Colchicine

In June 2025, several breakthrough drugs received their first global approvals and market launches, marking significant advances in therapeutic innovation. 1. Genakumab (Firsekibart) Firsekibart (Genakumab), developed by Changchun Genescience Pharmaceutical, is a fully human monoclonal antibody targeting interleukin-1β (IL-1β). It functions as an IL-1β inhibitor and was approved in China on June 30, 2025, for the treatment of gouty arthritis, especially in patients who are intolerant or unresponsive to NSAIDs or colchicine. The approval addresses a significant gap in long-acting, targeted anti-inflammatory therapies in China, offering a new option for over 14 million gout patients. Gout is a chronic inflammatory disease caused by urate crystal deposition in joints. It is often referred to as the "king of pain" due to its severe and recurrent attacks. The underlying pathology involves activation of the NLRP3 inflammasome in macrophages, leading to a surge in IL-1β secretion, which triggers a cascade of inflammatory responses. Firsekibart is a fully human anti-IL-1β monoclonal antibody that binds specifically to IL-1β and neutralizes its biological activity, blocking receptor interaction and suppressing inflammation at its source. This mechanism allows for rapid pain relief during acute attacks and sustained anti-inflammatory effects during intercritical periods, helping reduce recurrence rates and inflammatory flares during urate-lowering therapy. It may also offer systemic benefits for cardiorenal and metabolic comorbidities. The approval was based on data from a randomized, double-blind, multicenter Phase III trial involving 313 patients with acute gout flares. Results showed that a single dose of Firsekibart provided pain relief within 6 hours, with a comparable 72-hour efficacy to compound betamethasone. The primary endpoint—the difference in VAS pain score at 72 hours—was -3.32 mm (95% CI: -7.56, 0.91), confirming non-inferiority. Notably, Firsekibart demonstrated superior performance in preventing recurrence: ·Median time to first gout recurrence: not reached (Firsekibart) vs. 45 days (steroid group) ·Risk reduction in first recurrence within 6 months: up to 90% ·Recurrence reduction over 24 weeks: 87%, with 85.3% of patients remaining recurrence-free—the best result among its class. The drug was well tolerated, with a treatment-related adverse event rate similar to the control group and no serious adverse events reported. Future indications for Firsekibart may include cardiovascular inflammation, osteoarthritis, diabetes, obesity, and tumor microenvironment modulation, suggesting broad clinical potential. 2. Gemcitabine (AVGEMSI, D07001-Softgel) Gemcitabine, a small-molecule chemotherapeutic agent originally developed by FUJIFILM Corporation, received its first approval in the United States on June 27, 2025, for the treatment of multiple malignancies, and was granted orphan drug designation. Gemcitabine is a pyrimidine-based antitumor drug with a mechanism of action similar to cytarabine (Ara-C). It is activated intracellularly by deoxycytidine kinase (dCK) and metabolized by cytidine deaminase. Once metabolized, its active form incorporates into DNA during the G1/S phase, inhibiting DNA synthesis. Unlike cytarabine, gemcitabine also inhibits ribonucleotide reductase, decreasing intracellular dNTP pools and further impairing DNA replication. Additionally, it inhibits deoxycytidine deaminase, reducing the degradation of its own active metabolites—effectively self-potentiating its efficacy. These pharmacodynamic features give gemcitabine a broader antitumor spectrum than cytarabine, and it has demonstrated efficacy across multiple solid tumors, including metastatic breast cancer, non-small cell lung cancer (NSCLC), ovarian cancer, and pancreatic adenocarcinoma. Clinically, gemcitabine has become a standard first-line therapy for locally advanced and metastatic NSCLC. A pivotal Phase III trial in advanced NSCLC patients showed that the gemcitabine + cisplatin combination significantly improved overall survival compared to cisplatin alone: ·Median OS: 10.7 months (combination) vs. 8.6 months (cisplatin monotherapy) ·The difference was statistically significant (p < 0.05) Gemcitabine’s safety profile has been extensively studied. While myelosuppression, gastrointestinal discomfort, and fever are common adverse effects, they are generally manageable and acceptable, confirming the drug’s suitability for routine clinical use in oncology. 