6- FDA Hopes To Bring Rare Disease Uses On-Label With New DatabaseMoscovitch, Ben
1、Abeona Gets FDA Nod for Rare Skin Disease Gene Therapy;biospace 2、As Krystal's Vyjuvek gains momentum, analysts spotlight 'transition point' in topical gene therapy launch;fiercepharma 3、Abeona官网
参考资料: FDA missesPDUFA date for Stealth’s ultra-rare disease candidate, delaying approval decision—again (Fierce Biotech) 其它公开资料 图片来源:网络
Sensitive Personal Data and Government-Related Data by Countries of Concern or Covered Persons2025年4月8日,美国总统拜登于2024年2月28日签署的第14117号行政令《防止受关注国家获取美国人大量敏感个人数据和美国政府相关数据》正式生效。根据美国司法部2024年12月发布的实施细则,该行政令以“国家安全”为由,旨在...
万邦德(002082.SZ)：石杉碱甲获美国FDA罕见儿科疾病药物认定 万邦德(002082.SZ)发布公告，公司全资子公司万邦德制药集团有限公司于2024年5月1日收到美国食品药品监督管理局(简称“FDA”)的认定函，石杉碱甲用于治疗新生儿缺氧缺血性脑病(HIE)获得FDA授予的“罕见儿科疾病”(Rare Pediatric Disease,RPD)药物认定。本文...
Estimating cumulative point prevalence of rare diseases: analysis of the Orphanet database. Eur J Hum Genet. 2020;28:165–73. National Organization for Rare Disorders (NORD). Rare Disease Facts & Statistics. Accessed 27 October 2023. 作者 Karen Driver Karen Driver拥有20年的制药行业经验。她领导...
CDER and CBER leaders update initiatives in the rare disease space within the past year. In the FDA Rare Disease Town Hall at DIA 2023, Chair James Valentine, Director for Hyman, Phelps & McNamara, PC, laid the foundation for FDA rare disease therapeutic approvals and then introduced the ...
Over the past 4 decades since the Orphan Drug Act was passed in 1983, there have been tremendous advances in research, innovation, and regulatory policies that have spurred development of new treatments for rare diseases, but less than 5% of rare diseases have approved treatments on the market...
25, No. 6 表 3 FAERS 数据库中 AE 报告数居前 20 位的氯苯唑酸相关不良事件的首选术语及信号检测结果 Tab 3 The preferred terms and detection results of signal of the top 20 adverse event reports related to tafamidis in the FAERS database 首选术语 系统器官分类 呼吸困难 呼吸系统/胸及纵膈疾病...
在医药监管领域,近期美国食品药品监督管理局(FDA)专员马蒂・马卡里(Marty Makary)的一系列言论引发了广泛关注。在接受前福克斯新闻记者梅根・凯利(Megyn Kelly)的采访中,马卡里不仅介绍了针对罕见病疗法的全新审批路径,还就公众对 FDA 的信任问题发表了看法,这些观点在行业内外激起了层层涟漪。