients can control disease progression and return to normal life as soon as possible with their caregivers."
Leo Wang, Executive Vice President of AstraZeneca Global, President of International Business and ChinaIndicates:"We deeply understand the challenges faced by NF1 patients and the rare disease community in China, and we are very proud to bring the first drug treatment option for Chinese children with NF1, helping patients achieve a hopeful, high-quality, and dignified life. With leading scientific innovation capabilities and forward-looking efforts in the rare disease field, AstraZeneca will continue to accelerate the introduction of innovative medicines for rare diseases. Collaborating with partners, we will promote drug accessibility, support the establishment of a sound rare disease diagnosis and treatment ecosystem, benefiting rare disease patients in China."
As of now, Selumetinib has been approved in the United States, the European Union, Japan and other countries for the treatment of symptomatic, inoperable plexiform neurofibromas.Children with Type I Neurofibromatosis.
AboutType I Neurofibromatosis (NF1)
NF1 is a genetic disorder caused by NF1 gene or hereditary mutations, affecting multiple systems.[12], accompanied by various symptoms, including the formation of neurofibromas and pigmentation (café-au-lait spots) on or under the skin, etc. Among these, 30-50% of NF1 patients will develop plexiform neurofibromas.[11, 12]Plexiform neurofibromas (PN) can cause disfigurement, pain, motor and respiratory dysfunction, vision impairment, and bladder or bowel dysfunction, among others.[2,11,13,16,17]Plexiform neurofibromas often occur in early childhood, with varying degrees of severity, and may reduce life expectancy by 15 years.[11-14]。
About Selumetinib
Selumetinib is the first and only treatment approved by the National Medical Products Administration of China forDrug Therapy for Children with Type I Neurofibromatosis (NF1) Aged 3 Years and Above, Accompanied by Symptoms and Inoperable Plexiform Neurofibromas (PN)18, which can block specific enzymes (MEK1 and MEK2) involved in promoting cell growth.[18]In NF1, these enzymes are overactive, causing plexiform neurofibromas to grow in an uncontrolled manner. By blocking specific enzymes, selumetinib can slow down the progression of the condition.18Selumetinib has been approved in the United States, the European Union, Japan, and China, and has been designated as an "orphan drug" in many countries and regions around the world.
About AstraZeneca
AstraZeneca (LSE/STO/Nasdaq: AZN) is a science-led global biopharmaceutical enterprise dedicated to the research, development, manufacturing, and marketing of prescription medicines. The company focuses on key areas such as oncology, biopharmaceuticals including cardiovascular, renal, and metabolic diseases, respiratory and immunology, vaccines and infectious diseases, as well as rare diseases. AstraZeneca's global headquarters is located in Cambridge, UK, with operations spanning across the world.100In multiple countries, innovative drugs benefit millions of patients worldwide. For more information, please visitwww.astrazeneca.com。
About AstraZeneca China
AstraZenecaSince entering China in 1993, AstraZeneca has focused on the most urgent therapeutic needs of Chinese patients, including oncology, cardiovascular, renal, metabolic, respiratory, digestive, rare diseases, vaccines and antibodies, as well as autoimmune diseases, bringing nearly 40 innovative drugs to China. The headquarters of AstraZeneca China and the global R&D China Center are located in Shanghai, with global production and supply bases established in Wuxi, Taizhou, and Qingdao, delivering high-quality medicines to approximately 80 global markets. In recent years, the company has set up regional headquarters in Beijing, Guangzhou, Hangzhou, Chengdu, and Qingdao. AstraZeneca also collaborates with partners to build a diversified international innovation ecosystem for healthcare through its "three innovative drivers": the China Smart Health Innovation Center (CCiC), the International Life Science Innovation Park (iCampus), and the AstraZeneca Zhongjin Healthcare Industry Fund, collectively promoting the long-term development of regional economies and the broader healthcare industry. Today, China has become AstraZeneca's second-largest market globally.
References
[1] National Health Commission of the People's Republic of China, Diagnostic and Treatment Standards for Neurofibromatosis in Children and Adolescents (2021 Edition) [J]. 2021.
[2] Gross A M, Wolters P L, Dombi E, et al. New England Journal of Medicine, 2020, 382(15): 1430-1442. doi: 10.1056/NEJMoa1912735.
[3] Guidelines for Multidisciplinary Diagnosis and Treatment of Type I Neurofibromatosis (2023 Edition) [J], 2023.
[4] ERN GENTURIS tumour surveillance guidelines for individuals with neurofibromatosis type 1.
[5] Expert Consensus on the Clinical Diagnosis and Treatment of Neurofibromatosis Type I (2021).
[6] Buono FD, et al. J Pain Res. 2019 Aug 22;122555 2561.
[7] Bellampalli SS, et al. Pain. 2019 May;160(5)1007 1018.
[8] Hirbe AC, et al . Lancet Neurol. 2014 Aug;13(8)834 43.
[9] Blakeley JO et al. Neuro Oncol. 2016;18(5)624 638.
[10] Yang X, et al. Childs Nerv Syst. 2022 May 17.
[11] 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.
[12] 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. Last accessed: August 2023.
[13] NHS. Neurofibromatosis Type 1, Symptoms. Available at https://www.nhs.uk/conditions/neurofibromatosis-type-1/symptoms. Accessed August 2023.
[14] Evans DGR, Ingham SL. Reduced life expectancy seen in hereditary diseases which predispose to early-onset tumors. Appl Clin Genet. 2013;6:53-61.
[15] EClinicalMedicine.2023 Jan 13;56:101818. doi: 10.1016/j.eclinm.2022.101818. eCollection 2023 Feb.
[16] 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.
[17] Mayo Clinic. Neurofibromatosis. Available at: https://www.mayoclinic.org/diseases-conditions/neurofibromatosis/symptoms-causes/syc-20350490. Accessed July 2022.
[18] Koselugo (selumetinib) Chinese prescribing information; 2023.