Xu L, Irony I, Bryan W W, Dunn B
Office of Tissues and Advanced Therapies, Center for Biologics Evaluation and Research, United States Food and Drug Administration, Silver Spring, MD, USA.
Division of Neurology Products, Office of New Drugs, Center for Drug Evaluation and Research, United States Food and Drug Administration, Silver Spring, MD, USA.
Gene Ther. 2017 Sep;24(9):527-528. doi: 10.1038/gt.2017.64. Epub 2017 Jul 24.
The nusinersen development and approval process provide important lessons regarding the pathway to marketing approval for gene therapies. These lessons emphasize rigorous clinical trial design, flexibility in trial design and analysis, a collaborative effort with regular communications between the drug developer and the Food and Drug Administration (FDA), and use of FDA's expedited programs. These lessons are critical to the development of gene therapies for the treatment of serious or life-threatening rare diseases.
诺西那生的研发和批准过程为基因疗法的上市批准途径提供了重要经验教训。这些经验教训强调了严格的临床试验设计、试验设计和分析中的灵活性、药物开发商与美国食品药品监督管理局（FDA）之间定期沟通的协作努力以及对FDA加速项目的使用。这些经验教训对于开发用于治疗严重或危及生命的罕见疾病的基因疗法至关重要。
Zotero 插件