2025年，FDA共批准44款新药，治疗多种癌症、高血脂、老花眼、湿疹等疾病

　　编者按：截至2025年12月19日，FDA旗下的药物评价和研究中心（CDER）已经批准了44款创新药，其中“first-in-class”药物占比约55%，展现了新药开发领域的不断创新。小分子药物占比66%，并有多款新药靶向全新蛋白靶点。4款多肽和核酸类药物获批上市，显示这些治疗模式成为新药开发的重要方向之一。作为创新的赋能者、客户信赖的合作伙伴以及全球健康产业的贡献者，药明康德将持续通过独特的“CRDMO”业务模式，助力更多合作伙伴，为全球病患带来突破性创新疗法。本文将回顾在2025年获得FDA批准新药的亮点。

　　创新不断，“first-in-class”药物占比约55%

　　截至12月19日，在2025年获批的新药中，具有独特作用机制的“first-in-class”疗法占比约55%（定义为FDA或开发公司官方新闻稿中描述为“first-in-class”的药物或针对特定适应症获批的首款药物），显示了新药开发领域的不断创新。其中，8款药物同时获得突破性疗法认定。这意味着它们不仅具有独特的作用机制，而且与以往疗法相比能为患者带来显著的疗效改善。

　　▲获得FDA突破性疗法认定的“first-in-class”药物（数据截至2025年12月19日）

　　小分子药物占比66%，多个创新靶点被攻克

　　在2025年CDER批准的新药中，小分子药物占比高达66%，显示了这一治疗模式旺盛的生命力。其中，4款新药靶向的此前未被其他获批药物针对的创新靶点。例如爱尔康（Alcon）公司开发的眼药水Tryptyr（acoltremon）通过激活全新靶点TRPM8刺激三叉神经信号传导，从而促进天然泪液的分泌。它在今年5月获批治疗干眼症。

　　Insmed公司开发的Brinsupri（brensocatib）是一款靶向创新靶点二肽基肽酶1（DPP1）的“first-in-class”抑制剂。它在今年8月获批用于治疗12岁及以上成人及儿童的非囊性纤维化支气管扩张症（NCFB）。

　　Jazz Pharmaceuticals开发的Modeyso（dordaviprone）在今年8月获FDA批准，用于治疗1岁及以上、携带H3 K27M突变且在既往治疗后疾病进展的弥漫性中线胶质瘤成人和儿童患者。这款药物除了可以抑制多巴胺受体，减弱多巴胺受体介导的RAS信号通路的激活，还可以过度激活创新靶点ClpP，导致线粒体蛋白质的选择性降解，让癌细胞因能量供应短缺而死亡。

　　由Vertex Pharmaceuticals公司开发的Journavx（suzetrigine）是一种口服选择性NaV1.8抑制剂，通过别构抑制的机制达到对NaV1.8的高度选择性，它对NaV1.8离子通道的选择性与其他NaV相比达到了3万到4万倍。在今年1月Journavx获得FDA批准治疗中度或重度急性疼痛。

　　图片来源：123RF

　　别构抑制的机制在其他FDA获批新药中也得到应用。例如SpringWorks Therapeutics公司开发的别构MEK1和MEK2抑制剂Gomekli（mirdametinib）在今年2月获批上市，用于治疗年龄不低于2岁的1型神经纤维瘤病相关丛状神经纤维瘤（NF1-PN）成人和儿童患者。Cytokinetics公司开发的Myqorzo（aficamten）是一款别构心肌肌球蛋白抑制剂，它在12月获批治疗有症状的梗阻性肥厚型心肌病（oHCM）成人患者。

　　别构药物之外，多款共价药物也获得FDA批准。共价药物通过与靶点形成稳定的化学键，实现持久的抑制效果。凭借这一独特机制，它们不仅能够以更低的剂量给药，还可显著提升靶点占有率，展现出广阔临床应用前景。2025年，FDA批准的共价药物包括EGFR抑制剂Zegfrovy（sunvozertinib），HER2抑制剂Hernexeos（zongertinib）和BTK抑制剂Wayrilz（rilzabrutinib）与Rhapsido（remibrutinib）。它们分别为携带

