2025年FDA新藥盤(pán)點(diǎn)：“first-in-class”藥物占比過(guò)半

編者按：截至2025年12月19日，F(xiàn)DA旗下的藥物評(píng)價(jià)和研究中心（CDER）已經(jīng)批準(zhǔn)了44款創(chuàng)新藥，其中“first-in-class”藥物占比約55%，展現(xiàn)了新藥開(kāi)發(fā)領(lǐng)域的不斷創(chuàng)新。小分子藥物占比66%，并有多款新藥靶向全新蛋白靶點(diǎn)。4款多肽和核酸類藥物獲批上市，顯示這些治療模式成為新藥開(kāi)發(fā)的重要方向之一。作為創(chuàng)新的賦能者、客戶信賴的合作伙伴以及全球健康產(chǎn)業(yè)的貢獻(xiàn)者，藥明康德將持續(xù)通過(guò)獨(dú)特的“CRDMO”業(yè)務(wù)模式，助力更多合作伙伴，為全球病患帶來(lái)突破性創(chuàng)新療法。本文將回顧在2025年獲得FDA批準(zhǔn)新藥的亮點(diǎn)。

創(chuàng)新不斷，“first-in-class”藥物占比約55%

截至12月19日，在2025年獲批的新藥中，具有獨(dú)特作用機(jī)制的“first-in-class”療法占比約55%（定義為FDA或開(kāi)發(fā)公司官方新聞稿中描述為“first-in-class”的藥物或針對(duì)特定適應(yīng)癥獲批的首款藥物），顯示了新藥開(kāi)發(fā)領(lǐng)域的不斷創(chuàng)新。其中，8款藥物同時(shí)獲得突破性療法認(rèn)定。這意味著它們不僅具有獨(dú)特的作用機(jī)制，而且與以往療法相比能為患者帶來(lái)顯著的療效改善。

▲獲得FDA突破性療法認(rèn)定的“first-in-class”藥物（數(shù)據(jù)截至2025年12月19日）

小分子藥物占比66%，多個(gè)創(chuàng)新靶點(diǎn)被攻克

在2025年CDER批準(zhǔn)的新藥中，小分子藥物占比高達(dá)66%，顯示了這一治療模式旺盛的生命力。其中，4款新藥靶向的此前未被其他獲批藥物針對(duì)的創(chuàng)新靶點(diǎn)。例如愛(ài)爾康（Alcon）公司開(kāi)發(fā)的眼藥水Tryptyr（acoltremon）通過(guò)激活全新靶點(diǎn)TRPM8刺激三叉神經(jīng)信號(hào)傳導(dǎo)，從而促進(jìn)天然淚液的分泌。它在2025年5月獲批治療干眼癥。

Insmed公司開(kāi)發(fā)的Brinsupri（brensocatib）是一款靶向創(chuàng)新靶點(diǎn)二肽基肽酶1（DPP1）的“first-in-class”抑制劑。它在2025年8月獲批用于治療12歲及以上成人及兒童的非囊性纖維化支氣管擴(kuò)張癥（NCFB）。

Jazz Pharmaceuticals開(kāi)發(fā)的Modeyso（dordaviprone）在2025年8月獲FDA批準(zhǔn)，用于治療1歲及以上、攜帶

H3 K27M

由Vertex Pharmaceuticals公司開(kāi)發(fā)的Journavx（suzetrigine）是一種口服選擇性NaV1.8抑制劑，通過(guò)別構(gòu)抑制的機(jī)制達(dá)到對(duì)NaV1.8的高度選擇性，它對(duì)NaV1.8離子通道的選擇性與其他NaV相比達(dá)到了3萬(wàn)到4萬(wàn)倍。在2025年1月Journavx獲得FDA批準(zhǔn)治療中度或重度急性疼痛。

圖片來(lái)源：123RF

別構(gòu)抑制的機(jī)制在其他FDA獲批新藥中也得到應(yīng)用。例如SpringWorks Therapeutics公司開(kāi)發(fā)的別構(gòu)MEK1和MEK2抑制劑Gomekli（mirdametinib）在2025年2月獲批上市，用于治療年齡不低于2歲的1型神經(jīng)纖維瘤病相關(guān)叢狀神經(jīng)纖維瘤（NF1-PN）成人和兒童患者。Cytokinetics公司開(kāi)發(fā)的Myqorzo（aficamten）是一款別構(gòu)心肌肌球蛋白抑制劑，它在12月獲批治療有癥狀的梗阻性肥厚型心肌?。╫HCM）成人患者。