3. Mazdutide (IBI362) Mazdutide (also known as IBI362) is a synthetic polypeptide drug co-developed by Eli Lilly & Co. and Innovent Biologics. It acts as a dual agonist of GLP-1 and GIP receptors, designed for the treatment of endocrine and metabolic disorders. On June 24, 2025, it received its first market approval in China, with an indication for obesity and overweight. The drug also shows promising potential in other metabolic conditions such as type 2 diabetes, obstructive sleep apnea, non-alcoholic steatohepatitis (NASH), and various forms of heart failure. Mazdutide exerts synergistic effects by simultaneously activating GLP-1 and glucagon (GCG) receptors. Activation of GLP-1 receptors delays gastric emptying, suppresses appetite, and enhances satiety, leading to reduced caloric intake. Activation of the GCG receptor promotes lipolysis and increases energy expenditure, thereby enhancing weight loss. This dual mechanism provides superior efficacy compared to single-target agents, positioning Mazdutide as a representative of next-generation metabolic regulators. Clinical studies in Chinese populations have demonstrated significant weight loss and good safety. The pivotal Phase III GLORY-1 trial, published in the New England Journal of Medicine (NEJM), enrolled 610 Chinese subjects who were overweight or obese. After 48 weeks of once-weekly subcutaneous injections, the 4 mg group experienced a mean weight loss of 11.00%, and the 6 mg group lost 14.01%, compared to 0.3% in the placebo group. Notably, nearly 50% of patients in the 6 mg group achieved at least 15% body weight reduction, with effects evident as early as week 32. Beyond weight reduction, Mazdutide also improved glycemic control, lipid profiles, and blood pressure, making it particularly beneficial for Chinese patients with metabolic syndrome. In terms of safety, Mazdutide was well tolerated. The most common adverse events were mild to moderate gastrointestinal symptoms, including nausea, vomiting, and diarrhea, which mostly occurred during early treatment and resolved over time. The once-weekly injection regimen enhances patient adherence and supports long-term disease management. 4. Tetravalent Influenza Vaccine Developed by Fosun Apexvac (Dalian) Biopharmaceutical Co., Ltd., the tetravalent split inactivated influenza vaccine was first approved in China on June 17, 2025, for the prevention of influenza virus infection. The vaccine is produced using internationally standardized embryonated egg-based manufacturing techniques, free from antibiotics and preservatives, and provides broad protection by inducing immunity against four different influenza virus strains. Influenza is an acute respiratory infectious disease caused by influenza viruses. Due to the virus’s high genetic variability and broad host range, it spreads easily among humans, resulting in 3–5 million severe cases and 290,000–650,000 deaths globally each year. The World Health Organization (WHO) emphasizes that vaccination is the most effective method for influenza prevention, especially for high-risk individuals and their close contacts. This tetravalent vaccine contains inactivated antigens from four influenza strains: two A subtypes (e.g., H1N1 and H3N2) and two B lineages. The viruses are chemically split to release key antigens, such as hemagglutinin (HA) and neuraminidase (NA), which trigger immune responses. Upon administration, antigen-presenting cells (e.g., dendritic cells) process these viral antigens and present them to helper T cells, which in turn activate B cells to produce strain-specific antibodies—especially anti-HA antibodies capable of neutralizing the virus. The vaccine also induces memory B and T cells, enabling rapid immune responses upon future exposure to similar strains. 5. 