Hepatitis B vaccine(GlaxoSmithKline Plc)

iStock, quantic69 Pfizer and Metsera, Sarepta and uniQure made the list with dramatic tales. The other two spots went to the regulatory challenges facing biopharma under the new administration, especially in the vaccines sector. A constant refrain over the past year in the BioSpace newsroom chat has been, “Will this news cycle ever slow down?” Well, we sit here in late December with the answer: No. December has been anything but slow, with market-moving, hotly anticipated data still coming in, regulatory and personnel drama at the FDA and CDC, and much more keeping this little newsroom busy. The truth is, we thrive on this stuff and while we may throw up our hands from our keyboards on some mornings—and Friday afternoons—and ask the news gods, Why? , we secretly love every second of it. So going into the holiday break, the BioSpace news team took a look back at the stories we thought were the most important from 2025. These sagas intersected all of our beats, from business to clinical development to the regulatory environment. FDA FDA Policy Tracker: 2025 Was a Year of Change Policy initiatives have come fast and furious at the FDA this year. While guidances on rare diseases and vaccines have consumed most of the ink, policy shifts aimed at improving FDA efficiencies and reshoring U.S. manufacturing also got some attention. Here, BioSpace rounds up more than a dozen initiatives relevant to the biopharma industry. December 22, 2025 · 13 min read · Heather McKenzie Read More Pfizer-Metsera-Novo Bidding War Fit for Reality TV Where does one even begin with the saga that was the Metsera acquisition? Or as BMO Capital Markerts analyst Evan David Seigerman dubbed it: The Real Housewives of Biopharma . In September, Pfizer offered a cool $4.9 billion upfront plus a contingent value right to buy obesity hotshot biotech Metsera. The company debuted in 2024 to much fanfare, then met the hype with multiple clinical readouts that quickly put it as the front of the pack of up-and-comers in the ballooning weight loss space. It conducted one of the most successful IPOs of 2025 in January. The Pfizer buyout seemed on its way to closing. In regulatory filings about the deal, Metsera noted that there had been other parties interested, including one—later revealed to be Novo Nordisk—that was likely to have regulatory challenges in closing. Then Novo dropped a bomb . On October 30, the Danish pharma, desperate to replenish a flagging obesity pipeline, submitted an unsolicited bid to usurp Pfizer and buy Metsera for about $8.5 billion. A normally restrained Pfizer seemed livid, calling the offer “reckless and unprecedented.” The New York pharma quickly filed litigation against Novo, Metsera and the biotech’s shareholders. A bidding war began, with both parties upping the ante all the way to $10 billion. Novo’s CEO even called out Pfizer in an appearance at the White House: “Our message to Pfizer is that if they would like to buy the company, then put your hand in the pocket and bid higher,” Maziar Mike Doustdar said from behind a lecturn in the Oval Office. Days later, Pfizer finally secured its prize. The company matched Novo’s $10 billion bid but offered something Novo could not: certainty. Pfizer had already secured the Federal Trade Commission’s blessing. Novo was likely to take as long as two years to finalize the deal, using a framework that had already drawn antitrust concerns from the regulator. Metsera shareholders approved the deal and it closed in November, thereby capping off the most exciting biopharma M&A story in years. Obesity Is Metsera Worth the Fuss? Pfizer and Novo Nordisk seem to want Metsera bad. Analysts are wondering, though: Is the obesity biotech really worth this much effort? November 5, 2025 · 4 min read · Annalee Armstrong Sarepta’s Gene Therapy Platform Causes Deaths and Drama This summer, Sarepta Therapeutics found itself in hot water with investors and the FDA after failing to disclose the death of a patient taking one of its investigational gene therapies. News of the death broke via media reports the day