辉瑞B型血友病基因疗法在美获批上市，定价350万美元

医药魔方04-27

4月26日，辉瑞宣布，FDA已批准其基因疗法BEQVEZ（fidanacogene elaparvovec-dzkt，SPK-9001）上市，用于治疗中重度B型血友病成人患者。BEQVEZ的定价为350万美元，与之前批准的B型血友病基因疗法Hemgenix价格一致。随着BEQVEZ的获批应用，辉瑞正在根据患者对治疗反应的持久性推出一项创新保修计划。该计划旨在为支付方提供更大的确定性，最大限度地让...

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elaparvovec-dzkt，SPK-9001）上市，用于治疗中重度B型血友病成人患者。BEQVEZ的定价为350万美元，与之前批准的B型血友病基因疗法Hemgenix价格一致。随着BEQVEZ的获批应用，辉瑞正在根据患者对治疗反应的持久性推出一项创新保修计划。该计划旨在为支付方提供更大的确定性，最大限度地让符合条件的患者获得BEQVEZ，并对治疗后疗效不佳的患者提供财务保护。BEQVEZ最初由Spark Therapeutics（已被罗氏收购）开发，这是一种针对FIX的一次性基因疗法，含有生物工程腺相关病毒（AAV）衣壳（蛋白质壳）和人FIX基因的高活性变体。2014年12月，辉瑞与Spark达成合作，以2.8亿美元的总交易额获得该产品的全球权益。此次获批主要基于III期BENEGENE-2研究（n=45）的积极数据。结果显示，患者接受单剂次BEQVEZ治疗12个月内，年化出血率（ABR）相比标准预防治疗组（接受FIX类药物作为预防替代治疗）显著降低（1.3 vs. 4.43，P<0.0001），达到非劣效标准并且也达到了优效标准。除BEQVEZ外，辉瑞目前还有两款处于III期临床阶段的基因疗法，分别为用于A型血友病的giroctocogene fitelparvovec和用于杜氏肌营养不良症的fordadistrogene movaparvovec。此外，辉瑞的TFPI单抗marstacimab也已于2023年12月在欧美申报上市，拟用于治疗A型以及B型血友病。FDA将PDUFA日期定在2024年Q4，EMA预计在2025年Q1做出审批决定。值得注意的是，诺和诺德开发的TFPI单抗concizumab已在加拿大和日本获批上市，不过，该产品暂被FDA拒之门外。诺和诺德在2023Q1财报中透露，已收到FDA就concizumab用于预防治疗伴抑制物的A型和B型血友病的生物制品许可申请（BLA）发出的完整回复函（CRL）。FDA要求其提供额外的与患者相关的监测和给药信息，以确保concizumab按预定方案给药。B型血友病是一种因先天缺乏FIX导致的遗传性出血性疾病。治疗B型血友病的标准方法是预防性输注FIX替代疗法，以暂时替代或补充低水平的凝血因子。尽管采取了预防措施并定期进行静脉输液，但许多中度至重度 B 型血友病患者仍有自发性出血发作的风险。根据世界血友病联合会统计，全球B型血友病患者超过3.8万例。Copyright © 2023 PHARMCUBE. 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