AMSTERDAM, April 15, 2024 /PRNewswire/ -- Norgine B.V. today announced its first marketing authorisation application submissions on 10 April 2024, seeking approval for eflornithine in high-risk neuroblastoma (HRNB), via Project Orbis in Australia, Switzerland and the United Kingdom. This milestone supports Norgine's efforts to deliver patient access to eflornithine and bring a further treatment option in the field of paediatric oncology..

阿姆斯特丹，2024年4月15日/PRNewswire/--Norgine B.V.今天宣布了其于2024年4月10日提交的第一份上市许可申请，通过澳大利亚、瑞士和英国的Orbis项目，寻求批准依氟鸟氨酸治疗高危神经母细胞瘤（HRNB）。这一里程碑支持了Norgine为患者提供依氟鸟氨酸的努力，并为儿科肿瘤学领域带来了进一步的治疗选择。。

Norgine and USWM, LLC (dba US WorldMeds), a Kentucky-based specialty pharmaceutical company, have an exclusive licensing agreement by which Norgine will register and commercialise eflornithine, also referred to as DFMO, in Europe, Australia and New Zealand.

Norgine和总部位于肯塔基州的专业制药公司USWM，LLC（dba US WorldMeds）签订了独家许可协议，根据该协议，Norgine将在欧洲、澳大利亚和新西兰注册和商业化依氟尼汀（也称为DFMO）。

On 13 December 2023, the US Food and Drug Administration (FDA) approved eflornithine as the first oral maintenance therapy for high-risk neuroblastoma (HRNB), indicated to reduce the risk of relapse in adult and paediatric patients who have received certain prior therapies.1 The approval decision was based on findings from a trial comparing outcomes from Study 3b (NCT02395666; investigational arm)2,4 and Study ANBL0032 (NCT00026312; clinical-trial-derived external control arm)3,4, where use of eflornithine resulted in improved event-free survival and overall survival when compared to outcomes for  patients with high-risk neuroblastoma treated with the standard of care (SoC) without the drug.1.

2023年12月13日，美国食品和药物管理局（FDA）批准依氟鸟氨酸作为高危神经母细胞瘤（HRNB）的第一种口服维持疗法，表明可以降低接受过某些先前治疗的成人和儿科患者的复发风险。1批准决定是基于一项试验的结果，该试验比较了研究3b（NCT02395666；研究组）2,4和研究ANBL0032（NCT00026312；临床试验衍生的外部对照组）3,4的结果，与标准治疗的高危神经母细胞瘤患者的结果相比，使用依氟鸟氨酸可提高无事件生存率和总生存率没有药物的护理（SoC）。

Dr David Gillen, Chief Medical Officer at Norgine, added, 'These submissions via Project Orbis represent an important first step in the regulatory process for eflornithine and re-emphasise Norgine's passion and commitment in attempting to secure additional treatment options for patients living with HRNB, a condition with a high level of unmet medical need.' .

Norgine首席医疗官David Gillen博士补充说，“通过Orbis项目提交的这些文件是依氟鸟氨酸监管过程中重要的第一步，并再次强调了Norgine的热情和承诺，即试图为患有HRNB的患者提供额外的治疗选择，HRNB是一种医疗需求高度未得到满足的疾病。”。

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Notes to Editors:

编辑注意事项：

Project Orbis

与奥比斯眼科飞行医院

Project Orbis is an initiative (since May 2019) of the US FDA Oncology Center of Excellence (OCE) and provides a framework for concurrent submission and collaborative review of innovative oncology products among international regulatory authorities. It was created with the overarching goal to speed worldwide patient access to innovative cancer therapies.

Orbis项目是美国FDA肿瘤学卓越中心（OCE）的一项举措（自2019年5月以来），为国际监管机构同时提交和合作审查创新肿瘤学产品提供了一个框架。它的首要目标是加速全球患者获得创新癌症疗法。

Project Orbis is coordinated by the FDA, and its partners include United Kingdom Medicines and Healthcare Products regulatory Agency (UK MHRA), Australia Therapeutic Goods Administration (TGA), Canada (Health Canada), Singapore (Health Sciences Authority (HSA), Switzerland (Swissmedic), Brazil (Agência Nacional de Vigilância Sanitária (ANVISA), Israel (Ministry of Health)..

Orbis项目由FDA协调，其合作伙伴包括英国药品和保健品管理局（UK MHRA）、澳大利亚治疗用品管理局（TGA）、加拿大（加拿大卫生部）、新加坡（卫生科学局（HSA）、瑞士（Swissmedic）、巴西（Agência National de Vigilência Santiária）（ANVISA）、以色列（卫生部）。。

HRNB background

HRNB背景

Children diagnosed with HRNB undergo an intense SoC regimen that still leaves them vulnerable to relapse and death, a risk that is particularly acute during the first two years.5 Approximately 30% of patients who attain remission following upfront therapy will relapse, resulting in a poor prognosis and low likelihood of long-term survival (e.g.

被诊断患有HRNB的儿童接受了强烈的SoC方案，仍然容易复发和死亡，这种风险在头两年特别严重[5]。大约30%的前期治疗后缓解的患者会复发，导致预后不良和长期生存的可能性低（例如。

estimates as low as 15% of patients will live for five years after relapsing).6 Avoiding relapse is key to long-term survival, yet outside of the United States there are no approved therapies for sustaining remission following SoC treatment. The data with eflornithine demonstrate using it as maintenance therapy extends remission and reduces risk of relapse in patients with HRNB..

