戈芮宁®：中国首个自主研发的酶替代药物正式进入临床，开启全球化进程

近日，北海康成-B（01228.HK）自主研发的酶替代治疗药物戈芮宁®（Velaglucerase Alfa for Injection）在上海交通大学医学院附属新华医院正式开出首张处方，标志着这一国产创新药物正式进入临床使用阶段，也为全球罕见病治疗带来新的中国方案。

🔬 产品介绍

**戈芮宁®**适用于 12岁及以上青少年与成人I型与III型戈谢病患者的长期酶替代治疗，是中国首个本土研发、拥有完全知识产权的酶替代药物。

• 成分：维拉苷酶β（Velaglucerase Alfa）
• 适应症：I型和III型戈谢病（罕见遗传性代谢病）
• 治疗机制：为体内缺乏的β-葡萄糖脑苷脂酶提供替代，缓解肝脾肿大、贫血、骨痛等症状
• 生产基地：中国制造，质量标准对标国际

💡 市场亮点

• ✅ 成本优势显著：预期上市定价**至少低于现有进口疗法50%**以上，极大提升药物可及性
• ✅ 医保前景积极：公司已启动2024年医保谈判流程，致力于扩大覆盖范围
• ✅ 患者服务体系完善：搭建“患者识别-用药指导-长期管理”闭环服务体系
• ✅ 临床落地加速：已在国内三甲医院完成首方开具，市场启动节奏良好

🌍 出海进程启动

在国内落地的同时，戈芮宁®正加速国际化布局。北海康成已明确表示将推动其向全球罕见病治疗市场出口，针对东南亚、中东、拉美等医疗资源不平衡市场进行优先注册与渠道布局，致力于将这一中国原研药推向更广泛的人群。

🧭 企业简介：北海康成-B（01228.HK）

北海康成是一家专注于罕见病治疗药物研发与商业化的生物医药企业，致力于解决中国及全球未被满足的临床需求。公司拥有覆盖从研发、注册、生产到商业化的一体化能力。

🤝 海外合作对接中

戈芮宁®目前正在积极寻找国际合作伙伴，包括：

• 药品代理商与分销商
• 医疗渠道资源方
• 跨境注册与市场准入服务机构
• 罕见病治疗领域的医疗机构与基金组织

📧 合作请联系：globalfrontier1@proton.me
📱 微信咨询：globalfrontier01
🌐 平台主页：www.globalfrontier1.com

我们欢迎全球健康产业链伙伴共建“中国创新药出海”的新路径。

GORINING®: China’s First Homegrown Enzyme Replacement Therapy Enters Clinical Use and Eyes Global Expansion

Beigene Kangcheng-B (01228.HK) has officially launched clinical use of its innovative enzyme replacement therapy drug GORINING® (Velaglucerase Alfa for Injection), marking a major milestone for China’s rare disease treatment sector. The drug was prescribed for the first time at Xinhua Hospital affiliated with Shanghai Jiao Tong University School of Medicine, signaling the start of its commercial application and a new chapter for Chinese biopharma’s global ambitions.

🔬 Product Overview

GORINING® is indicated for long-term enzyme replacement therapy (ERT) in patients aged 12 and above with Type I and III Gaucher disease, a rare genetic lysosomal storage disorder.

• Active ingredient: Velaglucerase Alfa
• Mechanism of action: Replaces deficient β-glucocerebrosidase in the body, relieving symptoms such as hepatosplenomegaly, anemia, and bone pain
• Indication: Type I and III Gaucher disease
• Manufacturing: Developed and produced in China under international quality standards

💡 Key Advantages

• ✅ Significant Cost Advantage: Expected to be priced at least 50% lower than imported competitors, increasing affordability and accessibility
• ✅ Positive Reimbursement Outlook: The drug has entered China’s 2024 national insurance negotiation pipeline
• ✅ Comprehensive Patient Support: From diagnosis to long-term management, the company offers a complete service ecosystem
• ✅ Fast Clinical Adoption: Initial hospital prescriptions confirm strong market readiness

🌍 Global Expansion Underway

Alongside domestic launch, Beigene Kangcheng is actively advancing the global rollout of GORINING®. Initial overseas targets include Southeast Asia, the Middle East, and Latin America — regions where medical access to rare disease therapies remains limited. The company is accelerating cross-border registration, licensing, and partnership development.

🧬 About the Company

Beigene Kangcheng-B (HKEX: 01228) is a biotech company dedicated to rare disease treatment and drug innovation. With full capabilities from R&D and manufacturing to registration and commercialization, the company is committed to improving clinical access for underserved patients in China and beyond.

🤝 Global Partnership Opportunities

GORINING® is now open for international business collaboration, including:

• Licensing & distribution partners
• Regulatory consultants & market access providers
• Rare disease hospitals & treatment centers
• Global NGOs, health foundations, and procurement platforms

📧 Contact Email: globalfrontier1@proton.me
📱 WeChat: globalfrontier01
🌐 Website: www.globalfrontier1.com

Join us in bringing China’s biopharmaceutical innovation to the global stage.

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