KOSELUGO approved in Canada for pediatric patients with neurofibromatosis type 1 (NF1) plexiform neurofibromas (PN)

2023-04-03

临床结果上市批准孤儿药临床2期

~First and only therapy approved to treat this rare and debilitating genetic condition~ TORONTO, April 3, 2023 /CNW/ - Alexion, AstraZeneca Rare Disease, today announced that Health Canada has granted a Notice of Compliance (NOC) for KOSELUGO (selumetinib) for the treatment of pediatric patients aged two years and above, with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN).1 "The approval of KOSELUGO has the potential to change the treatment paradigm for children with NF1 PN," said Dr. Sébastien Perreault, Neuro-Oncologist at CHU Sainte-Justine. "This is a debilitating condition that significantly affects young children. With KOSELUGO, physicians and patients now have an approved treatment option that has demonstrated a clinically meaningful benefit in reducing tumour volume." NF1 is a debilitating genetic condition affecting one in 3,000 individuals worldwide.2 In 30-50% of people with NF1, tumours develop on the nerve sheaths (plexiform neurofibromas) and can cause clinical issues such as disfigurement, motor dysfunction, pain, airway dysfunction, visual impairment and bladder or bowel dysfunction.3,4,5,6 The authorization of KOSELUGO represents an important step forward as the first and only therapy approved by Health Canada for the treatment of NF1 PN. "There is significant unmet need for children living with neurofibromatosis with plexiform neurofibromas, who until now, had no approved treatment option for their condition," said Véronique Maheux, President, Quebec Neurofibromatosis Association (ANFQ) and parent of a child with NF1. "The authorization and availability in Québec of a new treatment is an important milestone, and we hope that reimbursement decision-makers will move swiftly to provide access to children who may benefit from it." Health Canada's authorization of KOSELUGO was based on positive results from the National Cancer Institute (NCI) Cancer Therapy Evaluation Program (CTEP)-sponsored Phase II SPRINT Stratum 1 trial coordinated by the NCI's Center for Cancer Research, Pediatric Oncology Branch.1 Results of the SPRINT Phase II Stratum 1 trial showed an objective response rate (ORR) of 66% (33 of 50 patients, confirmed partial response) in pediatric patients with NF1 PN when treated with KOSELUGO as a twice-daily oral monotherapy. ORR was defined as the percentage of patients with complete response or confirmed partial response of at least 20% reduction in tumour volume.1 "KOSELUGO is the first therapy in Canada to be approved specifically for children with NF1 PN," said Gaby Bourbara, Alexion Canada VP & General Manager. "At Alexion we recognize the difficulties faced by children living with NF1 and we are proud to be able to bring a new therapeutic option to patients in Canada for this rare and life-altering disease." About NF1 NF1 is a debilitating genetic condition that is caused by a spontaneous or inherited mutation in the NF1 gene.7 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 (plexiform neurofibromas).3,8 These plexiform neurofibromas (PNs) can cause clinical issues such as disfigurement, motor dysfunction, pain, airway dysfunction, visual impairment, and bladder/bowel dysfunction.3-6 PNs begin during early childhood, with varying degrees of severity, and can reduce life expectancy by up to 15 years.3,4,7,9 About the SPRINT Trial The SPRINT Phase I/II trial was designed to evaluate the objective response rate and impact on patient-reported and functional outcomes in pediatric patients with NF1-related inoperable, symptomatic PNs treated with KOSELUGO monotherapy.1 Results were published in The New England Journal of Medicine.5 This trial sponsored by NCI CTEP was conducted under a Cooperative Research and Development Agreement between NCI and AstraZeneca with additional support from the Neurofibromatosis Therapeutic Acceleration Program (NTAP). About KOSELUGO KOSELUGO (selumetinib) is the first and only approved therapy by Health Canada for the treatment of symptomatic, inoperable plexiform neurofibromas (PNs) in pediatric patients with neurofibromatosis type 1 (NF1) aged two years and above.1 KOSELUGO blocks specific enzymes (MEK1 and MEK2) which are involved in stimulating cells to grow.1 In NF1, these enzymes are overactive, causing cells to grow in an unregulated way resulting in tumours. By blocking these enzymes, KOSELUGO slows down the growth of tumour cells.1 KOSELUGO is approved for use in the US, EU and Japan and has received Orphan Drug Designation in Russia, Switzerland, South Korea, Taiwan and Australia with health authorities worldwide reviewing regulatory submissions. About 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 30 years, Alexion is focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and commercialization 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. About AstraZeneca Canada AstraZeneca is a global, innovation-driven biopharmaceutical business with a focus on the discovery, development, and commercialization of medicines that transform lives. Our core scientific focus is in the areas of Cardiovascular, Renal and Metabolic (CVRM) disease; Oncology; Rare Disease; Respiratory & Immunology; and Vaccine & Immune Therapies. AstraZeneca operates in more than 100 countries and its innovative medicines are used by millions of patients worldwide. In Canada, the company employs more than 1,200 people across Canada, including roughly 700 employees at our head office and clinical research hub in Mississauga, Ontario. For more information, please visit the company's website at . REFERENCES _________________________ SOURCE Alexion AstraZeneca Rare Disease