13-Valent Pneumococcal Polysaccharide Conjugate Vaccine (CRM197, TT) The 13-valent pneumococcal polysaccharide conjugate vaccine, developed by CanSino Biologics, was first approved in China on June 17, 2025, for the prevention of Streptococcus pneumoniae infections. This multivalent conjugate vaccine targets 13 distinct pneumococcal serotypes responsible for pneumonia, meningitis, septicemia, and other invasive diseases. The vaccine functions by covalently linking capsular polysaccharide antigens of pneumococcal serotypes to protein carriers such as CRM197 (a non-toxic diphtheria toxin mutant) and tetanus toxoid (TT). This conjugation converts the normally T cell-independent polysaccharides into T cell-dependent antigens, capable of eliciting a stronger and longer-lasting immune response, particularly in infants and young children. After administration, the conjugated antigens are taken up and processed by antigen-presenting cells, then presented to CD4+ T helper cells, which activate B cells to generate serotype-specific antibodies. Additionally, the vaccine promotes the formation of memory B cells, providing long-term protection. This mechanism enables the immune system to mount a robust defense against the 13 pneumococcal serotypes, significantly reducing the risk of invasive pneumococcal disease (IPD). The vaccine is especially effective in pediatric populations, offering enhanced and durable immunity. 6. Garadacimab Garadacimab (formerly CSL312) is a fully human recombinant IgG4/λ monoclonal antibody developed by CSL Behring LLC. It was approved in the United States on June 16, 2025, for the treatment of hereditary angioedema (HAE), with additional potential applications in diseases such as idiopathic pulmonary fibrosis. HAE is a rare but potentially life-threatening genetic disorder that affects approximately 1 in 50,000 individuals. It is characterized by recurrent, painful episodes of swelling (angioedema), which can involve the larynx, gastrointestinal tract, and other critical areas. Garadacimab exerts its therapeutic effect by selectively binding to and inhibiting coagulation factor XIIa (FXIIa), a key initiator in the contact activation system involved in inflammatory and coagulation cascades. By targeting FXIIa, Garadacimab helps reduce pathological processes such as vascular leakage and inflammation, which are central to HAE pathogenesis. FXII is particularly important in HAE patients due to its role in activating the kallikrein-kinin system, leading to bradykinin-mediated angioedema. Garadacimab features an unusually long CDR-H3 loop, allowing specific binding to the β-chain of FXIIa, thereby blocking its protease activity and downstream pro-inflammatory and pro-coagulant pathways. The FDA approval was supported by efficacy and safety data from the pivotal Phase III VANGUARD trial (NCT04656418) and its open-label extension (NCT04739059). VANGUARD evaluated Garadacimab as a routine prophylactic treatment in 64 patients aged ≥12 years with Type I or II HAE, all of whom experienced ≥2 attacks during the run-in period and discontinued prior prophylactic therapies. Participants were randomized 3:2 to receive 400 mg loading dose followed by 200 mg monthly Garadacimab (n=39) or matched placebo (n=25) for 6 months. On-demand medication for breakthrough attacks was allowed. At baseline, 59.4% of participants had ≥3 HAE attacks per month (mean: 3.07/month in the Garadacimab group vs. 2.52/month in the placebo group). Garadacimab significantly reduced attack frequency and demonstrated a favorable safety profile, reinforcing its potential as a long-term preventive therapy for HAE. Additional trials in fibrotic and inflammatory diseases are ongoing, highlighting its broad therapeutic promise. 