针对肺癌、亨廷顿病和阿尔茨海默病的在研疗法通过创新许可与准入 pathway 获得创新通行证
正文 · 中文译文
新闻稿 用于肺癌、亨廷顿病和阿尔茨海默病的在研疗法通过创新许可与准入路径获得创新护照 药物和医疗保健产品监管局（MHRA）今日宣布，三种有望满足肺癌、亨廷顿病和阿尔茨海默病领域未被满足需求的创新药物已获得创新护照。 来源： 药物和医疗保健产品监管局 发布时间： 2026年11月9日 这意味着它们可以通过英国的创新许可与准入路径（ILAP）在整个开发过程中获得协调支持。这是全球唯一一条端到端准入路径，开发者可以从临床开发早期阶段起，与国家卫生系统、监管机构和卫生技术评估机构合作。 ILAP旨在通过提供单一 integrated 协作平台，降低产品开发风险，并加速患者获得变革性新药和药物-器械组合产品。 经ILAP遴选小组评估后，创新护照已授予： 一种化学小干扰RNA（siRNA，可通过切割mRNA沉默基因），用于治疗亨廷顿病 一种化学小分子肿瘤疗法，预计以一线非小细胞肺癌（NSCLC）为主要适应症。 一种化学反义寡核苷酸（ASO），用于治疗早期阿尔茨海默病 亨廷顿病是一种罕见的遗传性、最终致命的神经退行性疾病，给患者及其家庭带来沉重负担。 非小细胞肺癌（NSCLC）是最常见的肺癌类型，约占所有病例的80–85%。英国每年约有50,200人被诊断为肺癌。 阿尔茨海默病是一种进行性神经退行性疾病，也是痴呆最常见的病因。据估计，2024年英国约有982,000人患有痴呆，且这一数字预计还将上升。 英国药品和健康产品管理局首席医疗和科学官Jacob George教授表示： 创新许可与准入路径是一项全球独一无二的倡议，旨在加速患者获得具有变革性的新药及药物-器械组合产品。 今天，我们已向三种疗法颁发了创新护照，这些疗法在应对阿尔茨海默病、肺癌和亨廷顿病方面展现出真正的前景，有望满足尚未满足的需求。 NICE药物评估副主任John Spoors表示： 我们欢迎这些药物通过创新许可与准入路径获得创新护照的公告。NICE欢迎有机会通过ILAP与开发者和整个卫生系统的合作伙伴合作，支持证据生成，并就未来评估和患者准入相关议题进行早期沟通。 苏格兰药物联盟主席Rob Peel博士表示： 我们很高兴支持创新许可与准入路径（Innovative Licensing and Access Pathway），认可针对亨廷顿病、阿尔茨海默病和肺癌的有前景的新疗法。这些疾病持续给患者、家庭和医疗系统带来沉重负担，看到创新被引导至存在重要未满足需求的领域令人鼓舞。 AWTTC项目主任Andrew Champion博士表示： 为这三种在研疗法授予创新通行证（Innovation Passport）是朝着解决肺癌、亨廷顿病和阿尔茨海默病未满足需求迈出的令人鼓舞的一步。AWTTC很高兴与我们的英国合作伙伴携手，支持其开发与评估，将患者和威尔士国民医疗服务体系（NHS Wales）的需求纳入这一合作过程。我们的共同目标是帮助有前景的药物惠及威尔士及全英国的患者，并以安全性、有效性和价值的可靠证据为支撑。 Alnylam首席执行官Yvonne Greenstreet医学博士表示： 亨廷顿病是一种毁灭性疾病，目前尚无获批疗法可延缓其进展，凸显了对新治疗方案的迫切需求。我们很荣幸我们的在研RNAi疗法brezosiran获得了创新通行证，并欢迎在推进其开发过程中与英国药品和健康产品管理局（MHRA）及ILAP合作伙伴密切合作的机会。我们期待通过这一路径开展合作，高效推进brezosiran，希望切实改善亨廷顿病患者的生活。 James Harris，BeOne Medicines UK & Ireland Ltd.临时总经理表示： 尽管非小细胞肺癌的治疗已取得进展，但仍存在显著未满足的需求，尤其是MTAP缺陷型肿瘤患者，目前尚无靶向疗法可用。BGB-58067获得创新通行证（Innovation Passport），标志着近几个月来BeOne第二款研究性药物进入英国创新许可与准入路径（Innovative Licensing and Access Pathway），体现了我们肿瘤管线的实力，以及我们致力于将科学创新转化为迫切需要的患者潜在新治疗选择的承诺。 我们欢迎有机会与MHRA及ILAP合作伙伴密切合作，推进这款研究性PRMT5抑制剂的开发及其为非小细胞肺癌患者带来创新新治疗选择的潜力。 Biogen UK & Ireland董事总经理Kylie Bromley博士表示： 阿尔茨海默病仍然是我们这个时代最大的健康和社会照护挑战之一，我们欢迎MHRA对diranersen的创新通行证认定。我们现在有机会与整个卫生系统的合作伙伴协作，确保创新成果惠及需要它们的患者。这一成果增强了我们对阿尔茨海默病创新未来的乐观信心。 获得创新通行证的产品仍处于开发阶段，任何未来的可及性将取决于安全性、有效性和价值方面的证据。 鼓励开发创新药物以满足所有治疗领域未满足患者需求的公司查阅资格标准并提交申请。完整指南可在MHRA网站上获取。 下一轮创新通行证的申请于今日（11月9日）开放，并将持续开放至2027年2月11日。 致编辑的说明： ILAP由药品和医疗保健产品监管局（MHRA）、英格兰NHS、英国卫生技术评估（HTA）机构——国家卫生与临床优化研究所（NICE）、苏格兰药品联盟（SMC）、全威尔士治疗与毒理学中心（AWTTC）以及北爱尔兰卫生部合作实施。 ILAP面向全球商业和非商业开发机构开放。申请者必须提交尚未进入确证性试验的药品或药物-器械组合，这将提供更多机会从该路径内提供的支持中获益。更多详情可在MHRA网站上查阅。 ILAP是一项全英国范围的计划，由以下合作伙伴组成： 全威尔士治疗与毒理学中心（AWTTC）（代表威尔士NHS卫生委员会和信托机构） 药品和医疗保健产品监管局（MHRA） 国家卫生与临床优化研究所（NICE） 英格兰NHS 苏格兰药品联盟（SMC）（代表苏格兰NHS委员会） 北爱尔兰卫生部 支持合作伙伴包括： 卫生与社会保障部（DHSC） 国家卫生与护理研究所（NIHR） 生命科学办公室（OLS） 苏格兰政府 威尔士政府 近期发表的文章，提供关于创新许可与准入途径（ILAP）目标开发概况的获益信息，以及ILAP与MHRA–NICE协同路径如何协同运作。 药品和保健产品监管局（MHRA）负责监管英国所有药品和医疗器械，通过确保其有效且具有可接受的安全性。我们的所有工作均以稳健且基于事实的判断为基础，以确保获益证明任何风险是合理的。 MHRA是卫生与社会护理部的执行机构。 媒体查询，请联系newscentre@mhra.gov.uk，或致电020 3080 7651。
正文 · 英文原文
Press release Investigational treatments for lung cancer, Huntington’s disease and Alzheimer's gain Innovation Passports through the Innovative Licensing and Access Pathway Three innovative medicines with the potential to meet unmet needs in lung cancer, Huntington’s disease and Alzheimer’s disease have been given Innovation Passports, the Medicines and Healthcare products Regulatory Agency (MHRA) announced today. From: Medicines and Healthcare products Regulatory Agency Published: 9 November 2026 This means they can access coordinated support throughout development through the UK’s Innovative Licensing and Access Pathway (ILAP). This is the only end-to-end access pathway in the world where developers can work, from early stages of clinical development, with the national health system, the regulator and health technology assessment bodies. The ILAP is designed to de-risk product development and accelerate