医学
血友病
销售授权
授权
药物治疗
血友病A
病毒学
儿科
腺相关病毒
内科学
生物信息学
基因
重组DNA
载体(分子生物学)
遗传学
生物
计算机安全
计算机科学
出处
期刊:Drugs
[Springer Nature]
日期:2022-09-01
卷期号:82 (14): 1505-1510
被引量:26
标识
DOI:10.1007/s40265-022-01788-y
摘要
Valoctocogene roxaparvovec (ROCTAVIAN™) is a gene therapy being developed by BioMarin Pharmaceutical Inc. for the treatment of haemophilia A. In August 2022, valoctocogene roxaparvovec was granted conditional marketing authorization in the EU for the treatment of severe haemophilia A [congenital factor VIII (FVIII) deficiency] in adults without a history of FVIII inhibitors and without detectable antibodies to adeno-associated virus serotype 5 (AAV5). This article summarizes the milestones in the development of valoctocogene roxaparvovec leading to this first approval for severe haemophilia A.
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