EMA has recommended granting a conditional marketing authorisation in the European Union (EU) for Durveqtix (fidanacogene elaparvovec) to treat severe and moderately severe haemophilia B in adults who do not have factor IX inhibitors (auto-antibodies produced by the immune system against factor IX replacement medicines) and who have no detectable antibodies to variant adeno-associated virus serotype Rh74 (AAVRh74var).Haemophilia B is a rare inherited bleeding disorder.

EMA建议在欧盟（EU）授予Durveqtix（fidanacogene elaparvovovec）有条件的上市许可，以治疗没有IX因子抑制剂（免疫系统产生的针对IX因子替代药物的自身抗体）且没有可检测到的变异腺相关病毒血清型Rh74（AAVRh74var）抗体的成年人的重度和中度血友病B。血友病B是一种罕见的遗传性出血性疾病。

The condition is caused by the lack of coagulation factor IX, a protein needed to produce blood clots to stop bleeding and seal wounds. Without that protein, patients with haemophilia B bruise easily and bleed more frequently and for longer periods of time. It can lead to serious complications, such as bleeding in joints, muscles or internal organs, including the brain. Most of the currently authorised medicines for haemophilia B require frequent and lifelong intravenous infusions to prevent or treat bleeding.

这种情况是由于缺乏凝血因子IX引起的，凝血因子IX是一种产生血块以止血和封闭伤口所需的蛋白质。如果没有这种蛋白质，B型血友病患者很容易瘀伤，出血频率更高，时间更长。它可能导致严重的并发症，如关节、肌肉或包括大脑在内的内脏出血。目前大多数经授权的B型血友病药物都需要经常和终身静脉输注以预防或治疗出血。

Patients need more new treatments that provide sustained bleed protection, reduce frequency of infusions and improve their quality of life.Durveqtix is a gene therapy delivered as a single infusion that aims at enabling the body to produce factor IX itself and prevent and control bleeding.The recommendation is based on the results of an ongoing single-arm, open-label, phase 3 trial in 45 adult male patients with moderately severe or severe haemophilia B who tested negative for neutralising antibody to AAVRh74var and received a single intravenous infusion of fidanacogene elaparvovec.

患者需要更多新的治疗方法，以提供持续的出血保护，减少输液频率并提高生活质量。Durveqtix是一种单次输注的基因疗法，旨在使身体自身产生IX因子并预防和控制出血。该建议基于正在进行的单臂开放标签3期临床试验的结果，该试验对45名患有中度重度或重度B型血友病的成年男性患者进行了检测，这些患者的AAVRh74var中和抗体检测为阴性，并接受了单次静脉输注fidanacogene elaparvovec。

The study compares the annualised bleeding rate (ABR), which included both treated and untreated bleeding events, in participants treated with gene therapy versus the period when they were treated with routine factor IX .

该研究比较了接受基因治疗的参与者与接受常规因子IX治疗的参与者的年出血率（ABR），其中包括治疗和未治疗的出血事件。

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