两款抗体偶联药物获批

抗体偶联药物（ADC）将靶向特定抗原的单克隆抗体通过连接子与细胞毒性药物等载荷连接在一起，让载荷只能在表达特异性抗原的细胞中起作用。这一治疗模式的开发目标是将细胞毒性药物与靶向血液肿瘤或实体瘤的单克隆抗体连接在一起，精准靶向杀伤肿瘤细胞。

FDA在2025年上半年批准了两款ADC上市。今年FDA批准的首款新药是阿斯利康（AstraZeneca）与第一三共（Daiichi Sankyo）联合开发的抗体偶联药物Datroway（datopotamab deruxtecan），用于治疗无法切除或转移性激素受体（HR）阳性、人表皮生长因子受体2（HER2）阴性的成年乳腺癌患者，这些患者曾接受过内分泌疗法和化疗。Datroway是一款由人源化、靶向Trop2的单克隆抗体与创新DNA拓扑异构酶I抑制剂（DXd），通过可裂解的四肽连接子偶联生成的ADC。DXd具有独特的作用机制，与常见化疗药物伊立替康相比，活性提高10倍。而且此药物具有很强渗透细胞膜的能力，让它们在杀伤吞入ADC的癌细胞之后，能够杀死附近的癌细胞，产生“旁观者效应”（bystander effect）。

艾伯维（AbbVie）开发的ADC药物Emrelis（telisotuzumab vedotin）在今年5月获批上市，用于治疗具有高c-Met蛋白过度表达的局部晚期或转移性经治非鳞状非小细胞肺癌（NSCLC）成年患者。Emrelis是一款靶向c-Met的ADC，以微管蛋白抑制剂MMAE作为毒性有效载荷。c-Met是一种在包括NSCLC在内的多种肿瘤中过度表达的受体酪氨酸激酶。

抗体偶联药物是癌症治疗领域中发展最快的治疗模式之一。据统计，2024年全球启动了284项抗体偶联药物临床试验，比2023年增加了近100项，彰显了抗体偶联药物领域的迅猛增长。期待随着临床开发的推进，更多抗体偶联药物在未来获批上市。

创新不断，“first-in-class”药物占比50%

截至2025年6月27日，CDER已经批准了16款创新药，其中8款为“first-in-class”药物，占比50%，这些药物具有独特的作用机制。下面让我们来看一看这些“first-in-class”疗法能够让哪些患者获益。

Journavx（suzetrigine）：20多年来首款用于治疗急性疼痛的新机制药物

1月30日，FDA批准Vertex Pharmaceuticals公司开发的Journavx（suzetrigine）口服片剂上市，用于治疗成人中度至重度急性疼痛。Journavx是一种口服选择性NaV1.8抑制剂，通过选择性抑制NaV1.8，干预疼痛信号传导路径。与阿片类药物相比，该药物可能在提供更好镇痛效果的同时，避免上瘾等副作用。

Blujepa（gepotidacin）：近30年来首款用于治疗单纯性尿路感染的新机制口服抗生素

3月25日，FDA批准GSK公司开发的Blujepa（gepotidacin）上市，用于治疗单纯性尿路感染的成人及12岁以上青少年患者。Blujepa能够均衡抑制两种不同的II型拓扑异构酶，从而对大多数目标病原体，包括对现有抗生素耐药的菌株产生抗菌活性。由于能够均衡抑制两种酶的活性，细菌需要在两种酶上同时出现突变才能显著降低对Blujepa的敏感性，因此预期产生耐药性的可能性较低。它为克服抗微生物药物耐药性（AMR）提供了有力的新工具。

Qfitlia（fitusiran）：可治疗所有血友病类型的siRNA疗法

3月28日，FDA批准赛诺菲（Sanofi）和Alnylam联合开发的siRNA疗法Qfitlia（fitusiran）上市，用于预防或减少12岁以上血友病A和血友病B患者的出血事件，无论他们体内是否含有凝血因子VIII或IX的抑制物。Qfitlia通过降低抗凝血酶水平，从而促进凝血酶生成，重新平衡止血功能并预防出血。由于这一独特作用机制，它可以治疗多种不同类型的血友病，大大扩展了它的应用范围。在两项3期临床试验中，它只需每月一次皮下注射，就可将患者的年化出血率降低90%。在有些患者中，给药次数可降低到每两个月一次。

