FDA提供明确指引以支持细胞和基因疗法的开发与质量
正文 · 中文译文
FDA新闻稿 FDA提供明确性以支持细胞和基因疗法的开发与质量 FDA发布细胞和基因疗法生产商最终指南，建议将效价保证纳入疗法生命周期的每个阶段——从早期开发到患者给药 更多新闻公告 即时发布： 2026年11月2日 美国食品药品监督管理局（FDA）今日发布了针对细胞和基因疗法（CGT）产品生产商的最终指南，以实施一项全面的、基于科学和风险的计划，确保其产品符合效价标准。该指南提供了FDA关于效价保证策略的建议，指导生产商在产品到达患者之前进行检测和验证，确保每一批次均符合规定的效价标准，能够发挥产品预期的治疗作用。 《细胞和基因疗法产品效价保证》最终指南替代了FDA 2011年针对CGT产品的效价检测指南，适用于严重且通常危及生命的疾病的治疗。根据法律规定，生产商应验证其产品从批准之时起至批准后监测期间按预期发挥作用。该最终指南提供了FDA关于生产商如何满足该要求的当前思考和建 “接受细胞和基因疗法的患者往往正在与严重或危及生命的疾病作斗争，例如癌症和罕见病，他们应当得到保证，即施用于他们的产品将按预期发挥作用，”生物制品评价与研究中心主任、药学学士、理学硕士Karim Mikhail表示。“本指南体现了FDA的承诺，即确保美国始终是世界上最适合创新的地方——并且这种创新与最高标准的产品质量和患者安全相匹配。” 该指南为制造商提供了灵活的、基于风险的推荐意见，说明如何在开发和商业化的每个阶段整合效价考量——从早期研究性新药（IND）申报到生物制品许可申请（BLA）获批以及持续的上市后验证。关键主题包括原材料变异性控制、生产过程中的检测，以及确认每批产品以其预期治疗效果相匹配的方式具有生物活性的实验室检测。该指南还涉及对短货架期产品、病毒载体基因疗法和患者特异性疗法的特殊考量。 通过为制造商提供清晰、可预测的路线图，本指南支持患者更快获得安全有效的疗法，这些患者往往没有其他治疗选择——其中许多人接受的是为单一个体单独制造的产品。在此过程中，它推进了FDA的公共卫生支柱：通过严格的质量标准保护患者，通过提供监管清晰度促进创新，并加强现存最复杂医疗产品中部分产品的供应链完整性。 FDA将于2026年12月10日举办一场网络研讨会，帮助利益相关者理解和实施该指南。 媒体： FDA征求意见 202-690-6343 消费者： 888-INFO-FDA FDA是美国卫生与公众服务部下属机构，通过确保人用和兽用药物、疫苗及其他人用生物制品和医疗器械的安全性、有效性和安保性来保护公众健康。该机构还负责我国食品供应、化妆品、膳食补充剂、辐射发射电子产品以及烟草产品监管的安全与安保。 ### 内容截至日期： 2026年11月2日 受监管产品 生物制品 关注FDA 在X上关注@US_FDA 在Facebook上关注FDA 在X上关注@FDAMedia
正文 · 英文原文
FDA News Release FDA Provides Clarity to Support Cell and Gene Therapy Development and Quality FDA issues final guidance for cell and gene therapy manufacturers, recommends building potency assurance into every stage of a therapy’s lifecycle — from early development through patient delivery More Press Announcements For Immediate Release: November 02, 2026 The U.S. Food and Drug Administration today issued final guidance for manufacturers of cellular and gene therapy (CGT) products to implement a comprehensive, science- and risk-based plan for making sure their products meet potency standards. The guidance provides FDA’s recommendations for a Potency Assurance Strategy, which guides manufacturers to test and verify — before a product reaches a patient — that every batch meets a defined standard of potency to do what the product is supposed to do therapeutically. The Potency Assurance for Cellular and Gene Therapy Products final guidance replaces FDA's 2011 potency testing guidance for CGT products and applies to treatments for serious and often life-threatening conditions. By law, manufacturers are expected to verify that their products work as intended, from the time of approval through post-approval monitoring. This final guidance provides FDA’s current thinking and recommendations on how manufacturers can meet that requirement. "Patients who receive cellular and gene therapies are often battling serious or life-threatening conditions, such as cancers and rare diseases, and they deserve the assurance that the product administered to them will perform as intended," said Karim Mikhail, B. Pharm., M.S., Director of the Center for Biologics Evaluation and Research. "This guidance reflects the FDA's commitment to ensuring America remains the best place in the world to innovate — and that innovation is matched by the highest standards of product quality and patient safety." The guidance provides flexible, risk-based recommendations for manufacturers on how to integrate potency considerations at every stage of development and commercialization — from early-phase investigational new drug (IND) submissions through biologics license application (BLA) approval and continued post-market verification. Key topics include controls for variability in raw materials, testing during manufacturing, and lab tests that confirm each batch is biologically active in a way that matches its intended therapeutic effect. The guidance also addresses specialized considerations for products with short shelf lives, viral vector gene therapies, and patient-specific therapies. By giving manufacturers a clear, predictable roadmap, this guidance supports faster access to safe and effective therapies for patients who often have no other treatment options — many of whom receive products made individually for a single person. In doing so, it advances the FDA's public health pillars: protecting patients through rigorous quality standards, promoting innovation by providing regulatory clarity, and reinforcing the integrity of the supply chain for some of the most complex medical products in existence. The FDA will host a webinar on December 10, 2026, to help stakeholders understand and implement the guidance. Media: FDA Request for Comment 202-690-6343 Consumer: 888-INFO-FDA The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, radiation-emitting electronic products, and for regulating tobacco products. ### Content current as of: 11/02/2026 Regulated Product(s) Biologics Follow FDA Follow @US_FDA on X Follow FDA on Facebook Follow @FDAMedia on X