EGFR

　　▲2025年CDER批准的新药中，小分子药物占比约66%（数据截至2025年12月19日）

　　4款多肽和核酸类药物获批，2款ADC获批

　　在传统小分子和抗体药物之外，多款具有创新治疗模式的新药获得FDA批准。随着多肽和核酸类药物开发技术的日渐成熟，FDA近年来平均每年批准4款这类新药。2025年共有3款“first-in-class”寡核苷酸疗法获批上市。其中，siRNA疗法Qfitlia（fitusiran）通过抑制肝脏中抗凝血酶的生产，有望成为一种革命性的预防手段，造福所有血友病患者。反义寡核苷酸疗法Dawnzera（donidalorsen）是用于预防遗传性血管性水肿（HAE）发作的RNA靶向药物。Redemplo（plozasiran）则是FDA批准的用于降低家族性乳糜微粒血症综合征（FCS）成人患者甘油三酯的siRNA疗法。

　　在多肽药物领域，FDA加速批准“first-in-class”药物Forzinity（elamipretide）上市，为Barth综合征患者带来了获批疗法。

　　抗体偶联药物（ADC）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。2025年，FDA批准了两款ADC，其中Datroway（datopotamab deruxtecan）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。它在今年年初获批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。

　　靶向c-Met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。

　　2025年FDA批准的潜在重磅疗法（按商品名首字母排序，根据行业媒体Evaluate发表的潜在重磅药物榜单）

　　Brinsupri（brensocatib）

　　Brinsupri是一款“first-in-class”二肽基肽酶1抑制剂，旨在抑制中性粒细胞内可驱动NCFB慢性气道炎症的中性粒细胞丝氨酸蛋白酶的活化。它在今年8月获批治疗12岁及以上成人及儿童的非囊性纤维化支气管扩张症。

　　Datroway（datopotamab deruxtecan）

　　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）连接的ADC。DXd具有独特的作用机制，与常见化疗药物伊立替康（irinotecan）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）。它在今年年初获批治疗无法切除或转移性HR阳性、HER2阴性的成年乳腺癌患者。

　　Exdensur（depemokimab）

　　Exdensur是一款靶向IL-5的超长效生物制品。它能够与IL-5以高亲和力结合，以每6个月给药一次的频率用以治疗重度哮喘患者。IL-5是2型炎症中的关键细胞因子之一。超过80%的严重哮喘患者的病情是由2型炎症引起的。它在今年12月获得FDA批准，作为附加维持治疗，用于治疗以嗜酸性粒细胞表型为特征的重度哮喘，适用于12岁及以上的成人和青少年患者。

　　Imaavy（nipocalimab）

　　Imaavy是一款潜在“best-in-class”、靶向新生儿Fc受体的抗体疗法。它通过与FcRn结合，让被单核细胞和内皮细胞摄入的自身抗体不会被重新释放到血液中，而是在细胞内被降解。这款抗体疗法有望治疗多种自身抗体介导的免疫疾病。它在今年获得批准，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。

　　Journavx（suzetrigine）

　　Journavx是一种口服选择性NaV1.8抑制剂，与其他NaV离子通道相比，它对NaV1.8具有高度选择性。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。根据FDA新闻稿，Journavx是FDA所批准的基于新机制的非阿片类止痛药物，标志着疼痛管理领域进入一个新的治疗时代。

　　Keytruda Qlex（pembrolizumab和berahyaluronidase alfa）

　　Keytruda Qlex是Keytruda皮下注射制剂，是一款将Keytruda和由Alteogen公司所开发的透明质酸酶变体berahyaluronidase alfa结合的配方，旨在增加药物使用的便捷性。它在今年9月获得FDA批准，用于成人及儿童（12岁及以上）人群，所涵盖的实体瘤适应症与Keytruda静脉输注制剂已获批的适应症一致。此前研究显示，该剂型的中位注射时间仅需约2分钟。

　　Myqorzo（aficamten）

　　Myqorzo是一种别构小分子心肌肌球蛋白抑制剂。它通过直接抑制产力横桥的形成，减轻心肌过度收缩。它在今年12月获得FDA批准，用于治疗有症状的梗阻性肥厚型心肌病（oHCM）成人患者。3期临床试验SEQUOIA-HCM结果显示，与安慰剂相比，Myqorzo治疗24周显著改善了患者的运动能力。

　　图片来源：123RF

　　结语

　　2025年CDER新药批准总数维持在较高水平，“first-in-class”药物占比约55%，4款多肽和核酸类药物获批，创新不断涌现。

　　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。

　　附录：FDA旗下CDER今年获批疗法列表（截至12月19日）

　　Review of 2025 FDA Novel Drug Approvals: First-in-Class Therapies Make Up 55% of All Approvals

　　As of December 19, 2025,the FDA’s Center for Drug Evaluation and Research (CDER) has approved 44 innovative drugs, of which approximately 55% are first-in-class, reflecting continued innovation in drug development. 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 key new drugs approved by the FDA in 2025.