7. Taletrectinib Taletrectinib, also known by its compound name ADCT-601 or AnHeart Therapeutics' ROS1 inhibitor, is a next-generation small molecule tyrosine kinase inhibitor (TKI) targeting ROS1-positive non-small cell lung cancer (NSCLC). Co-developed by AnHeart Therapeutics (Hangzhou) and Innovent Biologics, Taletrectinib received FDA approval on June 11, 2025, for the treatment of locally advanced or metastatic ROS1-positive NSCLC. ROS1 gene rearrangements are oncogenic drivers found in approximately 1–2% of NSCLC cases and are associated with poor response to standard chemotherapy. Taletrectinib is a highly potent and selective ROS1 TKI, designed to inhibit the aberrant signaling caused by ROS1 fusions, which drive tumor growth in affected patients. Notably, it also shows efficacy against certain resistance mutations, offering therapeutic options for both treatment-naïve patients and those with acquired resistance to first-generation ROS1 inhibitors like crizotinib. FDA approval was based on results from two pivotal Phase II global multicenter studies, TRUST-I and TRUST-II, which evaluated the efficacy and safety of Taletrectinib in ROS1-positive advanced NSCLC across both treatment-naïve and pretreated populations. Patients received 600 mg orally once daily, and endpoints included objective response rate (ORR), duration of response (DoR), progression-free survival (PFS), and intracranial response in patients with brain metastases. Taletrectinib demonstrated robust clinical activity in both cohorts. It achieved high ORR, prolonged PFS, and demonstrated notable intracranial efficacy, making it a strong candidate for managing CNS-involved disease. Safety outcomes were favorable, with manageable adverse events, supporting its use as a next-generation targeted therapy for ROS1-positive NSCLC. Its ability to overcome resistance mechanisms further positions it as a valuable treatment option for patients who have progressed on earlier ROS1-targeted agents. 8. Clesrovimab Clesrovimab (MK-1654), developed by Merck Sharp & Dohme Corp., is a long-acting monoclonal antibody that received its first FDA approval on June 9, 2025, for the prevention of respiratory syncytial virus (RSV) infection. Clesrovimab specifically targets the RSV fusion (F) protein, a critical viral surface protein required for viral entry into host cells. By binding with high affinity to the F protein, Clesrovimab not only blocks the fusion process between the virus and host cell membranes—thereby preventing viral entry—but also enhances the immune system’s ability to recognize and eliminate the virus. Importantly, since the F protein is highly conserved across all RSV subtypes, Clesrovimab exhibits broad-spectrum antiviral activity against a wide range of RSV strains. As a passive immunization therapy, Clesrovimab’s extended half-life allows it to deliver direct, rapid, and long-lasting protection against RSV infection in healthy preterm infants, full-term infants, and high-risk infants. The antibody is designed to protect infants during their first RSV season against mild, moderate, and severe RSV disease. Data from the pivotal Phase 2b/3 CLEVER trial demonstrated that a single dose of Clesrovimab reduced medically attended RSV lower respiratory tract infections by 60.5% and hospitalizations due to RSV by 84.3% within 150 days post-treatment. Further evidence from the SMART Phase 3 study validated Clesrovimab’s efficacy and safety in both preterm and full-term infants, as well as high-risk populations, where it showed superior performance compared to existing standard preventive therapies. 9. Amlodipine Besylate/Indapamide/Telmisartan Amlodipine Besylate/Indapamide/Telmisartan, marketed as Widaplik, is a fixed-dose triple combination therapy developed by George Medicines Pty Ltd. It received its first FDA approval on June 5, 2025, for the treatment of hypertension. Widaplik is the first and currently only FDA-approved triple-combination product specifically designed for patients who may require multiple antihypertensive agents to achieve optimal blood pressure control. Widaplik combines three active components, each with a distinct antihypertensive mechanism: ·Amlodipine, a calcium channel blocker, relaxes vascular smooth muscle by inhibiting calcium influx into cardiac and arterial wall cells. ·Telmisartan, an angiotensin II receptor blocker (ARB), prevents vasoconstriction and reduces fluid retention by blocking angiotensin II. ·Indapamide, a thiazide-like diuretic, promotes the excretion of sodium and water, further lowering blood pressure. Approval was supported by two pivotal clinical studies: Study 1 enrolled 295 patients with systolic