patient access to transformative new medicines and drug-device combinations, by providing a single integrated platform for collaboration. Following assessment by the ILAP Selection Panel, Innovation Passports have been awarded to: A chemical siRNA (small interfering RNA that can silence genes by cleaving mRNA) for the treatment of Huntington’s disease A chemical small molecule oncology therapy, with first-line Non-Small Cell Lung Cancer (NSCLC) expected to be the lead indication. A chemical Antisense Oligonucleotide (ASO) for the treatment of early Alzheimer’s disease Huntington’s disease is a rare, inherited and ultimately fatal neurodegenerative disease that places substantial burden on those affected and their families. Non-small cell lung cancer (NSCLC) is the most common type of lung cancer, accounting for around 80–85% of cases. Around 50,200 people are diagnosed with lung cancer in the UK each year. Alzheimer’s disease is a progressive neurodegenerative disorder and the most common cause of dementia. An estimated 982,000 people were living with dementia in the UK in 2024, with the number expected to rise. Professor Jacob George, Chief Medical and Scientific Officer of the Medicines and Healthcare products Regulatory Agency said: The Innovative Licensing and Access Pathway is a globally unique initiative designed to accelerate patient access to transformative new medicines and drug-device combinations. Today, we have issued Innovation Passports to three therapies that show real promise in addressing the unmet needs in Alzheimer’s, lung cancer and Huntington’s disease. John Spoors, Deputy Director of Medicines Evaluation at NICE said: We welcome the announcement that these medicines have been granted Innovation Passports through the Innovative Licensing and Access Pathway. NICE welcomes the opportunity, through ILAP, to work with developers and partners across the health system to support evidence generation and early engagement on the issues that will shape future evaluation and patient access. Dr Rob Peel, chair of the Scottish Medicines Consortium, said: We are pleased to support the Innovative Licensing and Access Pathway in recognising promising new therapies for Huntington’s disease, Alzheimer’s disease and lung cancer. These conditions continue to place a substantial burden on patients, families and healthcare systems, and it is encouraging to see innovation being directed towards areas of important unmet need. Dr Andrew Champion, AWTTC Programme Director, said: The award of Innovation Passports to these three investigational treatments is an encouraging step towards addressing unmet needs in lung cancer, Huntington’s disease and Alzheimer’s disease. AWTTC is pleased to work alongside our UK partners to support their development and assessment, bringing the needs of patients and NHS Wales into this collaborative process. Our shared ambition is to help promising medicines reach patients in Wales and across the UK, supported by robust evidence of safety, effectiveness and value. Yvonne Greenstreet, M.D., Chief Executive Officer of Alnylam, said: Huntington’s disease is a devastating condition with no therapies approved to slow its progression, underscoring the urgent need for new treatment options. We are honored that our investigational RNAi therapeutic brezosiran has received an Innovation Passport and welcome the opportunity to work closely with the MHRA and ILAP partners as we advance its development. We look forward to collaborating through this pathway to advance brezosiran efficiently, with the hope of meaningfully improving the lives of people living with Huntington’s disease. James Harris, Interim General Manager, BeOne Medicines UK & Ireland Ltd., said: Despite advances