Pictured: Entrance to Pfizer's office in Belgium/iStock, Alexandros Michailidis

图为辉瑞驻比利时办事处入口/亚历山大·米切利迪斯（AlexandrosMichailidis）伊斯托克（iStock）

The FDA on Friday gave its approval to Pfizer’s one-time gene therapy fidanacogene elaparvovec-dzkt, which will now carry the brand name Beqvez, for the treatment of moderate to severe hemophilia B in adults.

周五，FDA批准了辉瑞公司的一次性基因疗法fidanacogeneelaparvovec dzkt，该疗法现在将使用品牌Beqvez，用于治疗成人中度至重度B型血友病。

Per its label, Beqvez is indicated for patients who are currently on factor IX prophylaxis or who have a history of life-threatening hemorrhage. The gene therapy can also be administered to those who have had repeated serious spontaneous bleeding episodes and are negative for neutralizing antibodies against the adeno-associated virus (AAV) serotype Rh74var..

根据其标签，Beqvez适用于目前正在进行IX因子预防或有危及生命的出血史的患者。基因疗法也可以用于那些反复出现严重自发性出血发作且对腺相关病毒（AAV）血清型Rh74var的中和抗体阴性的患者。。

Aamir Malik, chief U.S. commercial officer at Pfizer, in a statement said that Beqvez’s approval will provide patients with a “medicine that has the potential to offer both long-term bleed protection,” as well as add “value to the healthcare system because of its one-time administration.”

辉瑞（Pfizer）首席美国商务官阿米尔·马利克（AamirMalik）在一份声明中表示，贝奎兹（Beqvez）的批准将为患者提供一种“具有长期止血保护潜力的药物”，并“由于其一次性管理，为医疗保健系统增加价值”

Pfizer will leverage its expertise in the hemophilia space and collaborate with treatment centers, payers and other stakeholders to “ensure the healthcare system is prepared to readily deliver Beqvez to the patients who can benefit from it,” Malike added.

Malike补充道，辉瑞将利用其在血友病领域的专业知识，与治疗中心、付款人和其他利益相关者合作，以“确保医疗保健系统做好准备，随时向可以从中受益的患者提供贝维兹”。

As part of its efforts to ensure broad access to Beqvez, Pfizer also announced on Friday that it will launch an “innovative warranty program” based on the therapy’s durability of response, which in turn will provide “financial protection” for patients and payers by “insuring against the risk of efficacy failure.” The pharma has not yet announced a price for Beqvez..

作为确保广泛使用Beqvez的努力的一部分，辉瑞制药周五还宣布，它将推出一项基于该疗法反应持久性的“创新保修计划”，该计划将通过“确保疗效失败的风险”为患者和付款人提供“财务保障”。该制药公司尚未宣布Beqvez的价格。。

Delivered via an AAV vector, Beqvez is a gene therapy that works by delivering a functional copy of the factor IX gene, which encodes a high-activity protein variant that restores the patient’s ability to clot blood and addresses the hallmark hemophilia B symptom of excessive bleeding. Beqvez was first approved in Canada, which handed Pfizer the regulatory win in January 2024..

Beqvez是一种通过AAV载体传递的基因疗法，它通过传递IX因子基因的功能拷贝起作用，该基因编码一种高活性蛋白质变体，可恢复患者的凝血能力，并解决血友病B的标志性症状，即过度出血。Beqvez首次在加拿大获得批准，这使辉瑞在2024年1月获得监管胜利。。

Beqvez’s FDA approval on Friday was supported by data from the pivotal Phase III BENEGENE-2 study, an open-label and single-arm trial that enrolled 45 male patients with factor IX circulating activity of 2% or lower.

Beqvez周五获得FDA批准的数据得到了关键的III期BENEGENE-2研究的支持，该研究是一项开放标签的单臂试验，招募了45名IX因子循环活性为2%或更低的男性患者。

Results showed that a single intravenous dose of Beqvez led to a median annualized bleeding rate (ABR) of zero during the study’s efficacy evaluation period. By comparison, patients in the control arm who received a standard factor IX prophylaxis replacement regimen had a median ABR of 1.3. Beqvez also eliminated bleeding in 60% of patients versus 29% in the prophylaxis group..

结果显示，在研究的疗效评估期间，单次静脉注射Beqvez导致中位年化出血率（ABR）为零。相比之下，接受标准IX因子预防替代方案的对照组患者的中位ABR为1.3。Beqvez还消除了60%的患者出血，而预防组为29%。。

In terms of safety, BENEGENE-2 found that Beqvez was generally well-tolerated with no serious adverse events or deaths associated with the gene therapy or with infusion reactions. There were also no thrombotic events reported in the study. Pfizer will continue to monitor patients for 15 years for long-term durability and safety..

就安全性而言，BENEGENE-2发现Beqvez通常耐受性良好，没有与基因治疗或输注反应相关的严重不良事件或死亡。研究中也没有报道血栓形成事件。辉瑞将在15年内继续监测患者的长期耐用性和安全性。。

With Beqvez’s approval, Pfizer now follows in the footsteps of CSL Behring, which secured the FDA’s greenlight for its own hemophilia gene therapy Hemgenix (etranacogene dezaparvovec-drlb) in November 2022. For the full year ended June 2023, Hemgenix made nearly $1.2 billion in revenue and brought in $662 million more by the end of December 2023..

在Beqvez的批准下，辉瑞现在追随CSL Behring的脚步，该公司于2022年11月为其自己的血友病基因治疗Hemgenix（etranacogene dezaparvovec drlb）获得了FDA的批准。截至2023年6月，Hemgenix全年收入近12亿美元，截至2023年12月底，收入增加6.62亿美元。。

Tristan Manalac is an independent science writer based in Metro Manila, Philippines. Reach out to him on LinkedIn or email him at tristan@tristanmanalac.com or tristan.manalac@biospace.com.

特里斯坦·马纳拉克（TristanManalac）是一位独立的科学作家，总部位于菲律宾马尼拉。在LinkedIn上联系他，或发电子邮件给他tristan@tristanmanalac.com或tristan.manalac@biospace.com.