after the company announced a black box warning for its approved Duchenne muscular dystrophy (DMD) gene therapy Elevidys, following two earlier deaths tied to the treatment. The company also stated that it was pivoting away from the vector technology used for both gene therapies and for which Sarepta had received the first-ever FDA platform designation. Sarepta CEO Doug Ingram later told investors that the company didn’t disclose the death in this corporate update “because it was neither material nor central to the topics at hand.” Sarepta had previously reported the death to the FDA, but the agency later claimed “it was lost in the bureaucracy” and that the agency had only found out about the third death with the rest of the world. Communication mishaps aside, the FDA reacted to news of the third death by pausing clinical trials for the investigational therapy, revoking Sarepta’s platform designation and asking the company to discontinue shipments of Elevidys. To that final request—which was a request and not a demand because Elevidys has full market approval—Sarepta initially said no . But after just a few days, the company relented, and paused Elevidys shipments for all U.S. patients for 10 days . In November, the FDA made official the black box warning for Elevidys and limited its use to ambulatory patients. Sarepta’s future now hangs in the balance. After starting the year with a share price of $124, the company’s stock hit its lowest point in nearly 10 years and continues to hover around just $20. Editorial Lack of Transparency Tarnishes Sarepta’s Sheen as Patient Deaths Trigger FDA Battle Sarepta Therapeutics’ stock has dropped precipitously as questions swirl around the safety of its gene therapies. Meanwhile, the Duchenne patient community fears losing access to Elevidys while the regulator considers more drastic action. July 22, 2025 · 6 min read · Jef Akst UniQure Brings Hope in Huntington’s but Highlights Regulatory Woes While the Alzheimer’s and amyotrophic lateral sclerosis spaces have seen incremental breakthroughs over the past five years, patient with Huntington’s disease have continued to wander in a desert of hopelessness . Until this September. That’s when uniQure reported three-year data from a pivotal trial of its gene therapy AMT-130 showing 75% slowing of the devastating neurodegenerative disease. While small—the Phase I/II study consisted of just 29 patients—experts hailed the moment as “historic” and BBC News declared that Huntington’s had been successfully treated for the first time. Still, Ignacio Munoz-Sanjuan, CEO and chairman at Rumi Scientific and president of the Huntington’s-focused non-profit Factor-H, cautioned against communicating the results in a way that conveyed unrealistic expectations at this stage of development. The space had been here before. In an interview with BioSpace , Munoz-Sanjuan recalled a similar reaction to Roche’s tominersen when it was shown to lower mutant huntingtin (mHTT) levels in the cerebrospinal fluid in a Phase I trial. Tominersen would later fail in Phase III. Still, uniQure’s trial was considered pivotal and the company communicated plans in a Sept. 24 press release to FDA approval of AMT-130 in the first quarter of 2026, with a potential launch later next year. Five weeks later, the FDA said not so fast. On Nov. 3, following a pre-BLA meeting, uniQure revealed that the agency “no longer agrees” that the Phase I/II data for AMT-130 will be “adequate to provide the primary evidence in support of a BLA submission.” This, despite the company having aligned with the agency on the protocols and statistical analyses used. This news ultimately placed uniQure at the heart of another key story of 2025—apparent FDA 180s faced by several biotechs, including Replimune, Biohaven and Capricor Therapeutics. Since uniQure’s latest announcement, more than 10,000 Huntington’s patients, families and supporters have signed two petitions calling for the FDA to reconsider its latest guidance. “For families who have long awaited new treatment options, the FDA’s actions feel like a serious setback and have raised urgent