估计低至15%的患者在复发后将存活五年）[6]。避免复发是长期生存的关键，但在美国以外，SoC治疗后维持缓解的治疗方法尚未获得批准。依氟鸟氨酸的数据表明，将其用作维持治疗可延长HRNB患者的缓解期并降低复发风险。。

About Norgine

关于诺金

Norgine is a leading European specialist pharmaceutical company that has been bringing transformative medicines to patients for over a century. Our commitment to transforming people's lives drives everything we do, and our European experience, fully integrated infrastructure and exceptional partnership approach enable us to quickly apply creative solutions to bring life-changing medicines to patients that they may not otherwise be able to access.

Norgine是欧洲领先的专业制药公司，一个多世纪以来一直在为患者提供变革性药物。我们致力于改变人们的生活，这推动了我们所做的一切，而我们在欧洲的经验、全面整合的基础设施和卓越的伙伴关系方法使我们能够快速应用创造性的解决方案，为患者带来改变生活的药物，否则他们可能无法获得这些药物。

Norgine is proud to have helped more than 25 million patients around the world in 2022 and generated €530 million in net product sales, a growth of nearly 5% over 2021..

诺金（Norgine）为2022年在全球帮助了2500多万患者而感到骄傲，产品净销售额为5.3亿欧元，比2021年增长了近5%。。

Norgine has a direct presence in 16 European countries, as well as Australia and New Zealand. We also have a strong global network of partnerships in non-Norgine markets. We are a flexible and fully integrated pharmaceutical business, with manufacturing (Hengoed, Wales and Dreux, France), third party supply networks and significant product development capabilities, in addition to our sales and marketing infrastructure.

Norgine在16个欧洲国家以及澳大利亚和新西兰有直接业务。我们还在非挪威市场拥有强大的全球合作网络。我们是一家灵活且全面整合的制药企业，除了销售和营销基础设施外，还拥有制造业（威尔士亨格德和法国德鲁）、第三方供应网络和重要的产品开发能力。

This enables us to acquire, develop and commercialise specialist and innovative products that make a real difference to the lives of patients around the world..

这使我们能够获得、开发和商业化专业和创新产品，从而真正改变世界各地患者的生活。。

NORGINE and the sail logo are trademarks of the Norgine group of companies.

NORGINE和sail标志是NORGINE集团公司的商标。

References

参考文献

FDA approves eflornithine for adult and pediatric patients with high-risk neuroblastoma. FDA. News release. 13 December 2023. Available from: FDA approves eflornithine for adult and pediatric patients with high-risk neuroblastoma | FDA

FDA批准依氟鸟氨酸用于成人和儿科高危神经母细胞瘤患者。FDA。新闻稿。2023年12月13日。可从以下网站获得：FDA批准依氟鸟氨酸用于成人和儿科高危神经母细胞瘤患者| FDA

Clinical Trial NCT02395666; Preventative trial of difluoromethylornithine (DFMO) in high risk patients with neuroblastoma that is in remission. Available from: Study Details | Preventative Trial of Difluoromethylornithine (DFMO) in High Risk Patients With Neuroblastoma That is in Remission | ClinicalTrials.gov..

临床试验NCT02395666；二氟甲基鸟氨酸（DFMO）在缓解期神经母细胞瘤高危患者中的预防性试验。可从以下网站获得：研究细节|缓解期神经母细胞瘤高危患者二氟甲基鸟氨酸（DFMO）的预防性试验| ClinicalTrials.gov。。

Clinical Trial NCT00026312; Isotretinoin with or without dinutuximab, aldesleukin, and sargramostim following stem cell transplant in treating patients with neuroblastoma. Available from: Study Details | Isotretinoin With or Without Dinutuximab, Aldesleukin, and Sargramostim Following Stem Cell Transplant in Treating Patients With Neuroblastoma | ClinicalTrials.gov.

临床试验NCT00026312；干细胞移植治疗神经母细胞瘤患者后，异维A酸联合或不联合丁妥昔单抗、白细胞介素和沙格司亭。可从以下网站获得：研究详细信息|异维A酸联合或不联合丁妥昔单抗，Aldesleukin和Sargramostim干细胞移植治疗神经母细胞瘤患者| ClinicalTrials.gov。

Oesterheld J, Ferguson W, Kraveka JM, Bergendahl G, Clinch T, Lorenzi E et al. Eflornithine as Postimmunotherapy Maintenance in High-Risk Neuroblastoma: Externally Controlled, Propensity Score-Matched Survival Outcome Comparisons. 2024; 42 (1): 90-102.

Oesterhold J，Ferguson W，Kraveka JM，Bergendahl G，Clinch T，Lorenzi E等人。依氟鸟氨酸作为高危神经母细胞瘤的免疫治疗后维持：外部控制，倾向评分匹配的生存结果比较。2024年；42（1）：90-102。

Basta NO, Halliday GC, Makin G, Birch J, Feltbower R, Bown N, et al. Factors associated with recurrence and survival length following relapse in patients with neuroblastoma. Br J Cancer. 2016;115:1048-57.

Basta NO，Halliday GC，Makin G，Birch J，Feltbower R，Bown N等。与神经母细胞瘤患者复发和复发后生存期相关的因素。Br J癌症。2016年；115:1048年至1047年。

London WB, Bagatell R, Weigel BJ, Fox E, Guo D, Van Ryn C, et al. Historical time to disease progression and progression-free survival in patients with recurrent/refractory neuroblastoma treated in the modern era on Children's Oncology Group early-phase trials. Cancer. 2017;123:4914-23.

London WB，Bagatell R，Weigel BJ，Fox E，Guo D，Van Ryn C等。现代儿童肿瘤组早期试验中复发/难治性神经母细胞瘤患者的疾病进展历史时间和无进展生存期。癌症。2017年；123:4914-23。

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