　　Continuous Innovation: First-in-Class Therapies Account for Approximately 55%

　　Among new drugs approved in 2025, first-in-class therapies with unique mechanisms of action accounted for nearly 55%, demonstrating ongoing innovation in drug R&D. Notably,8 of these drugs also received Breakthrough Therapy Designation, indicating not only novel mechanisms but also significant clinical benefit compared to existing therapies.

　　▲Percentage of first-in-class drugs in CDER approval from 2021-2025 (Data as of December 19, 2025)

　　Small-Molecule Drugs Account for 66%, with Multiple Innovative Targets Tackled

　　Among the new drugs approved by CDER in 2025,small-molecule therapies accounted for as much as 66%, highlighting the continued strength and vitality of this modality in modern drug development.Notably, four of these newly approved agents target innovative molecular mechanisms that had not previously been addressed by approved therapies, underscoring the industry’s ongoing progress in expanding the druggable target space.

　　One example is Tryptyr (acoltremon), an ophthalmic solution developed by Alcon. By activating a novel target TRPM8 and stimulating trigeminal nerve signal transmission, Tryptyr promotes natural tear secretion. The drug was approved in May this year for the treatment of dry eye disease.

　　Another notable approval is Brinsupri (brensocatib), developed by Insmed. Brinsupri is a first-in-class inhibitor targeting the innovative target dipeptidyl peptidase 1 (DPP1). In August, it received FDA approval for the treatment of non-cystic fibrosis bronchiectasis (NCFB) in adults and children aged 12 years and older.

　　In the field of oncology, Modeyso (dordaviprone), developed by Jazz Pharmaceuticals, was approved by the FDA in August for the treatment of adults and children aged 1 year and older with diffuse midline glioma harboring the H3 K27M mutation whose disease has progressed following prior therapy. Mechanistically, Modeyso exerts a dual mode of action: in addition to inhibiting dopamine receptors and attenuating dopamine receptor–mediated activation of the RAS signaling pathway, it hyperactivates the novel target ClpP. This leads to selective degradation of mitochondrial proteins, ultimately causing cancer cell death due to insufficient energy supply.

　　Advances in target selectivity were also evident in pain management.Journavx (suzetrigine), developed by Vertex Pharmaceuticals, is an oral selective NaV1.8 inhibitor that achieves its high specificity through an allosteric inhibition mechanism. Its selectivity for the NaV1.8 ion channel is approximately 30,000- to 40,000-fold greater than that for other NaV channels. In January this year, Journavx received FDA approval for the treatment of moderate to severe acute pain.

　　The application of allosteric inhibition extends beyond pain therapeutics.For example, Gomekli (mirdametinib), an allosteric MEK1 and MEK2 inhibitor developed by SpringWorks Therapeutics, was approved in February for the treatment of adults and children aged 2 years and older with neurofibromatosis type 1–associated plexiform neurofibromas (NF1-PN).

　　In parallel with allosteric approaches, covalent inhibition continued to gain regulatory traction in 2025.Covalent drugs achieve durable pharmacological effects by forming stable chemical bonds with their targets. This mechanism enables sustained target inhibition, often at lower doses, while significantly increasing target occupancy—features that collectively translate into meaningful clinical advantages. During the year, the FDA approved several covalent small-molecule therapies, including the EGFR inhibitor Zegfrovy (sunvozertinib), the HER2 inhibitor Hernexeos (zongertinib), and the BTK inhibitor Rhapsido (remibrutinib). These agents have expanded treatment options for adult patients with non-small cell lung cancer (NSCLC) harboring

EGFR

　　4 Peptide and Nucleic Acid Drugs Approved, 2 ADCs Gained Approval

　　Beyond traditional small molecules and antibody drugs, several innovative modalities were approved by the FDA. As peptide and nucleic acid drug development technologies continue to mature, the FDA approves about 4 new drugs in these modalities in recent years.

　　In 2025, three first-in-class oligonucleotide therapies were approved:

　　• Qfitlia (fitusiran), an siRNA therapy, prevents bleeding by inhibiting antithrombin production in the liver, offering a revolutionary prevention strategy for all hemophilia patients.