concerns about transparency, consistency, and trust in the regulatory process,” Christina DeGryse, a Huntington’s disease advocate, wrote in an email to BioSpace on Nov. 16. FDA FDA Reversals Send UniQure, Biohaven, Capricor, More Into a ‘Tailspin’ Since July, several biotechs have been forced to pivot as previous agreements with the FDA around evidence required for approval were reversed, a phenomenon that, according to experts, could portend a more restrictive regulator. November 24, 2025 · 7 min read · Heather McKenzie FDA Turmoil Creates Uncertain Regulatory Environment What is FDA policy in 2025? It’s the operational question of the year for biopharma. Proposals for regulatory changes have come via unusual channels—on social media, in media interviews and in editorials published by medical journals—often with little detail about how the new policies will be implemented. In BioSpace ’s Policy Tracker, we attempted to round up all the changes the industry has faced this year, but due to the uncertainty of these communications, it was often difficult to decide whether or not to include an initiative introduced by Commissioner Marty Makary, Center for Biologics Evaluation and Research (CBER) head Vinay Prasad or other key regulators. Promo: Policy Tracker (Notably, the CDC has faced similar challenges with communication, with former CDC Chief Medical Officer Debra Houry telling Senators that she learned of changes to the CDC’s COVID-19 vaccine guidance from an X social media post—messaging she said she could not use to implement new public health guidance.) Setting aside the unusual modes of communication, policies implemented by the FDA in 2025 have caught their share of controversy; from the requirement announced in April that all new vaccines be tested in placebo-controlled trials to the introduction of the Commissioner’s National Priority Voucher program in June—a program whose intent and feasibility has been questioned by experts . One area where industry responded with some positivity was in the cell and gene therapy space—particularly around those therapies being developed for rare and ultrarare diseases—where myriad initiatives and guidances focused on accelerating their path to the market. The FDA’s Rare Disease Evidence Principles framework, introduced in September, is intended to streamline the approval process for therapies targeting diseases affecting less than 1,000 people in the U.S., while a trio of guidances published later that month elucidated the FDA’s thinking on how to speed cell and gene therapies specifically. But many questions remain about the implementation of these and other policy changes, especially in light of unprecedented churn at the agency, which has experienced a near-complete turnover of senior leadership since Donald Trump was elected U.S. president. BioSpace learned in the spring after the forced resignation of CBER director Peter Marks that more than half of FDA leaders had left the agency since the presidential election. By December, after the sudden retirement of newly appointed CDER Director Richard Pazdur, that number was up to almost 90%. Only three names on the organizational chart from a year ago remain today. Going into 2026, industry is calling for consistency and stability at the U.S. drug regulator. Vaccines in the Cross Hairs of Health Secretary Robert F. Kennedy Jr. Observers feared that the Trump administration, along with new Health secretary and frequent vaccine opponent Robert F. Kennedy Jr., would make sweeping changes to federal vaccine policy. The administration validated that concern almost immediately. In a post on X in June, Kennedy announced that he was removing the CDC’s COVID-19 vaccination recommendation for healthy children and pregnant women. A few weeks later, he removed all 17 members of the CDC’s influential Advisory Committee on Immunization Practices, replacing them with new members more aligned with his antivaccine views. Over the summer, Kennedy also pulled back funding from vaccine research, to the tune of $500 million for mRNA-based projects. That funding would’ve gone to vaccine