FDA Grants Gene Therapy Orphan Drug Status for Rare Genetic DisorderLea EslavaKimPharmD
访问 fda.gov/drugs，点击页面下拉菜单中的"Drug Approvals and Databases"，浏览药物数据库。执行药物搜索 点击"Drug@FDA search"，在红色框内输入美获批药物的英文名称或活性成分进行搜索。例如，输入"KEYTRUDA"，页面会显示相关搜索结果，点击展开即可下载药物批准时的说明书与综合信息。下载药物说明书与...
FDA(fda.gov/)监管的项目除了包含药品(化药、生物制品)、器械(一般设备、辐射器械),还包括食品烟草等。药物临研通常关注药品部分,主要是Drug版块和Vaccines, Blood & Biologics版块。本期通过图解的方法,简单明了地介绍FDA这两个版块搜索美国已获批药品的说明书、综述资料的方法。 Drugs 常规药物 信息搜索疫苗、...
(http://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products)。 实操举例 作为新药研发人员,我们更关心FDA新审批药物的审批文件,包括说明书和审评报告。 下面就简要说明一下使用方法(以KRAZATI为例)。 首先进入Drugs@FDA的搜索栏输入KRAZATI,然后点击Se...
[1]https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-first-adenoviral-vector-based-gene-therapy-high-risk-bacillus-calmette-guerin [2]https://www.ferring.com/ferring-receives-approval-from-u-s-fda-for-adstiladrin-for-...
1.https://www.fda.gov/drugs/new-drugs-fda-cders-new-molecular-entities-and-new-therapeutic-biological-products/novel-drug-approvals-2023 2.https://www.fda.gov/vaccines-blood-biologics/development-approval-process-cber/2023-biological-license-application-approvals 3.https://www.fda.gov/media/...
As with other kinds of drugs, FDA regulates the quality & safety of gene therapy products and supervises how these products are used clinically.; Therapeutic alteration of the human genome falls under the same regulatory requirements as any other medical treatment. Research involving human subjects,...
12. Guidance for Industry: Potency Tests for Cellular and Gene Therapy Products, January 2011, https://www.fda.gov/media/79856/download. 13. Analytical Procedures and Methods Validation for Drugs and Biologics; Guidance for Industry, July 2015, https://www.fda.gov/media/87801/download. ...
12. Guidance for Industry: Potency Tests for Cellular and Gene Therapy Products, January 2011,https://www.fda.gov/media/79856/download. 13. Analytical Procedures and Methods Validation for Drugs and Biologics; Guidance for Industry, July 2015,https://www.fda.gov/media/87801/download. ...
1.https://www.fda.gov/drugs/new-drugs-fda-cders-new-molecular-entities-and-new-therapeutic-biological-products/novel-drug-approvals-2023 2.https://www.fda.gov/vaccines-blood-biologics/development-approval-process-cber/2023-biological-license-application-a...