Nirsevimab

一、RSV 防控领域：Moderna 与 Merck 的双重突破6 月 13 日，Moderna 的 mRNA RSV 疫苗 mResvia 获 FDA 批准扩大适应症，将接种人群扩展至 18-59 岁的高危成人。这一决策基于 III 期试验数据，显示该疫苗在年轻高危群体中中和抗体滴度达老年人群的 90%。但分析师指出，真正的市场考验在于 CDC 免疫实践咨询委员会（ACIP）的推荐意见 —— 尤其是在新任 ACIP 主席、疫苗怀疑论者 Dr. Robert Malone 的领导下，审批后的接种指南可能面临限制。值得注意的是，HHS 部长 Robert F. Kennedy Jr. 此前罢免全部 17 名 ACIP 成员并重组委员会，其中两名成员曾公开反对 mRNA 技术，这为 mResvia 的临床推广蒙上阴影。Merck 则在 6 月 10 日凭借 RSV 单克隆抗体 clesrovimab（商品名 Enflonsia）打入婴幼儿市场。该药物通过 CLEVER 试验证实，可将新生儿 RSV 相关下呼吸道感染风险降低 60.5%，住院率减少 84.3%。其独特的 105mg 固定剂量方案无需根据体重调整，配合长达 5 个月的保护期，直接对标 Sanofi 与阿斯利康的 Beyfortus。6 月 26 日，ACIP 投票建议为 8 个月以下未获母源抗体保护的婴儿接种 Enflonsia，为 Merck 抢占约 200 万新生儿市场铺平道路。二、肿瘤治疗领域：争议审批与突破性疗法并存UroGen 的 Zusduri（丝裂霉素膀胱内注射剂）获批成为六月最具争议的审批事件。尽管 FDA 肿瘤药物咨询委员会以 5:4 的微弱劣势反对该申请，认为 ENVISION 试验缺乏随机对照且随访时间不足，但 FDA 最终基于 80.6% 的 18 个月持续缓解率数据批准其用于低级别中危非肌层浸润性膀胱癌。这是该适应症首个获批疗法，预计 7 月上市后将挑战现有卡介苗（BCG）疗法的主导地位。UroGen 承诺完成 ENVISION 试验以补充数据，此举被视为 FDA 在未满足医疗需求领域的灵活审批范例。Nuvation Bio 的 ROS1 抑制剂 taletrectinib（Ibtrozi）则凭借 TRUST 系列试验的惊艳数据获加速批准。在未接受过酪氨酸激酶抑制剂（TKI）的患者中，客观缓解率达 90%，脑转移患者颅内缓解率 73%。尽管单臂试验设计未纳入无进展生存期数据，但 Jefferies 分析师指出，该药物在 ROS1 阳性非小细胞肺癌（NSCLC）中的突破，或将重塑靶向治疗格局。Ibtrozi 作为 Nuvation 首款商业化产品，其上市进度将直接影响公司估值。三、多领域审批亮点：从前列腺癌到肝炎的全面覆盖Bayer 的 Nubeqa 在 6 月 4 日获批单药用于转移性去势敏感前列腺癌（mCSPC），打破此前需与多西他赛联合使用的限制。ARANOTE 试验显示，Nubeqa 联合雄激素剥夺治疗（ADT）使高负荷肿瘤患者疾病进展风险降低 40%，低负荷患者降低 70%。2024 年该药物已为 Bayer 带来 17.5 亿美元收入，新适应症预计将推动年销售额突破 20 亿美元。Beigene（现 BeOne）的 Brukinsa 片剂剂型获批则提升了用药便利性。160mg 片剂可替代原有的 80mg 胶囊，实现每日两次给药，成为首个兼具灵活给药方案的 BTK 抑制剂。生物等效性试验显示，片剂与胶囊在健康受试者中的药代动力学特征一致，预计 2025 年 10 月完成剂型替换。AbbVie 的 Mavyret 成为首个获批用于急性丙型肝炎的药物，8 周疗程的持续病毒学应答率达 96%。该适应症扩展基于 300 例初治患者的单臂试验，填补了急性 HCV 治疗的空白。尽管 2024 年 Mavyret 销售额同比下降 7% 至 13 亿美元，新适应症有望重新激活这一直接抗病毒药物的市场潜力。四、行业影响与未来展望六月的审批潮凸显 FDA 在公共卫生需求与科学严谨性间的平衡策略。RSV 领域的双雄并立（Moderna 疫苗 vs Merck 抗体）将引发市场细分竞争，而 UroGen 的争议获批或为同类 “单臂试验 + 高缓解率” 药物树立审批先例。值得关注的是，阿斯利康与第一三共的 Datroway（TROP2 ADC）凭借 45% 的客观缓解率获加速批准用于 EGFR 突变 NSCLC，但其后续确证性试验的成败将决定该类 ADC 药物的监管走向。随着 FDA 在 AI 工具 Elsa 争议中加速审批流程，下半年肿瘤免疫、基因治疗等领域的突破性疗法审批可能增多。但行业普遍认为，在 ACIP 重组与医保谈判压力下，创新药的市场准入挑战将与审批突破同步增加，真正的商业成功仍需跨越临床指南、支付政策等多重障碍。参考来源：https://www.biospace.com/fda/moderna-merck-urogen-score-rsv-and-cancer-nods-in-june识别微信二维码，添加生物制品圈小编，符合条件者即可加入生物制品微信群！请注明：姓名+研究方向！版权声明本公众号所有转载文章系出于传递更多信息之目的，且明确注明来源和作者，不希望被转载的媒体或个人可与我们联系(cbplib@163.com)，我们将立即进行删除处理。所有文章仅代表作者观不本站。

iStock, Grandbrothers The FDA delivered two notable approvals for RSV immunization, UroGen overcame a negative advisory committee vote to secure an approval in bladder cancer, and more key regulatory nods from the past month. Moderna Expands mRNA RSV Shot—But CDC Recommendation Still Uncertain The FDA on June 13 signed off on the broader use of Moderna ’s mRNA vaccine mResvia for respiratory syncytial virus, opening up inoculation for at-risk adults aged 18 through 59 years. For analysts at William Blair, however, approval is just the first regulatory hoop the vaccine will have to go through. “In our view, investor focus will likely shift to the CDC’s Advisory Committee on Immunization Practices (ACIP)’s next meeting,” they wrote in a note at the time, noting that the shot—and the broader vaccine market—could be battered by more headwinds still if the ACIP recommends “restrictive” guidelines. Currently, the CDC recommends a narrower use of RSV shots, where blanket vaccination is only recommended in adults 75 and older. In those 60 through 74 years of age, the CDC endorses immunization for those who are at risk of severe RSV. Earlier this month, Health and Human Services Secretary Robert F. Kennedy Jr. purged the ACIP of all 17 members in a move he claimed was “necessary to reestablish public confidence in vaccine science.” The next day, he named eight new