GSK plc today announced that risvutatug rezetecan (or Ris-Rez, for short), a B7-H3-targeted antibody-drug conjugate (ADC), has received Orphan Drug Designation (ODD) from Japan’s Ministry of Health, Labour and Welfare for the treatment of small-cell lung cancer (SCLC). The ODD was supported by preliminary clinical data showing durable responses in patients with extensive-stage SCLC (ES-SCLC) who were treated with Ris-Rez in the phase I ARTEMIS-001 clinical trial.1 This is the sixth regulatory designation for Ris-Rez, which is being developed in a range of solid tumours, including lung, prostate and colorectal cancers..

GSK plc 今天宣布，其靶向 B7-H3 的抗体药物偶联物 (ADC) risvutatug rezetecan（简称 Ris-Rez）已获得日本厚生劳动省授予的孤儿药资格 (ODD)，用于治疗小细胞肺癌 (SCLC)。该孤儿药资格得到了初步临床数据的支持，数据显示，在 I 期 ARTEMIS-001 临床试验中，接受 Ris-Rez 治疗的广泛期小细胞肺癌 (ES-SCLC) 患者表现出持久的缓解反应。这是 Ris-Rez 获得的第六项监管认定，该药物正在开发用于多种实体瘤，包括肺癌、前列腺癌和结直肠癌。

Lung cancer is the second most common cancer in Japan, and SCLC makes up 10-15% of cases.2,3 Of patients with SCLC, 70% have ES-SCLC, meaning the cancer has spread throughout one or both lungs and/or to other parts of the body.4 ES-SCLC is an aggressive and difficult-to-treat cancer with limited treatment options and poor long-term survival.

肺癌是日本第二常见的癌症，其中小细胞肺癌 (SCLC) 占病例的10-15%。在小细胞肺癌患者中，70%患有广泛期小细胞肺癌 (ES-SCLC)，这意味着癌细胞已扩散至单侧或双侧肺部和/或身体其他部位。广泛期小细胞肺癌是一种侵袭性强、难以治疗的癌症，治疗选择有限，长期生存率低。

Most patients with ES-SCLC relapse after initial treatment and the median overall survival with standard-of-care for these patients is approximately 8 months.5.

大多数ES-SCLC患者在初始治疗后复发，接受标准治疗的这些患者的中位总生存期约为8个月。

About Ris-Rez

关于Ris-Rez

Ris-Rez is a novel investigational B7-H3-targeted antibody-drug conjugate composed of a fully human anti-B7-H3 monoclonal antibody covalently linked to a topoisomerase inhibitor payload. GSK acquired exclusive worldwide rights (excluding China’s mainland, Hong Kong, Macau, and Taiwan) from Hansoh Pharma to progress clinical development and commercialisation of Ris-Rez.

Ris-Rez 是一种新型的研究性 B7-H3 靶向抗体药物偶联物，由完全人源化的抗 B7-H3 单克隆抗体与拓扑异构酶抑制剂有效载荷共价连接而成。GSK 从翰森制药获得了 Ris-Rez 的独家全球权利（不包括中国大陆、香港、澳门和台湾地区），以推进其临床开发和商业化。

GSK’s global phase III trial (NCT07099898) for Ris-Rez in relapsed ES-SCLC began in August 2025. .

GSK针对Ris-Rez治疗复发性ES-SCLC的全球III期试验（NCT07099898）于2025年8月开始。

Regulatory designations received for Ris-Rez to date include ODDs from the US Food and Drug Administration (FDA) in SCLC and the European Medicines Agency (EMA) in a category of cancer that includes SCLC, called pulmonary neuroendocrine carcinoma; Priority Medicines (PRIME) Designation from the EMA for relapsed or refractory ES-SCLC; and Breakthrough Therapy Designations for relapsed or refractory ES-SCLC and relapsed or refractory osteosarcoma from the US FDA.6,7,8.

迄今为止，Ris-Rez获得的监管指定包括来自美国食品药品监督管理局（FDA）在小细胞肺癌（SCLC）中的孤儿药指定（ODD），以及欧洲药品管理局（EMA）在包含SCLC的一类癌症（称为肺神经内分泌癌）中的指定；EMA针对复发或难治性广泛期小细胞肺癌（ES-SCLC）的优先药物（PRIME）指定；以及美国FDA针对复发或难治性广泛期小细胞肺癌（ES-SCLC）和复发或难治性骨肉瘤的突破性疗法指定。

About GSK

关于GSK

GSK is a global biopharma company with a purpose to unite science, technology, and talent to get ahead of disease together. Find out more at www.gsk.com.

GSK是一家全球生物制药公司，致力于联合科学、技术和人才，共同战胜疾病。欲了解更多信息，请访问www.gsk.com。

Cautionary statement regarding forward-looking statements

关于前瞻性声明的警示性声明

GSK cautions investors that any forward-looking statements or projections made by GSK, including those made in this announcement, are subject to risks and uncertainties that may cause actual results to differ materially from those projected. Such factors include, but are not limited to, those described in the “Risk Factors” section in GSK’s Annual Report on Form 20-F for 2025..

GSK提醒投资者，GSK所做的任何前瞻性声明或预测，包括本公告中所做的声明或预测，均受可能导致实际结果与预测结果存在重大差异的风险和不确定性的影响。这些因素包括但不限于GSK 2025年Form 20-F年度报告“风险因素”部分中描述的内容。

References

参考文献

Source: gsk.com

来源：gsk.com