Dive Brief:

简报：

Weak demand for Pfizer’s hemophilia gene therapy Beqvez has led the pharmaceutical company to “cease further development and commercialization” of the one-time treatment, a Pfizer spokesperson confirmed to BioPharma Dive.

辉瑞公司发言人向《生物医药潜水》证实，由于对辉瑞的血友病基因疗法Beqvez需求疲软，该公司已决定“停止进一步开发和商业化”这种一次性治疗药物。

Originally developed by Spark Therapeutics, Beqvez was licensed by Pfizer in 2014 and

最初由Spark Therapeutics开发，Beqvez于2014年获得辉瑞的许可，并

gained U.S. approval last April for people with hemophilia B

去年四月获得了美国对B型血友病患者的批准

, the less common form of the genetic bleeding condition. No sales have been disclosed by Pfizer.

，这是遗传性出血疾病的较少见形式。辉瑞公司尚未披露任何销售数据。

Pfizer intends to focus its resources on treatments it thinks “will have the greatest impact on patients,” such as

辉瑞打算将其资源集中在它认为“将对患者产生最大影响”的治疗上，例如

a new drug for hemophilia A called Hympavzi

一种名为Hympavzi的新型A型血友病药物

that was recently cleared, the spokesperson added.

发言人补充说，那片区域是最近才清理出来的。

Dive Insight:

潜水洞察：

Pfizer’s decision to drop Beqvez essentially marks the company’s exit from the field of virally delivered gene replacement treatments. The drugmaker

辉瑞决定放弃Beqvez，这基本上标志着该公司退出了病毒递送基因替代疗法领域。这家制药商

backed away from early research in 2023

在2023年放弃了早期研究

to focus its efforts on later-stage gene therapies for Duchenne muscular dystrophy, hemophilia A and hemophilia B.

专注于后期阶段的杜氏肌营养不良症、甲型血友病和乙型血友病的基因疗法。

Those projects have now all been abandoned. Last July, Pfizer

那些项目现在都已经放弃了。去年七月，辉瑞公司

gave up developing the Duchenne therapy

放弃了开发杜氏治疗方案

after disappointing data from Phase 3 testing, laying off some 150 employees in the process. Then, in January, it surprised partner Sangamo Therapeutics by

在第三阶段测试数据令人失望之后，裁员了大约150名员工。然后，在一月份，它让合作伙伴Sangamo Therapeutics感到意外，

handing back rights

交回权利

to the hemophilia A treatment, which was set to be submitted for regulatory approval.

针对甲型血友病的治疗，即将提交监管审批。

Beqvez remained, but Pfizer appears to have lost confidence in its sales potential. In explaining Pfizer’s decision, the company spokesperson cited “the limited interest patients and their doctors have demonstrated in hemophilia gene therapies to date.”

贝克韦斯仍然存在，但辉瑞似乎已经对其销售潜力失去信心。在解释辉瑞的决定时，公司发言人提到了“迄今为止，患者及其医生对血友病基因疗法表现出的有限兴趣。”

Two others are approved in the U.S.: Hemgenix, which is sold by CSL Behring for hemophilia B, and Roctavian, marketed by BioMarin Pharmaceutical for hemophilia A.

美国还有另外两种基因疗法获批：CSL Behring销售的用于治疗B型血友病的Hemgenix，以及BioMarin Pharmaceutical销售的用于治疗A型血友病的Roctavian。

Sales of both have been slow. CSL hasn’t broken out specific figures for Hemgenix, which carries a

两种药物的销售均较为缓慢。CSL并未单独列出Hemgenix的具体数据，该药物的

list price in the U.S. of $3.5 million

美国标价为350万美元

. BioMarin reported $26 million in revenue for Roctavian, which it

BioMarin报告了Roctavian的收入为2600万美元，它

sells for $2.9 million

售价为290万美元

. The company has

公司已经

limited sales to only the U.S., Italy and Germany

仅限于美国、意大利和德国的销售

, while ending most clinical development work as it focuses on other parts of its business.

，同时终止大部分临床开发工作，集中精力于业务的其他部分。

All three therapies promise long-term control of bleeding by delivering functional copies of the genes mutated in people with the disease. Studies have proven them to be potent for at least several years, but hemophilia patients have other, effective treatments to choose from. And because of the way the therapies are built, patients may only be able to try one of the treatments, potentially incentivizing them to wait and see..

所有这三种疗法都通过提供功能性的基因拷贝，来实现对出血的长期控制，这些基因在患者体内发生了突变。研究已经证明，它们至少在几年内是有效的，但血友病患者还有其他有效的治疗方法可供选择。而且，由于这些疗法的构建方式，患者可能只能尝试其中一种治疗，这可能会促使他们选择等待和观望。

Pfizer said it will communicate its decision to the patients who are in the process of qualifying for Beqvez treatment. For anyone who was treated with the therapy in a clinical trial, Pfizer said it will continue to support the treatment sites in follow-up monitoring.

辉瑞表示，将把决定告知那些正在接受Beqvez治疗资格审查的患者。对于任何在临床试验中接受过该疗法治疗的人，辉瑞表示将继续支持治疗点进行后续监测。

Pfizer has also informed Roche, which previously bought Spark, of its decision and is discussing the “best next steps” for Beqvez.

辉瑞也已将其决定告知了此前收购Spark的罗氏，并正在讨论Beqvez的“最佳后续步骤”。