Atacicept

2025年对于美国FDA来说是极具挑战的一年。在特朗普政府任命Robert F. Kennedy Jr.领导美国卫生与公众服务部后，机构内部经历了大幅人事变动和政策调整，超过18%的CDER和CBER员工离职或被解雇，CDER甚至一年内更换了五位主任。尽管如此，FDA仍然批准了46款新治疗药物，保持了近年来较高的审批效率，最近的Nature子刊新闻即对此进行了全面报道。 01 在动荡中前行 2025年，FDA批准了46个新的治疗性药物，这一数字虽比近5年平均48个略有下降，但仍远高于自1993年以来的历史平均值36个（如图1所示）。这一数据清晰地表明，生物医药创新正以前所未有的速度推进，即使在FDA经历动荡之年的情况下，药物研发管线依然保持强劲活力。 ▲ 图1 02 肿瘤主导但非肿瘤领域亮点频现 从治疗领域来看（图2），癌症继续占据主导地位，35%（16个）的新批准药物针对各类肿瘤，较过去5年平均29%有所上升。这反映了肿瘤药物研发的活跃度和市场关注度。然而，值得注意的是，非肿瘤领域同样取得了突破性进展： ▲ 图2 肿瘤学(35%)、心脏病学(11%)和过敏/炎症疾病(9%)是2025年最活跃的三大治疗领域。特别引人注目的是，传统上以肿瘤为主的激酶抑制剂开始向更广泛的治疗领域拓展，如Novartis的remibrutinib(Rhapsido)获批用于慢性自发性荨麻疹，而不仅是血液癌症。 03 药物模态创新 图3揭示了2025年药物模态的分布特点，其中激酶抑制剂成为最大赢家，约占新批准小分子的三分之一。前面提到的Novartis的remibrutinib成为了第100个获FDA批准的激酶抑制剂，标志着这一药物类别从纯粹的抗癌工具转变为更广泛的治疗手段。 ▲ 图3 图3清晰展示了各类药物模态的占比情况。小分子药物(主要是激酶抑制剂)占据主导，但抗体-药物偶联物(ADC)、双特异性抗体等新型生物制剂增长显著。特别值得注意的是，adnectin技术首次获得批准，代表了生物制剂设计的新方向。 LIB Therapeutics的lerodalcibep(Lerochol)作为首个adnectin-based疗法获批，标志着这一技术平台的成熟。Adnectins利用纤连蛋白III结构域构建抗体模拟物，相比传统单抗具有更小、更稳定、室温保存等优势，可能解决PCSK9抑制剂市场因给药不便和成本高昂而受限的问题。 04 关于突破性疗法 2025年批准的药物中有多个"first-in-class"产品，针对严重疾病提供了全新治疗选择： 基因疗法里程碑：CBER批准了首个来自非营利组织的基因疗法etuvetidigene autotemcel(Waskyra)，用于治疗Wiskott-Aldrich综合征。这种ex vivo基因疗法使用慢病毒载体将正常WAS基因导入患者造血干细胞，恢复血小板功能。这一批准标志着非营利组织也能成功推动基因疗法上市，为罕见病治疗开辟新路径。 新型抗生素突破：GSK的gepotidacin(Blujepa)和Innoviva的zoliflodacin(Nuzolvence)分别获批用于尿路感染和淋病。它们属于新型拓扑异构酶II抑制剂，但采用不同化学骨架，规避了现有抗生素的耐药性问题。特别值得注意的是，zoliflodacin由全球抗生素研发伙伴关系(GARDP)资助开发，旨在确保全球可及性。 罕见病治疗进展：针对遗传性血管性水肿(HAE)，2025年一次批准了三种新疗法：garadacimab(Andembry)靶向激活因子XII，donidalorsen(Dawnzera)靶向前激肽释放酶，sebetralstat(Ekterly)则用于急性发作治疗。这些药物针对同一疾病的不同病理环节，为患者提供了更全面的治疗选择。 05 监管之变 如开篇所述，2025年对FDA而言是动荡的一年。尽管如此，FDA仍推出了多项影响深远的监管改革： CNPV试点计划：FDA推出"Commissioner's National Priority Voucher"(CNPV)项目，将审查周期从常规的10-12个月大幅缩短至2个月。虽然这一举措提高了审批效率，但引发了关于"政治化药物审查过程"和"降低监管标准"的担忧。 单一临床试验路径：FDA正考虑将默认要求从两个关键性临床试验改为一个，这一变化将显著加速药物审批，但也引发对证据充分性的质疑。 动物测试替代方案：FDA宣布计划逐步淘汰动物毒性测试，转向更现代的替代方法，这符合3R原则（减少、优化、替代动物实验）的全球趋势。 CRL透明度提升：FDA开始发布部分去标识化的完整响应信(CRL)，使被拒药物原因更加透明。例如Scholar Rock的apitegromab因第三方灌装设施问题被拒，Stealth Bio的elamipretide因有效性证据不足被拒但后获批准。  06 2026年上市预测 波士顿咨询集团分析显示，2025年获批新药的平均峰值销售额约为12亿美元（中位数6亿美元）。2026年，更多创新疗法有望登场（表4）： 靶向蛋白降解剂首秀： Arvinas/辉瑞的雌激素受体（ER）靶向PROTAC降解剂Vepdegestrant有望成为首个获批的靶向蛋白降解剂，验证这一解锁“不可成药”靶点的新技术。 突破血脑屏障： Denali的Tividenofusp alfa（酶替代疗法）和Regenxbio的Clemidsogene lanparvovec（基因疗法）均靶向亨特综合征，前者利用Fc片段结合转铁蛋白受体将酶递送入脑，后者直接向中枢神经系统注射AAV载体，旨在解决现有疗法无法改善神经症状的痛点。 代谢疾病巨头入场： 诺和诺德的CagriSema（GLP-1/胰淀素类似物组合）和礼来的Orforglipron（口服小分子GLP-1受体激动剂）将加入竞争激烈的减肥药市场。默沙东的口服PCSK9抑制剂Enlicitide（已获CNPV）有望冲击降脂药格局。 其他焦点： Replimune的溶瘤病毒Vusolimogene oderparepvec（重新提交）、Vera的Atacicept（BLyS/APRIL抑制剂治疗IgAN）、阿斯利康的Camizestrant（新一代SERD）、诺和诺德的Denecimig（FIXa×FX双抗用于血友病A）等也备受期待。 扫码关注我们 长按扫描二维码     关注我们获取更多行业资讯！ #FDA#CBER#CDER#基因治疗#GSK#诺和诺德#诺华#ADC#CART

FDA assigned PDUFA target action date of July 7, 2026. If approved, atacicept would be the first B cell modulator targeting both BAFF and APRIL for IgAN. Atacicept received FDA Breakthrough Therapy Designation for the treatment of IgAN. BRISBANE, Calif., Jan. 07, 2026 (GLOBE NEWSWIRE) -- Vera Therapeutics, Inc. (Nasdaq: VERA), a late clinical-stage biotechnology company focused on developing and commercializing transformative treatments for patients with serious immunological diseases, today announced the atacicept Biologics License Application (BLA) for the treatment of adults with immunoglobulin A nephropathy (IgAN) was accepted for Priority Review by the U.S. Food and Drug Administration (FDA). The BLA, which was submitted using the Accelerated Approval Program, was assigned a Prescription Drug User Fee Act (PDUFA) target action date of July 7, 2026. If approved, atacicept could offer patients an autoinjector for at-home self-administration of a once-weekly subcutaneous injection. “Atacicept offers a distinct approach through dual targeting of BAFF and APRIL, which we believe could advance the standard of care in IgAN, if approved,” said Marshall Fordyce, M.D., Founder