別構(gòu)藥物之外，多款共價(jià)藥物也獲得FDA批準(zhǔn)。共價(jià)藥物通過(guò)與靶點(diǎn)形成穩(wěn)定的化學(xué)鍵，實(shí)現(xiàn)持久的抑制效果。憑借這一獨(dú)特機(jī)制，它們不僅能夠以更低的劑量給藥，還可顯著提升靶點(diǎn)占有率，展現(xiàn)出廣闊臨床應(yīng)用前景。2025年，F(xiàn)DA批準(zhǔn)的共價(jià)藥物包括EGFR抑制劑Zegfrovy（sunvozertinib），HER2抑制劑Hernexeos（zongertinib）和BTK抑制劑Wayrilz（rilzabrutinib）與Rhapsido（remibrutinib）。它們分別為攜帶

EGFR

▲2025年CDER批準(zhǔn)的新藥中，小分子藥物占比約66%（數(shù)據(jù)截至2025年12月19日）

4款多肽和核酸類藥物獲批，2款A(yù)DC獲批

在傳統(tǒng)小分子和抗體藥物之外，多款具有創(chuàng)新治療模式的新藥獲得FDA批準(zhǔn)。隨著多肽和核酸類藥物開(kāi)發(fā)技術(shù)的日漸成熟，F(xiàn)DA近年來(lái)平均每年批準(zhǔn)4款這類新藥。2025年共有3款“first-in-class”寡核苷酸療法獲批上市。其中，siRNA療法Qfitlia（fitusiran）通過(guò)抑制肝臟中抗凝血酶的生產(chǎn)，有望成為一種革命性的預(yù)防手段，造福所有血友病患者。反義寡核苷酸療法Dawnzera（donidalorsen）是用于預(yù)防遺傳性血管性水腫（HAE）發(fā)作的RNA靶向藥物。Redemplo（plozasiran）則是FDA批準(zhǔn)的用于降低家族性乳糜微粒血癥綜合征（FCS）成人患者甘油三酯的siRNA療法。

在多肽藥物領(lǐng)域，F(xiàn)DA加速批準(zhǔn)“first-in-class”藥物Forzinity（elamipretide）上市，為Barth綜合征患者帶來(lái)了獲批療法。

抗體偶聯(lián)藥物（ADC）是癌癥治療領(lǐng)域中發(fā)展最快的治療模式之一。據(jù)統(tǒng)計(jì)，2024年全球啟動(dòng)了284項(xiàng)抗體偶聯(lián)藥物臨床試驗(yàn)，比2023年增加了近100項(xiàng)，彰顯了抗體偶聯(lián)藥物領(lǐng)域的迅猛增長(zhǎng)。2025年，F(xiàn)DA批準(zhǔn)了兩款A(yù)DC，其中Datroway（datopotamab deruxtecan）是一款由人源化、靶向Trop2的單克隆抗體，與創(chuàng)新DNA拓?fù)洚悩?gòu)酶I抑制劑（DXd）通過(guò)可裂解的四肽連接子偶聯(lián)生成的ADC。它在2025年年初獲批治療無(wú)法切除或轉(zhuǎn)移性激素受體（HR）陽(yáng)性、人表皮生長(zhǎng)因子受體2（HER2）陰性的成年乳腺癌患者。

靶向c-Met的ADC藥物Emrelis（telisotuzumab vedotin）在2025年5月獲批上市，用于治療具有高c-Met蛋白過(guò)度表達(dá)的局部晚期或轉(zhuǎn)移性經(jīng)治非鱗狀非小細(xì)胞肺癌（NSCLC）成年患者。

2025年FDA批準(zhǔn)的潛在重磅療法（按商品名首字母排序，根據(jù)行業(yè)媒體Evaluate發(fā)表的潛在重磅藥物榜單）

Brinsupri（brensocatib）

Brinsupri是一款“first-in-class”二肽基肽酶1抑制劑，旨在抑制中性粒細(xì)胞內(nèi)可驅(qū)動(dòng)NCFB慢性氣道炎癥的中性粒細(xì)胞絲氨酸蛋白酶的活化。它在2025年8月獲批治療12歲及以上成人及兒童的非囊性纖維化支氣管擴(kuò)張癥。