hypertension who were either treatment-naïve or on monotherapy. Following a 2-week placebo run-in, patients were randomized to receive different doses of Widaplik or placebo. At week 4, both Widaplik doses significantly reduced home-measured systolic blood pressure compared to placebo (P < 0.0001), with most of the antihypertensive effect observed within the first two weeks. Study 2 compared Widaplik to dual-component therapies. At week 12, Widaplik achieved superior reductions in home systolic blood pressure versus all dual-therapy arms (P < 0.0001). The effect was consistent across age, sex, and racial subgroups. In terms of safety, Widaplik was generally well tolerated. The most common adverse events were symptomatic hypotension and electrolyte imbalances such as hyponatremia and hypokalemia. Summary June 2025 marked the approval and launch of several milestone new drugs in the global pharmaceutical landscape, representing significant advances across multiple therapeutic areas. From targeted precision therapies like Taletrectinib and Garadacimab, to multimodal combination regimens like Widaplik, and passive immunization agents such as Clesrovimab and Genakumab, to next-generation vaccines like the quadrivalent influenza vaccine and the 13-valent pneumococcal conjugate vaccine, these innovative products exemplify the synergistic potential of biologics and small molecules. Together, they are not only enhancing treatment efficacy across various diseases but are also shaping the future of personalized, effective, and sustainable medicine. How to obtain the latest research advancements in the field of biopharmaceuticals? In the Synapse database, you can keep abreast of the latest research and development advances in drugs, targets, indications, organizations, etc., anywhere and anytime, on a daily or weekly basis. Click on the image below to embark on a brand new journey of drug discovery!

前言近日，国家药品监督管理局（NMPA）官网发文宣布，金赛药业自主研发的1类创新药——金蓓欣（伏欣奇拜单抗）已正式获批上市，适用于对非甾体类抗炎药和/或秋水仙碱禁忌、不耐受或缺乏疗效的，以及不适合反复使用类固醇激素的成人痛风性关节炎急性发作患者1。2025年7月14日，金蓓欣（伏欣奇拜单抗）在上海正式开出全国首张临床处方，标志着这款针对急性痛风发作的突破性药物正式进入临床应用阶段。作为国内首个IL-1β单抗，伏欣奇拜单抗的成功获批与应用将迅速填补我国痛风领域长效精准靶向治疗的空白，为饱受痛风性关节炎反复发作之苦的广大患者带来福音。首张处方落地，树立痛风治疗领域全新里程碑7月14日，华山医院风湿免疫科薛愉主任为47岁的痛风患者张先生开出全国首例金蓓欣（伏欣奇拜单抗）治疗处方。张先生自2002年诊断为痛风，目前已有20余年痛风病史，反复发作的关节包括脚踝、大脚趾、膝盖、手指、手腕和手肘。张先生的常用治疗药物为非甾体类抗炎药，但疗效不太理想，痛风发作时需每日服用。严重时会使用秋水仙碱治疗，但服用后会出现头晕、恶心等副作用。近日，张先生晨起脚背、手肘和膝盖关节再次出现不适，遂来院就诊。在详细评估病情后，华山医院风湿免疫科薛愉主任为其开具了伏欣奇拜单抗200mg的处方。复旦大学附属华山医院薛愉主任为患者开出首张处方邹和建教授点评复旦大学附属华山医院风湿免疫科学术带头人伏欣奇拜单抗III期研究主要研究者痛风领域抗炎治疗药物副作用大，无法长期使用维持疗效。患者往往陷入“用药-复发-再用药”的恶性循环。伏欣奇拜单抗通过精准阻断IL-1β信号通路，从根源上抑制炎症反应。Ⅲ期临床研究已经证实，伏欣奇拜单抗单次给药后即可快速起效，6-72小时镇痛效果与激素相当，6个月内首次复发风险降低87%，且未发现与药物相关的严重不良反应2。伏欣奇拜单抗的上市，为急性发作频繁，或对NSAIDs和/或秋水仙碱禁忌、不耐受及缺乏疗效，以及不适合反复使用激素的患者带来了治疗新选择。此次首张处方落地，为我国痛风治疗领域树立了全新里程碑，期待其未来在临床实践中的表现。关于伏欣奇拜单抗  //  伏欣奇拜单抗通过精准阻断引发痛风炎症的IL-1β起效，具有“长期控制、快速强效、安全放心”三大临床优势。此次获批是基于一项在痛风性关节炎患者中开展的伏欣奇拜单抗Ⅲ期临床试验结果，该多中心、随机、双盲、阳性对照临床试验共纳入313例患者，结果显示2：主要研究终点：72小时疼痛VAS评分改善伏欣奇拜单抗组非劣于复方倍他米松组，两组间差异为-3.32mm (95% CI：-7.56, 0.91)，95%的置信区间上限为0.91mm，小于预设的非劣效界值10mm，非劣效成立。共同主要研究终点：单次给药12周内，首次急性痛风复发中位时间，伏欣奇拜单抗组和复方倍他米松组分别为：未达到和45天（95%CI：28.00，63.00）；伏欣奇拜组对比复方倍他米松组降低90%（HR=0.10，p<0.0001）的首次复发风险。次要研究终点：给药24周内，伏欣奇拜单抗组对比复方倍他米松组降低87%（HR=0.13，p<0.0001）的首次复发风险，85.3%的患者无复发，创同类药物更优纪录，奠定其痛风抗炎药物领域"Best-in-class"潜力地位。在安全性方面，共计159例（51.0%）受试者出现治疗相关不良事件，其中伏欣奇拜单抗组79例（50.6%），复方倍他米松组80例（51.3%）。伏欣奇拜组无治疗相关严重不良事件，复方倍他米松组共有3例患者报告了治疗相关严重不良事件。由此，中国首款自主研发的痛风创新药伏欣奇拜单抗正式开启临床应用新征程，期待未来其在临床实践中的优秀表现，让“无激素治疗”成为痛风管理的常态。专家简介邹和建 教授复旦大学附属华山医院学术及社会任职复旦大学附属华山医院风湿免疫科学术带头人、教授复旦大学风湿免疫过敏性疾病研究中心主任国际硬皮病临床与研究协作网（InSCAR）副主席中华预防医学会风湿病预防专委会主任委员中华医学会风湿病学分会第7、8届委员会副主任委员中国医师协会风湿免疫科医师分会第1、2届委员会副会长薛愉 教授复旦大学附属华山医院复旦大学附属华山医院风湿科副主任、主任医师、硕士生导师上海市医学会风湿病专科分会副主任委员中华医学会风湿病专科分会委员中国女医师协会第一届风湿免疫专业委员会常务委员中国中西医结合学会风湿类疾病专业委员会青年副主任委员上海市医学会职业病与环境医学专科分会委员兼秘书中华预防医学会中毒与急救专业青年分会青年委员参考文献1.https://www.nmpa.gov.cn/zhuanti/cxylqx/cxypxx/20250702164944152.html2.Xue Y, et al. Efficacy and Safety of Genakumab versus Compound Betamethasone in Gout: The GUARD-1 Study [abstract]. Arthritis Rheumatol. 2024; 76 (suppl 9).撰写：ljy审校：zl排版：zyz执行：Atai本平台旨在为医疗卫生专业人士传递更多医学信息。本平台发布的内容，不能以任何方式取代专业的医疗指导，也不应被视为诊疗建议。如该等信息被用于了解医学信息以外的目的，本平台不承担相关责任。本平台对发布的内容，并不代表同意其描述和观点。若涉及版权问题，烦请权利人与我们联系，我们将尽快处理。