in the treatment of non-small cell lung cancer, significant unmet needs remain, particularly for patients with MTAP-deficient tumours, for whom no targeted therapies are currently available. The award of an Innovation Passport for BGB-58067 marks the second BeOne investigational medicine to enter the UK’s Innovative Licensing and Access Pathway in recent months, reflecting the strength of our oncology pipeline and our commitment to translating scientific innovation into potential new treatment options for patients who urgently need them. We welcome the opportunity to work closely with the MHRA and ILAP partners as we advance the development of this investigational PRMT5 inhibitor and its potential to bring an innovative new treatment option to patients with non-small cell lung cancer. Dr Kylie Bromley, Managing Director, Biogen UK & Ireland said: Alzheimer’s disease remains one of the greatest health and social care challenges of our time and we welcome the MHRA’s Innovation Passport designation for diranersen. We now have an opportunity to collaborate with partners across the health system to ensure that innovations reach the patients who need them. This achievement reinforces our optimism for the future of innovation in Alzheimer’s disease. The products awarded an Innovation Passport are still in development, and any future availability will depend on the evidence of safety, effectiveness and value. Companies developing innovative medicines that address unmet patient needs across all therapeutic areas are encouraged to review the eligibility criteria and submit an application. Full guidance is available on the MHRA website. Applications for the next round of Innovation Passports open today (9 November) and remain open until 11 February 2027. Notes to editors: The ILAP is delivered through a partnership between the Medicines and Healthcare products Regulatory Agency (MHRA), NHS England, the UK Health Technology Assessment (HTA) bodies – the National Institute for Health and Care Excellence (NICE), the Scottish Medicines Consortium (SMC) the All Wales Therapeutics and Toxicology Centre (AWTTC), and the Department of Health Northern Ireland. Entry to ILAP is open to commercial and non-commercial developers worldwide. Applicants must submit medicines or drug-device combinations that have not yet entered their confirmatory trial, which will give more opportunity to benefit from the support offered within the pathway. More details can be found on the MHRA website. The ILAP is a UK-wide initiative, comprising the following partners: The All Wales Therapeutics and Toxicology Centre (AWTTC) (on behalf of NHS Health boards and Trusts in Wales) The Medicines and Healthcare products Regulatory Agency (MHRA) National Institute for Health and Care Excellence (NICE) NHS England Scottish Medicines Consortium (SMC) (on behalf of NHS boards in Scotland) Department of Health Northern Ireland Supporting partners include: Department of Health and Social Care (DHSC) National Institute for Health Research (NIHR) Office for Life Sciences (OLS) Scottish Government Welsh Government Recently published articles that provide information on the benefit of the ILAP Target Development Profile and how the ILAP and the MHRA–NICE Aligned Pathway work together. The Medicines and Healthcare products Regulatory Agency (MHRA) is responsible for regulating all medicines and medical devices in the UK by ensuring they work and are acceptably safe. All our work is underpinned by robust and fact-based judgements to ensure that the benefits justify any risks. The MHRA is an executive agency of the Department of Health and Social Care. For media enquiries, please contact the newscentre@mhra.gov.uk, or call on 020 3080 7651.