FDA Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs)

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Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs)[附网盘链接]是于之前发布的FDA标准，适用于US。

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Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs) Guidance for Industry This guidance represents the current thinking of the Food and Drug Administration (FDA or Agency) on this topic. It does not establish any rights for any person and is not binding on FDA or the public. You can use an alternative approach if it satisfies the requirements of the applicable statutes and regulations. To discuss an alternative approach, contact the FDA staff responsible for this guidance as listed on the title page. I. INTRODUCTION Human gene therapy seeks to modify or manipulate the expression of a gene or to alter the biological properties of living cells for therapeutic use. We, FDA, are providing you, sponsors of human gene therapy Investigational New Drug Applications (INDs), recommendations regarding chemistry, manufacturing, and control (CMC) information submitted in an IND. The purpose of this guidance is to inform sponsors how to provide sufficient CMC information required to assure product safety, identity, quality, purity, and strength (including potency) of the investigational product (21 Code of Federal Regulations (CFR) 312.23(a)(7)(i)). This guidance 1

applies to human gene therapy products and to combination products that contain a human gene 2

therapy in combination with a drug or device. This guidance finalizes the draft guidance of the same title dated July 2018 and supersedes the document entitled “Guidance for FDA Reviewers and Sponsors: Content and Review of Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs),” dated April 2008 (April 2008 guidance). The field of gene therapy has progressed rapidly since we issued the April 2008 guidance. Therefore, we are updating that guidance to provide you with current FDA recommendations regarding the CMC content of a gene therapy IND. This guidance is organized to follow the structure of the FDA guidance on the Common Technical Document (CTD). Information on the CTD can be found in FDA’s Guidance for Industry: “M4Q: The CTD – Quality,” (Ref. 1). For information on the submission of an electronic CTD (eCTD), please see the FDA website https://www.fda.gov/drugs/electronic-regulatory-submission-and-review/electronic-common-

technical-document-ectd. 1

Combination products are comprised of any combination of a drug and a device; a device and a biological product; a biological product and a drug; or a drug, a device, and a biological product; see 21 CFR 3.2(e) for the complete definition of combination product. Combination products are assigned to a lead center for review; see 21 CFR 3.4. 2

This guidance does not apply to vaccines for infectious disease indications, bacteriophage products, live biotherapeutic products, fecal microbiota for transplantation (FMT) products and allergenic products.

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