FDA在1月29日发布了Human Gene Therapy Products Incorporating Human Genome Editing. Guidance for Industry的最终指南,指导赞助商开发人类体细胞基因组编辑的基因治疗产品。 指南阐述了IND 申请中应提供的信息,以评 GE 产品的安全性和质量,涵盖产品制造和测试...
The Fast Track designation is designed to facilitate the development of drugs that address serious conditions and unmet medical needs. Drugs with this designation benefit from more frequent interactions with the FDA, timely feedback, and guidance. Fast Track also allows for rolling submissions, where ...
[9]. 奥来恩专家解读(第8期):FDA孤儿药认定-新药研发与审评的加速通道系列之五。 [10]. FDA Guidance for Industry: Rare Diseases: Common Issues in Drug Development. January 2019, Revision 1. [11]. CDER’s Perspective on Working Together with Our Rare Disease Partners within CDER and across FDA...
研讨会和培训 ICH M12 Drug-Drug Interaction Studies Final Guidance, October 9, 2024 (Hosted by CDER SBIA)美东时间2024年10月9日，FDA CDER举行了题为“ ICH M12 药物相互作用研究最终指南”的研讨会。该网络研讨会将讨论ICH M12药物相互作用研究指南，该指南是第一份全球统一的监管指南，用于评估由代谢酶和...
例如2018年7月CBER发布的《罕见病基因治疗(草案稿)》(Human Gene Therapy for Rare Diseases ; Draft Guidance for Industry)、2017年11月(2017年12月更正)CBER、CDRH和OCP联合发布的《人类细胞、组织以及基于细胞和组织产品最小操作和同源使用的监管考虑》(Regulat...
ICH M12 Drug-Drug Interaction Studies Final Guidance, October 9, 2024 (Hosted by CDER SBIA) 美东时间2024年10月9日,FDA CDER举行了题为“ ICH M12 药物相互作用研究最终指南”的研讨会。该网络研讨会将讨论ICH M12药物相互作用研究指南,该指南是第一份全球统一的监管指南,用于评估由代谢酶和药物转运体介导...
[10]. FDA Guidance for Industry: Rare Diseases: Common Issues in Drug Development. January 2019, Revision 1. [11]. CDER’s Perspective on Working Together with Our Rare Disease Partners within CDER and across FDA, Kerry Jo Lee, M.D., REdI Annual Conference 2022. ...
About 80% rare diseases are caused by single-gene defect, and about half affect children. Because most rare diseases have no approved treatment, there is significant unmet demand. One the Human Gene Therapy for Rare Diseases Guidance is finalized, it will provide recommendations on pre-clinical,...
One of the first things the FDA touches on in thedraft guidanceis the unique nature of ASO therapies. Because of their individualized essence, patients and family members may function more as collaborators than research subjects and may sometimes attend sponsor-FDA meetings. ...
http://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/HowtoapplyforOrphanProductDesignation/ucm135122.htm (2) Wilate(R) Approved Complete Prescribing Information, December 2009. (3) Ibid. (4) Note for guidance on virus validation studies: The design, ...