FDA批准首创慢性乙肝新药Hibsago：GSK的bepirovirsen六个月疗程功能性治愈率达19%
美国食品药品监管局（FDA）10月20日批准 GSK 公司的 Hibsago（通用名 bepirovirsen）用于治疗慢性乙型肝炎，这是 FDA 批准的首个用于慢性乙肝的反义寡核苷酸药物。该药须与核苷（酸）类似物逆转录酶抑制剂联合使用，适用于无肝硬化、已接受该类治疗至少6个月、乙肝病毒 DNA 已受抑制且乙肝表面抗原（HBsAg）水平不高于3000 IU/ml 的成人患者；此次批准经 FDA 优先审评程序、并获突破性疗法认定。
支持批准的 B-WELL 三期临床试验汇总数据显示，六个月 bepirovirsen 治疗的功能性治愈率达19%（1,220名受试者中233人），安慰剂组为0，而现行标准治疗一年后的功能性治愈率通常不足1%。功能性治愈指停用全部治疗至少24周后，乙肝病毒 DNA 与表面抗原仍检测不到、即疾病由免疫系统在不用药情况下得到控制。GSK 首席科学官 Tony Wood 表示，许多慢性乙肝患者目前须无限期服药、并持续面临严重肝脏并发症风险，Hibsago 首次让美国患者有机会在六个月疗程后实现功能性治愈，且首剂之后可选择自行注射给药。
GSK 表示，慢性乙肝影响美国约170万人，全球有超过2.4亿人患慢性乙肝、每年约110万人死亡。Bepirovirsen 由 GSK 从 Ionis Pharmaceuticals 引进授权并合作开发，此前已在日本获批，在中国与欧洲的注册审评也在推进。FDA 公告同时载明，该药带有严重肝脏反应与血小板减少等黑框警告，常见不良反应包括注射部位反应、发热、疲劳与头痛，治疗前后须定期验血与临床随访。
FDA Approves First-in-Class Chronic Hepatitis B Drug Hibsago: GSK's Bepirovirsen Shows 19% Functional Cure Rate in Six-Month Course
The U.S. Food and Drug Administration (FDA) on October 20 approved GSK's Hibsago (bepirovirsen) for the treatment of chronic hepatitis B, the first antisense oligonucleotide approved by the FDA for the condition. The drug is to be used in combination with nucleos(t)ide analogue reverse transcriptase inhibitor therapy in adults without cirrhosis who have received that therapy for at least six months, have suppressed HBV DNA, and have hepatitis B surface antigen (HBsAg) levels of no more than 3,000 IU/ml. The approval was granted under the FDA's Priority Review program with Breakthrough Therapy Designation.
Pooled data from the B-WELL phase III trials showed a functional cure rate of 19 percent after a six-month course of bepirovirsen (233 of 1,220 participants), compared with zero in the placebo group; current standard of care typically achieves a functional cure rate of less than 1 percent after one year of treatment. A functional cure means HBV DNA and HBsAg remain undetectable for at least 24 weeks after all treatment stops, indicating the disease is controlled by the immune system without medication. Tony Wood, GSK's Chief Scientific Officer, said many people living with chronic hepatitis B remain on treatment indefinitely and continue to face the risk of serious liver complications, and that Hibsago gives U.S. patients, for the first time, the potential to achieve functional cure after a six-month course, with the option of self-administration after the first dose.
GSK said chronic hepatitis B affects approximately 1.7 million people in the United States, while more than 240 million people worldwide live with the disease and about 1.1 million die from it each year. Bepirovirsen was licensed from Ionis Pharmaceuticals and developed in collaboration with the company; it has already been approved in Japan, and regulatory reviews are under way in China and Europe. The FDA noted that the drug carries a boxed warning for serious liver reactions and thrombocytopenia, and that common adverse reactions include injection site reactions, fever, fatigue and headache, with regular blood testing and clinical follow-up advised before and during treatment.
发布：GSM·美国（发布链接）
图片来源：乙型肝炎病毒（Dane 颗粒）透射电子显微镜图像（资料图）。美国疾病控制与预防中心（CDC）发布，公共领域，经 Wikimedia Commons 发布／Transmission electron micrograph of hepatitis B virions (Dane particles) (file photo). U.S. Centers for Disease Control and Prevention, public domain, via Wikimedia Commons
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