and CEO of Vera Therapeutics. “FDA’s Priority Review designation reinforces the need for new therapies that can reshape the IgAN treatment landscape. We remain committed to working with the FDA to facilitate a thorough review of the BLA. Our team is focused on bringing a potential treatment to patients with the urgency they deserve.” The BLA submission for atacicept is supported by data from a prespecified interim analysis of the ORIGIN 3 trial, which met the primary endpoint of reduction in proteinuria at week 36. Participants treated with atacicept achieved a 46% reduction from baseline in proteinuria as measured by 24-hour urine protein-to-creatinine ratio (UPCR), with a statistically significant and clinically meaningful 42% reduction in UPCR compared to placebo (p<0.0001) at week 36. The safety pro atacicept across the ORIGIN program appears favorable, and comparable to placebo. Results of the interim analysis were presented as a late-breaking oral presentation at the opening plenary session of the American Society of Nephrology Kidney Week meeting and published in a manuscript in the New England Journal of Medicine on November 6. 1 IgAN is a serious and progressive autoimmune disease of the kidney for which there remains a high unmet need for new disease-modifying treatments that target the upstream source of the disease. In at least 50% of patients, IgAN can lead to end-stage kidney disease or kidney failure, which has considerable morbidity and impact on patients’ lives. Atacicept is being developed as an at-home self-administered subcutaneous once-weekly injection that inhibits B-cell Activating Factor (BAFF) and A Proliferation-Inducing Ligand (APRIL), cytokines that drive B-cell production of autoantibodies associated with IgAN and potentially other autoimmune kidney diseases. Lafayette R, et al. N Engl J Med. 2025 Nov 6. doi: 10.1056/NEJMoa2510198. Online ahead of print. About Atacicept Atacicept is an investigational recombinant fusion protein that contains the soluble transmembrane activator and calcium-modulating cyclophilin ligand interactor (TACI) receptor that binds to the cytokines B-cell activating factor (BAFF) and A PRoliferation-Inducing Ligand (APRIL). These cytokines are members of the tumor necrosis factor family that promote B-cell survival and autoantibody production associated with IgAN, lupus nephritis, and other autoimmune kidney diseases. About the Atacicept Clinical Program The ORIGIN Phase 2b clinical trial of atacicept in IgAN met its primary and key secondary endpoints, with statistically significant and clinically meaningful proteinuria reductions and stabilization of eGFR versus placebo through 36 weeks. The safety pro the randomized period was comparable between atacicept and placebo. Through 96 weeks, atacicept demonstrated further improvements in Gd-IgA1, hematuria, and proteinuria, as well as stabilization of eGFR reflecting a pro with that of the general population without IgAN. ORIGIN 3 (NCT04716231) is an ongoing global, multicenter, randomized, double-blind, placebo-controlled Phase 3 trial of 431 adults with IgA nephropathy. Participants were randomized 1:1 to atacicept 150 mg, self-administered at home via once weekly subcutaneous injection, or placebo. The primary efficacy endpoint of the prespecified 36-week interim analysis was the change in 24-hour UPCR compared to placebo. ORIGIN 3 met the primary endpoint with a statistically significant and clinically meaningful reduction in proteinuria at week 36. Across the ORIGIN program in IgAN, the safety pro atacicept appears favorable, and comparable to placebo. The trial continues in a placebo-controlled blinded manner to evaluate the change in kidney function over two years as measured by eGFR, with results expected in 2027. For more information about ORIGIN 3, please visit . Atacicept has received FDA Breakthrough Therapy Designation for the