panelists , many of whom have a documented history of vaccine skepticism and two who have previously spoken out specifically against the mRNA technology that underpins Moderna’s mResvia. The new committee members met on June 25 and 26 but did not vote on mResvia. The RSV vaccine was first approved in May 2024 for all adults 60 and above. Merck’s RSV Antibody Clears FDA Bar to Challenge Sanofi, AstraZeneca The FDA approved the use of Merck ’s anti-RSV antibody clesrovimab in infants on June 10. Merck will market the therapy under the brand name Enflonsia. The approval covers the use of Enflonsia to prevent RSV-related lower respiratory tract disease in newborns and infants who are entering their first season of circulating respiratory diseases. According to Merck, Enflonsia provides “rapid and durable” RSV protection persisting for five months, the length of a typical RSV season. Enflonsia is dosed at 105 mg regardless of patient weight. Enflonsia’s approval was backed by data from the Phase IIb/III CLEVER study, which found that in preterm and full-term infants up to 1 year, the antibody reduced RSV-associated medically attended lower respiratory infections by 60.5% versus placebo. Enflonsia also cut RSV hospitalizations by 84.3% as compared with placebo. Additional supporting data came from the Phase III SMART trial, which compared Enflonsia against Sobi’s Synagis, which is also a monoclonal antibody designed to prevent RSV. Interim results announced in October 2024 showed that Enflonsia could match Synagis’ safety and efficacy profiles through five months of observation. With Enflonsia, Merck will now go up against Sanofi and AstraZeneca, which own the immunizing antibody Beyfortus. Approved in July 2023 , Beyfortus has since become a top-performing asset for the companies, hitting blockbuster status in its first full commercial year with €1.7 billion (approximately $1.9 billion) in sales. On June 26, the CDC’s Advisory Committee for Immunization Practices gave Merck another win, voting to recommend Enflonsia to cover babies less than eight months who are not protected by maternal antibodies. Bayer Broadens Use of Nubeqa in Prostate Cancer On June 4, the FDA allowed the use of Bayer’s oral androgen receptor blocker Nubeqa for patients with metastatic castration-sensitive prostate cancer (mCSPC), regardless of concurrent chemotherapy. Before this approval, Nubeqa could only be used in the castration-sensitive patient population when it was combined with docetaxel, according to its label . The drug, taken twice daily, was originally approved in July 2019 for men whose cancers had yet to metastasize and were resistant to castration before winning an expansion in August 2022 for metastatic hormone-sensitive prostate cancer. Nubeqa’s latest approval was backed by data from the Phase III ARANOTE trial, a randomized, double-blinded and placebo-controlled trial with nearly 670 participants. Patients were given 600-mg Nubeqa on top of androgen deprivation therapy (ADT). Results, published in September 2024, showed that the Nubeqa regimen significantly improved radiographic progression-free survival, resulting in a 46% drop in the risk of disease progression or death as compared with placebo plus ADT. This efficacy was maintained in patients with high-volume mCSPC, who saw a 40% risk reduction, and was improved in those with low-volume disease, in which the risk was reduced by 70%. Nubeqa brought in €1.523 billion, or around $1.75 billion for Bayer in 2024. UroGen Surmounts AdComm Apprehension to Win Bladder Cancer Approval Despite failing to secure the backing of an external panel of advisors, UroGen