　　• Dawnzera (donidalorsen) is an RNA-targeted therapy to prevent hereditary angioedema (HAE) attacks.

　　• Redemplo (plozasiran) is an siRNA therapy to lower triglycerides in adults with familial chylomicronemia syndrome (FCS).

　　In the peptide drug category, the FDA granted accelerated approval to Forzinity (elamipretide), bringing a much-needed approved therapy to patients with Barth syndrome.

　　Antibody-drug conjugates (ADCs) remain one of the fastest-growing modalities in oncology.In 2024, 284 ADC clinical trials were initiated globally, nearly 100 more than in 2023, demonstrating accelerated growth.

　　In 2025, the FDA approved two ADCs:

　　• Datroway (datopotamab deruxtecan), an ADC composed of a humanized Trop2-targeting monoclonal antibody conjugated with a novel DNA topoisomerase I inhibitor (DXd) via a cleavable tetrapeptide linker, was approved early this year for adults with unresectable or metastatic hormone receptor (HR)-positive, HER2-negative breast cancer.

　　• Emrelis (telisotuzumab vedotin), targeting c-Met, was approved in May for adults with locally advanced or metastatic, previously treated non-squamous NSCLC with high c-Met overexpression.

　　Potential Blockbuster Therapies Approved by FDA in 2025 (Alphabetical by Brand Name, based on potential blockbuster drug lists published byEvaluate)

　　Brinsupri（brensocatib）

　　Brinsupri is a first-in-class DPP1 inhibitor designed to suppress the activation of neutrophil serine proteases within neutrophils that drive chronic NCFB airway inflammation. It was approved in August of this year for the treatment of non–cystic fibrosis bronchiectasis in adults and pediatric patients aged 12 years and older.

　　Datroway (datopotamab deruxtecan)

　　Datroway is an ADC made from a Trop2-targeting humanized monoclonal antibody linked with a novel DXd compound. DXd has a 10-fold higher activity than irinotecan and shows strong membrane permeability, enabling cancer cell killing after internalization and a bystander effect that also kills neighboring tumor cells.

　　Exdensur（depemokimab）

　　Exdensur is an ultra-long-acting biologic that targets IL-5. It binds to IL-5 with high affinity and is administered once every six months for the treatment of patients with severe asthma. IL-5 is one of the key cytokines involved in type 2 inflammation. More than 80% of patients with severe asthma have disease driven by type 2 inflammation. In December of this year, Exdensur received FDA approval as an add-on maintenance therapy for the treatment of severe asthma characterized by an eosinophilic phenotype in adults and adolescents aged 12 years and older.

　　Imaavy (nipocalimab)

　　Imaavy is a potential best-in-class antibody therapy targeting neonatal Fc receptor (FcRn). By binding FcRn, it prevents recycled autoantibodies from being released back into circulation and instead directs them for intracellular degradation. Approved this year, it treats adult and pediatric (≥12 years) patients with generalized myasthenia gravis (gMG) who are AChR- or MuSK-antibody positive. According to press releases, it is the first FcRn blocker approved for this patient group.

　　Journavx (suzetrigine)

　　Journavx is an oral selective NaV1.8 inhibitor. Compared with opioids, it may provide improved analgesia while avoiding addiction and other side effects. According to the FDA press release, Journavx is the first non-opioid analgesic approved based on a new mechanism, marking the start of a new era in pain management.

　　Keytruda Qlex (pembrolizumab and berahyaluronidase alfa)

　　Keytruda Qlex is the subcutaneous formulation of Keytruda, combining Keytruda with berahyaluronidase alfa (ALT-B4) developed by Alteogen to improve administration convenience. Approved in September, it covers the same solid tumor indications as the IV formulation in adults and pediatric patients ≥12 years. Studies show a median injection time of only ~2 minutes.

　　Myqorzo（aficamten）

　　Myqorzo is an allosteric small-molecule cardiac myosin inhibitor. It reduces myocardial hypercontractility by directly inhibiting the formation of force-generating cross-bridges. In December of this year, it received FDA approval for the treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM). Results from the Phase 3 SEQUOIA-HCM clinical trial showed that, compared with placebo, treatment with Myqorzo for 24 weeks significantly improved patients’ exercise capacity.

　　In 2025, CDER new drug approvals remained high, with first-in-class therapies about 55%. 4 peptide and nucleic acid drugs and 2 ADCs were approved, reflecting sustained innovation in new modalities.

　　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.”

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