Imaavy（nipocalimab）：首款用于治疗全身性重症肌无力的新生儿Fc受体阻断剂

4月29日，FDA批准强生公司（Johnson & Johnson）开发的抗体疗法Imaavy（nipocalimab）上市，用于治疗抗AChR、抗MuSK抗体阳性的全身性重症肌无力（gMG）成人与12岁以上儿童患者。Imaavy通过结合新生儿Fc受体（FcRn），阻止被细胞摄入的自身抗体重新进入血液，从而在细胞内降解这些抗体。这款抗体疗法有望治疗多种自身抗体介导的免疫疾病。

Avmapki Fakzynja Co-pack（avutometinib与defactinib）：首款获批治疗KRAS突变型复发性低级别浆液性卵巢癌的组合疗法

5月8日，FDA批准Verastem Oncology公司开发的组合疗法Avmapki Fakzynja Co-pack（avutometinib与defactinib）上市，用于治疗复发性低级别浆液性卵巢癌（LGSOC）成年患者，这些患者曾接受过系统性治疗并且其肿瘤携带KRAS突变。Avutometinib是一种RAF/MEK抑制剂，它能够诱导MEK与ARAF、BRAF和CRAF形成非活性复合物，可能通过最大程度地抑制RAS信号通路产生更全面和持久的抗肿瘤反应。美国FDA曾授予avutometinib与FAK抑制剂defactinib联用，治疗所有复发的低级别浆液性卵巢癌患者（无论KRAS状态）的突破性疗法认定。

Emrelis（telisotuzumab vedotin）：获批治疗c-Met高表达经治晚期非小细胞肺癌患者的首款疗法

5月14日，艾伯维（AbbVie）宣布，美国FDA已加速批准抗体偶联药物Emrelis（telisotuzumab vedotin）上市，用于治疗具有高c-Met蛋白过度表达的局部晚期或转移性非鳞状NSCLC成年患者，这些患者此前已接受过系统性治疗。Emrelis是一款靶向c-Met的ADC，以微管蛋白抑制剂MMAE作为毒性有效载荷。c-Met是一种在包括NSCLC在内的多种肿瘤中过度表达的受体酪氨酸激酶。

Tryptyr（acoltremon）：一天即可起效的干眼症眼药水

5月28日，FDA批准爱尔康（Alcon）公司的眼药水Tryptyr（acoltremon）上市，用于治疗干眼症（DED）。Tryptyr是一款局部TRPM8激动剂。研究显示，TRPM8的激活可刺激三叉神经信号传导，从而促进天然泪液的分泌。在临床试验中，Tryptyr在使用第一天即可导致天然泪液分泌具有统计学显著性的增加。

Andembry（garadacimab）：预防遗传性血管性水肿发作的首款因子XIIa靶向疗法

6月16日，FDA批准CSL公司开发的Andembry（garadacimab）上市，用于预防成人和12岁及以上儿童患者遗传性血管性水肿（HAE）发作。Andembry是一种抗FXIIa的单克隆抗体，特异性抑制血浆蛋白FXIIa。当FXII被激活时，会启动导致水肿形成的级联反应。通过靶向FXIIa，Andembry能在反应最初阶段就阻断级联反应的信号传导。

结语

2025年上半年CDER批准的新药中“first-in-class”药物占比达50%，创新不断涌现。期待更多创新药物在2025年获批上市，造福全球患者。

展望未来，药明康德将继续秉持“让天下没有难做的药，难治的病”的愿景，依托全球研发基地与生产网络，以独特的一体化、端到端的CRDMO模式，提供高效、灵活的解决方案，持续赋能全球合作伙伴释放创新潜能，加速将科学突破转化成为新药、好药。

