FDA于2023年12月出台了效价的指南来帮助生产商满足效价分别在INDs和BLAs时的要求，“Potency Assurance for Cellular and Gene Therapy Products: Draft Guidance for Industry”。除此之外，和CQAs有关的详细信息也可以参考 “Q8(R2) Pharmaceutical Development: Guidance for Industry,” November 2009，“Manufacturing ...
如果CMC数据不完整,审批过后会需要解决更多的问题:更多稳定性数据;额外的确认性验证(过程稳健性)研究;场地、生产规模、原料供应商的变化——原料药合成路线的变化;生物利用度研究支持的配方改变等。 面对由CMC问题导致的CRL急速增加的现状,FDA发言人曾...
We (FDA) recommend that you use this guidance as a companion to otherFDA guidance documents describing the chemistry, manufacturing, and control (CMC) information submitted and reviewed in an IND application for phase 1 clini...
FDA于2023年12月出台了效价的指南来帮助生产商满足效价分别在INDs和BLAs时的要求,“Potency Assurance for Cellular and Gene Therapy Products: Draft Guidance for Industry”。除此之外,和CQAs有关的详细信息也可以参考 “Q8(R2) Pharmaceutical Development: Guidance for Industry,” November 2009, “Manufacturing ...
The FDA has issued its guidance for chemistry, manufacturing, and control information that is included in investigational new drug applications (INDs) for gene therapies, called “Content and Review of Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New ...
Guidance for FDA Reviewers and Sponsors Content and Review of Chemistry, Manufacturing, and Control (CMC) Information for Human Somatic Cell Therapy Investigational New Drug Applications (INDs) Additional copies of this guidance are available from the Office of Communication, Training, and Manufacturers ...
比如I期临床样品应符合“CGMP for Phase 1 Investigational Drugs: Guidance for Industry,” July 2008的要求,II期及以后应符合21 CFR part 210 & 211的cGMP要求。BLA必须包含证明产品符合安全性,纯度和效价要求的数据,生产方法的完整描述以及既定效期的产品稳定性数据。必须进行符合cGMP的工艺表征和工艺验证。
• “FDA 审评员和申办方指南：人类体细胞疗法新药研究申请 (IND) 的化学、制造和控制 (CMC) 信息的内容和审查”指南, 2008年4月发布, 细胞疗法 CMC 指南。Recognition and Use of a Standard for Uniform Blood and Blood Component Container Labels: Guidance for Industry 2024年4月30日，FDA发布了一...
1. Guidance for FDA Reviewers and Sponsors: Content and Review of Chemistry,Manufacturing, and Control (CMC) Information for Human Somatic Cell Therapy Investigational New Drug Applications (INDs); dated April 2008 2. Guidance for Industry: Preclinical Assessment of Investigational Cellular and Gene ...
Human Gene Therapy for hemophilia guidance 目前，正在开发的血友病基因治疗产品有望“一劳永逸”——单次治疗可以让患者体内长期表达缺失或者异常的凝血因子。这一改变可以减少或者取消患者对凝血因子替代品的需要。为确保这类产品的研发途径，FDA发布了这一份新的指南草案。一旦最终定稿，这份指南将为血友病基因治疗...