won the FDA’s approval on June 13 for its intravesical drug mitomycin for the treatment of low-grade intermediate-risk non-muscle invasive bladder cancer. UroGen will market the drug under the brand name Zusduri. Zusduri is the first FDA-approved therapy for this indication and is meant for use in adults with recurrent disease, according to UroGen’s announcement. The drug, which is administered via a catheter directly into the bladder in an out-patient procedure, will be available “on or around” July 1. The Phase III ENVISION study, which is still ongoing, supported the regulatory decision with a 79.6% complete response rate at three months. The single-arm pivotal trial documented an 80.6% duration of response at 18 months, according to the latest data, released in April. Zusduri’s approval comes as a bit of a surprise, given that the FDA’s Oncologic Drugs Advisory Committee last month recommended against it—though only narrowly so. With a 5–4 split, the independent panel of experts pointed to several issues with UroGen’s application package, including the lack of a completely randomized study and the short follow-up in ENVISION. UroGen has promised to see the ENVISION trial through to completion “to further characterize the clinical benefit of Zusduri” and will provide the FDA with yearly updates regarding the drug’s duration of response for all treated patients with ongoing complete responses. Nuvation Wins First Commercial Approval With Lung Cancer Drug Ibtrozi On June 12, the FDA approved Nuvation Bio’s oral ROS1 blocker taletrectinib, which will be sold as Ibtrozi. The approval covers the drug’s use in non-small cell lung cancer. Ibtrozi is Nuvation’s first commercial product. Ibtrozi’s approval was backed by data from the Phase II TRUST-I and TRUST-II trials, which together account for “one of the largest global clinical trial programs in ROS1+ NSCLC [non-small cell lung cancer] to date,” according to Nuvation’s Wednesday release. Over 300 patients were enrolled across both trials. Results from TRUST-I showed a confirmed overall response rate (cORR) of 90% in patients with no prior exposure to tyrosine kinase inhibitor (TKI). TRUST-II confirmed these findings with an 85% cORR. Because of the single-arm nature of the TRUST program, progression-free survival isn’t included in Ibtrozi’s label. The TRUST studies also specifically looked at the benefits of Ibtrozi in patients with brain metastases—which Nuvation on Wednesday called “among the most common and devastating complications” in ROS1-positive NSCLC—and found high rates of treatment response. In patients with measurable brain metastasis at baseline, Ibtrozi demonstrated an intracranial response of 73% in the TKI-naïve subgroup and 63% in those who had previously been exposed to TKI treatments. Analysts at Jefferies at the time said that while Ibtrozi’s approval was positive for Nuvation, “patience may be needed by investors with several quarters to execute and demonstrate strong launch momentum.” BeOne Secures Approval for Twice-Daily Brukinsa Schedule BeOne—formerly known as BeiGene—received FDA approval on June 12 for a new tablet formulation of the tyrosine kinase inhibitor Brukinsa to be taken twice-daily. This label expansion covers all approved indications of Brukinsa , including mantle cell lymphoma, marginal zone lymphoma, chronic lymphocytic leukemia, small lymphocytic lymphoma and Waldenström’s macroglobulinemia. “The recommended dose of Brukinsa remains at 320 mg daily,” BeOne noted in a news release announcing the approval. The new formulation delivers a 160-mg dose of the drug in each tablet, giving patients the option to “take two tablets daily rather than four of the current 80 mg capsules.” With the tablet nod, Brukinsa has become “the only BTK inhibitor to offer the flexibility of once or twice daily dosing,” with some leeway to adjust the schedule based on patient needs, the company said. Supporting the FDA’s decision are bioequivalence data generated from two Phase I crossover trials of healthy adults, which showed that the tablets are as safe and as effective as the capsules. Matt Shaulis, North America general manager of BeOne, said the tablet approval makes “treatment simpler and more convenient” for patients. BeOne expects to completely replace the 80-mg capsules with the 160-mg tablets by October 2025. AbbVie’s Mavyret Becomes First Antiviral for Acute Hepatitis C Also notching a notable regulatory nod this month is AbbVie, which on June 11 won the FDA’s first approval for an acute hepatitis C virus (HCV) drug. The approval of Mavyret, an oral antiviral, was supported by data from a Phase III single-arm prospective trial that enrolled nearly 300 treatment-naïve adults who had acute infection. Mavyret was given once-daily for eight weeks. Results, which are reflected in the drug’s label , show that Mayret elicited a sustained virological response of 96% 12 weeks post-treatment. None of the study participants experienced virologic failure. According to AbbVie, the label expansion allows doctors to treat patients “immediately at the time of diagnosis.” While hepatitis C infection is a curable disease, it often goes undiagnosed, as per the pharma’s news release announcing the approval. “If left untreated, people with acute HCV could progress to chronic disease, including liver-related complications” like cancer and cirrhosis. Mavyret is a direct-acting antiviral tablet that delivers a combination of drugs: glecaprevir , a protease inhibitor that disrupts the replication of the virus’ genetic material, and pibrentasvir , which blocks viral RNA replication while also preventing the formation of the viral particle itself. First approved in August 2017 , Mavyret is the first-ever product to be indicated for all types of HCV. Mavyret brought in roughly $1.3 billion in 2024 , a nearly 7% decline year-on-year. AstraZeneca, Daiichi Sankyo Earn Accelerated Approval for Datroway in NSCLC Datroway, an antibody-drug conjugate jointly developed and commercialized by AstraZeneca and Daiichi Sankyo, can now be used for patients with advanced or metastatic non-small cell lung cancer (NSCLC) carrying EGFR mutations. The FDA granted the biologic accelerated approval in this indication on June 24. The regulatory decision was backed by data from a subgroup analysis of the Phase II TROPION-Lung05 study, also supported by findings from the Phase III TROPION-Lung01 trial. Findings from these studies showed that Datroway elicits a confirmed objective response rate of 45% in patients who had undergone prior lines of treatment. This includes 4.4% complete responses and 40% partial responses. Median duration of response was 6.4 months. As per the terms of the FDA’s accelerated pathway, AstraZeneca and Daiichi Sankyo will need to validate Datroway’s clinical benefit in future confirmatory trials to maintain this approval. Datroway won its first U.S. approval in January 2025, allowing its use in patients with unresectable or HR-positive, HER2-negative breast cancer who had been previously treated. The therapy works by binding to TROP2, a surface protein found on many tumor cells, and delivering its topoisomerase I inhibitor payload, according to its label . This mechanism damages DNA in cancer cells, ultimately leading to their death.