H1 2025 FDA Novel Drug Approvals Review: "First-in-Class" Therapies Make Up Half of All Approvals

As of June 27, the U.S. Food and Drug Administration (FDA)'s Center for Drug Evaluation and Research (CDER) had approved 16 novel drugs, with small molecule therapies accounting for approximately 60%. As an enabler of innovation, a trusted partner, and a contributor to the global pharmaceutical and life sciences industry, WuXi AppTec will continue to support its partners through its unique CRDMO business model—helping deliver breakthrough treatments to patients worldwide. This article highlights some of the key FDA-approved drugs in 2025, including antibody-drug conjugates (ADCs) and "first-in-class" drugs.

ADCs are a class of targeted therapies that link monoclonal antibodies directed against specific antigens to cytotoxic payloads via linkers. This design enables selective killing of cells expressing the target antigen. Developed for hematologic and solid tumors, ADCs offer precision in tumor targeting while minimizing off-target effects.

In the first half of 2025, the FDA approved two ADCs, both for oncology indications. The first was a Trop2-targeting ADC for adult patients with unresectable or metastatic HR-positive, HER2-negative breast cancer who had previously received endocrine therapy and chemotherapy. The second, approved in May, was a c-Met-targeting ADC that delivers MMAE, a microtubule inhibitor, as the cytotoxic payload. It represents the first approved therapy for patients with c-Met overexpressing non-small cell lung cancer (NSCLC) who have received a prior treatment.

ADCs remain one of the fastest-growing modalities in cancer treatment. In 2024 alone, 284 global ADC clinical trials were initiated—nearly 100 more than in 2023—demonstrating strong momentum in this space. Continued clinical advancement is expected to lead to additional approvals in the near future. 

Among the 16 novel drugs approved by CDER as of June 27, eight (50%) were classified as "first-in-class" therapies with novel mechanisms of action. These therapies offer new treatment options for patients with high unmet medical needs—some representing the first FDA-approved medicine for their respective indications.

One such therapy is an oral selective NaV1.8 inhibitor, the first non-opioid pain treatment of its kind. It offers effective pain relief while avoiding the addiction-related side effects associated with opioids. Another milestone is the approval of the first neonatal Fc receptor (FcRn) blocker for the treatment of generalized myasthenia gravis (gMG), expanding options for patients with antibody-positive disease.

For the c-Met overexpressing NSCLC patient population, the FDA approved the first targeted therapy. Similarly, the KRAS-mutant recurrent low-grade serous ovarian cancer (LGSOC) population now has its first FDA-approved combination therapy.

In the fight against antimicrobial resistance (AMR), the FDA approved a novel type II topoisomerase inhibitor—marking the first new oral antibiotic class in nearly three decades for the treatment of uncomplicated urinary tract infections.

Hemophilia patients also saw a breakthrough: the approval of the first siRNA therapy targeting antithrombin. By lowering antithrombin levels, the drug promotes thrombin generation and prevents bleeding. It is approved for both hemophilia A and B, regardless of whether patients have inhibitors to clotting factor VIII or IX. Importantly, it offers durable protection with just one subcutaneous injection per month.

The wave of “first-in-class” approvals in H1 2025 reflects the biopharmaceutical industry's ongoing innovation. More groundbreaking therapies are expected to receive regulatory approval in the remainder of the year—further improving outcomes for patients worldwide.

At WuXi AppTec, we remain committed to advancing transformative therapies for global patients. Together with our partners, we work toward a shared vision that “every drug can be made and every disease can be treated.”

参考资料：

[1] Novel Drug Approvals for 2025. Retrieved June 20, 2025, from https://www.fda.gov/drugs/novel-drug-approvals-fda/novel-drug-approvals-2025

[2] Global Oncology Trends 2025. Retrieved June 24, 2025, from https://www.iqvia.com/insights/the-iqvia-institute/reports-and-publications/reports/global-oncology-trends-2025