treatment of IgAN, which reflects the FDA’s determination that, based on an assessment of data from the ORIGIN Phase 2b clinical trial, atacicept may demonstrate substantial improvement on a clinically significant endpoint over available therapies for patients with IgAN. Vera Therapeutics believes atacicept is positioned for best-in-class potential, targeting B cells to reduce autoantibodies and having been administered to more than 1,500 patients in clinical trials across different disease areas. The ORIGIN Extend study provides ORIGIN study participants with extended access to atacicept until its potential commercial availability in their region and captures longer-term safety and efficacy data. Atacicept is also being evaluated in expanded IgAN populations, anti-PLA2R positive primary membranous nephropathy, and anti-nephrin positive focal segmental glomerulosclerosis (FSGS) and minimal change disease (MCD) patients in the PIONEER trial. About Vera Vera Therapeutics is a late clinical-stage biotechnology company focused on developing treatments for serious immunological diseases. Vera Therapeutics’ mission is to advance treatments that target the source of disease in order to change the standard of care for patients. Vera Therapeutics’ lead product candidate is atacicept, a fusion protein self-administered at home as a subcutaneous once weekly injection that blocks both B-cell Activating Factor (BAFF) and A PRoliferation-Inducing Ligand (APRIL), which stimulate B cells to produce autoantibodies contributing to certain autoimmune diseases, including immunoglobulin A nephropathy (IgAN) and lupus nephritis. Beyond IgAN, Vera Therapeutics is evaluating additional diseases where the reduction of autoantibodies by atacicept may prove clinically meaningful. In addition, Vera Therapeutics holds an exclusive license agreement with Stanford University for a novel, next generation fusion protein targeting BAFF and APRIL, known as VT-109, with wide therapeutic potential across the spectrum of B-cell-mediated diseases. Vera Therapeutics is also developing MAU868, a monoclonal antibody designed to neutralize infection with BK virus, which can have devastating consequences in kidney transplant recipients. Vera Therapeutics retains all global developmental and commercial rights to atacicept, VT-109 and MAU868. Forward-looking Statements . Statements contained in this press release regarding matters, events or results that may occur in the future are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, approval of atacicept by the FDA; the distinct approach of atacicept; atacicept's ability to advance the standard of care in IgAN; the strength of Vera Therapeutics’ clinical evidence; the timing of expected results of ORIGIN 3; atacicept’s positioning for best-in-class potential; and the plans, commitments, aspirations and goals under the caption “About Vera Therapeutics”. Words such as “believe,” “expect,” “may,” “plan,” “potential,” “will” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Vera Therapeutics’ current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks related to the regulatory approval process, results of earlier clinical trials may not be obtained in later clinical trials, preliminary results may not be predictive of topline results, risks and uncertainties associated with Vera Therapeutics’ business in general, the impact of macroeconomic and geopolitical events, and the other risks described in Vera Therapeutics' filings with the U.S. Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management’s assumptions and estimates as of such date. Vera Therapeutics undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law. For more information, please contact: Investor Contact: Joyce Allaire LifeSci Advisors 212-915-2569 jallaire@lifesciadvisors.com Media Contact: Debra Charlesworth Vera Therapeutics 415-854-8051 corporatecommunications@veratx.com