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Drugs. 2022 Sep;82(14):1505-1510. doi: 10.1007/s40265-022-01788-y.
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.
罗特西普（ROCTAVIAN）是一种基因治疗药物，由生物梅里埃公司（BioMarin Pharmaceutical Inc.）开发，用于治疗 A 型血友病。2022 年 8 月，罗特西普在欧盟获得有条件上市许可，用于治疗既往无 FVIII 抑制剂、无腺相关病毒血清型 5（AAV5）检测抗体的成人重度 A 型血友病（先天性 FVIII 缺乏症）。本文总结了导致该药首次获批用于治疗重度 A 型血友病的关键研发进展。