Datroway（datopotamab deruxtecan）

Datroway是一款由人源化、靶向Trop2的單克隆抗體與創(chuàng)新DNA拓?fù)洚悩?gòu)酶I抑制劑（DXd）連接的ADC。DXd具有獨(dú)特的作用機(jī)制，與常見(jiàn)化療藥物伊立替康（irinotecan）相比，活性提高10倍。而且此藥物具有很強(qiáng)滲透細(xì)胞膜的能力，讓它們?cè)跉倘階DC的癌細(xì)胞之后，能夠殺死附近的癌細(xì)胞，產(chǎn)生“旁觀者效應(yīng)”（bystander effect）。它在2025年年初獲批治療無(wú)法切除或轉(zhuǎn)移性HR陽(yáng)性、HER2陰性的成年乳腺癌患者。

Exdensur（depemokimab）

Exdensur是一款靶向IL-5的超長(zhǎng)效生物制品。它能夠與IL-5以高親和力結(jié)合，以每6個(gè)月給藥一次的頻率用以治療重度哮喘患者。IL-5是2型炎癥中的關(guān)鍵細(xì)胞因子之一。超過(guò)80%的嚴(yán)重哮喘患者的病情是由2型炎癥引起的。它在2025年12月獲得FDA批準(zhǔn)，作為附加維持治療，用于治療以嗜酸性粒細(xì)胞表型為特征的重度哮喘，適用于12歲及以上的成人和青少年患者。

Imaavy（nipocalimab）

Imaavy是一款潛在“best-in-class”、靶向新生兒Fc受體的抗體療法。它通過(guò)與FcRn結(jié)合，讓被單核細(xì)胞和內(nèi)皮細(xì)胞攝入的自身抗體不會(huì)被重新釋放到血液中，而是在細(xì)胞內(nèi)被降解。這款抗體療法有望治療多種自身抗體介導(dǎo)的免疫疾病。它在2025年獲得批準(zhǔn)，用于治療抗AChR、抗MuSK抗體陽(yáng)性的全身性重癥肌無(wú)力（gMG）成人與12歲以上兒童患者。

Journavx（suzetrigine）

Journavx是一種口服選擇性NaV1.8抑制劑，與其他NaV離子通道相比，它對(duì)NaV1.8具有高度選擇性。與阿片類藥物相比，該藥物可能在提供更好鎮(zhèn)痛效果的同時(shí)，避免上癮等副作用。根據(jù)FDA新聞稿，Journavx是FDA所批準(zhǔn)的基于新機(jī)制的非阿片類止痛藥物，標(biāo)志著疼痛管理領(lǐng)域進(jìn)入一個(gè)新的治療時(shí)代。

Keytruda Qlex（pembrolizumab和berahyaluronidase alfa）

Keytruda Qlex是Keytruda皮下注射制劑，是一款將Keytruda和由Alteogen公司所開(kāi)發(fā)的透明質(zhì)酸酶變體berahyaluronidase alfa結(jié)合的配方，旨在增加藥物使用的便捷性。它在2025年9月獲得FDA批準(zhǔn)，用于成人及兒童（12歲及以上）人群，所涵蓋的實(shí)體瘤適應(yīng)癥與Keytruda靜脈輸注制劑已獲批的適應(yīng)癥一致。此前研究顯示，該劑型的中位注射時(shí)間僅需約2分鐘。

Myqorzo（aficamten）

Myqorzo是一種別構(gòu)小分子心肌肌球蛋白抑制劑。它通過(guò)直接抑制產(chǎn)力橫橋的形成，減輕心肌過(guò)度收縮。它在2025年12月獲得FDA批準(zhǔn)，用于治療有癥狀的梗阻性肥厚型心肌?。╫HCM）成人患者。3期臨床試驗(yàn)SEQUOIA-HCM結(jié)果顯示，與安慰劑相比，Myqorzo治療24周顯著改善了患者的運(yùn)動(dòng)能力。

結(jié)語(yǔ)

2025年CDER新藥批準(zhǔn)總數(shù)維持在較高水平，“first-in-class”藥物占比約55%，4款多肽和核酸類藥物獲批，創(chuàng)新不斷涌現(xiàn)。

展望未來(lái)，藥明康德將繼續(xù)秉持“讓天下沒(méi)有難做的藥，難治的病”的愿景，依托全球研發(fā)基地與生產(chǎn)網(wǎng)絡(luò)，以獨(dú)特的一體化、端到端的CRDMO模式，提供高效、靈活的解決方案，持續(xù)賦能全球合作伙伴釋放創(chuàng)新潛能，加速將科學(xué)突破轉(zhuǎn)化成為新藥、好藥。

附錄：FDA旗下CDER2025年獲批療法列表（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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