makers like AstraZeneca, Moderna, Pfizer and Sanofi but was pulled because “the data show these vaccines fail to protect effectively against upper respiratory infections like COVID and flu,” Kennedy said in a statement from HHS, without providing evidence. The drama continued in August, as newly confirmed CDC head Susan Monarez was fired after refusing to pre-approve ACIP vaccine recommendations, she alleged. The following month, ACIP recommended splitting up the stalwart MMRV—measles, mumps, rubella and varicella (chicken pox)—shot into two separate MMR and V doses. And in its most recent meeting in December, the committee recommended delaying the hepatitis B vaccine birth dose—in use for 30 years with no prior issues—to two months post birth for babies whose mothers test negative for the virus, a move that came amid multiple condemnations from former CDC and FDA chiefs on the CDC’s policy changes. The CDC formally adopted that recommendation shortly afterward. To cap the year off, the FDA’s Vinay Prasad wrote, in a leaked internal memo, that COVID-19 vaccines had been linked to at least 10 pediatric deaths, without offering evidence. The FDA promised that that data would come soon. Soon after, news broke that the FDA planned to put a black box safety warning on COVID-19 vaccines—though in the meantime, the CDC published data showing that COVID-19 vaccines are effective at protecting children against disease. Eleven days after the FDA promised to release data relevant to Prasad’s claim, a report broke that internal research at the agency suggested the number of deaths linked to COVID-19 vaccines was between zero and seven, and that none of the seven potential deaths were conclusively tied to vaccination. The following day, Makary contradicted the reports of the FDA putting a black box warning on COVID-19 vaccines. Shares of Moderna and fellow vaccine biotech BioNTech both dropped about 20% this year, while four pharma giants— GSK , Pfizer, Sanofi and Merck—all reported declining vaccine sales due to dropping immunization rates in the U.S. Vaccines Pesky ‘Macro Factors’—aka RFK Jr.—Come for Vaccine Pharmas’ Wallets Following restricted vaccine approvals and changes to CDC immunization schedules, Merck, Pfizer, GSK and Sanofi are all suffering revenue hits to their vaccine programs. November 7, 2025 · 4 min read · Annalee Armstrong

全球医疗行业每日重点资讯 文 | 苏丁 企业动态 知情人士透露，美国白宫即将宣布与制药巨头诺华和罗氏控股达成药品定价协议。诺华证实与美国政府进行磋商，并致力于“寻找建设性的解决方案，以降低美国民众的药品成本，并解决美国与其他高收入国家之间的价格差距”。Genentech发言人表示，该公司致力于与美国政府合作，支持其降低美国药品成本、同时鼓励其他国家“奖励生物制药创新”的目标。辉瑞、阿斯利康等公司近月来也与特朗普政府达成协议，同意在某些情况下降低价格，以换取关税减免。 美国医疗用品供应商Medline募资约62.6亿美元，成为今年美国规模最大的首次公开募股，反映出投资者对大型医疗健康企业重新燃起兴趣。Medline股票12月17日正式登陆纳斯达克交易所，当日收盘价格为41美元，总市值757亿美元。Medline生产并经销医院与医生使用的医疗耗材，包括手套、手术衣、检查台等。截至9月27日的过去九个月，公司营收206亿美元，净利润9.77亿美元。 Addition Therapeutics完成1亿美元融资。依托RNA介导精准转基因插入（PRINT）技术平台，该公司旨在开发更安全、持久的一次性治疗方案，以克服现有基因医学技术路线的局限性。Addition正在推进基于PRINT技术的治疗产品管线，旨在从根本上重新定义慢性疾病和罕见疾病的治疗方式。 诊断影像服务运营商 Lumexa Imaging Holdings 在纳斯达克完成首次公开募股（IPO）。公司以每股 18.5 美元的价格发行 2500 万股普通股，合计募集资金约 4.63 亿美元。 美的医疗近日正式发布具备国产自主知识产权的“医学影像多模态智能诊断大模型”。该模型目前可一次性自动检测出包括肺结核、肺炎、气胸、骨折等在内的常见胸部疾病，并生成诊断报告。美的医疗旗下万里云研发团队还创新推出了可同时满足设备端与云端灵活部署的“双量级”模型，增强了临床应用的适应性。当前模型主要应用于胸部DR诊断，并计划于2026年迭代拓展至骨科DR领域。 华润医药集团有限公司发布与华润健康2026年-2028年的销售框架协议，协议总金额高达57.5亿元。华润医药需向华润健康及其管理的医院供应医疗、医药产品及医疗器械等，包括但不限于处方药、非处方药。本次为持续关联交易的深化合作，将合作期延长至3年，年度金额上限亦提升至每年的18.5亿元、19亿元和20亿元，逐年递增。 产业动态 葛兰素史克称其治疗重度哮喘的药物已获得美国批准，愈发有希望成为一款明星产品。该公司在声明中称，美国食品药品监督管理局(FDA)已批准Exdensur用作12岁及以上重度哮喘患者的附加维持治疗药物。 赛诺菲宣布，第二代心肌肌球蛋白抑制剂(CMI)星舒平(阿夫凯泰片)正式获得国家药监局(NMPA)批准，用于治疗纽约心脏协会(NYHA)心功能分级II-III级的梗阻性肥厚型心肌病(HCM)成人患者，以改善运动能力和症状。此次获批标志着阿夫凯泰片在中国实现“全球首发”，早于美国与欧盟等全球其他国家与地区。 赛诺菲公布其药物tolebrutinib在治疗原发进展型多发性硬化症（PPMS）方面的最新进展。PERSEUS 3期研究结果显示，托莱布替尼在延缓原发性进展型多发性硬化症患者出现6个月复合确认残疾进展的时间方面，未达到其主要终点。PPMS患者约占整体多发性硬化症患者群体的10%。基于这些结果，赛诺菲将不会寻求托莱布替尼在PPMS适应症上的监管注册。 Caris Life Sciences公司宣布，其治疗研究部门Caris Discovery已与罗氏（Roche）旗下基因泰克（Genentech）达成一项多年期合作与许可协议。根据该合作，Caris将致力于在实体瘤组织中发现并验证全新的肿瘤学靶点。根据协议条款，Caris有资格获得2500万美元的预付款及近期付款，并有资格获得最高达11亿美元的潜在研发、开发和商业化里程碑付款。Caris依托其包含近50万例实体瘤样本的庞大资源库，以及与之匹配的综合分子与临床数据，结合基于组织和以数据为核心的多种技术手段，为生物医药合作伙伴提供先进且灵活的靶点发现能力。 美敦力全球首个“脑机接口”脑起搏器Percept RC正式在国内各大医院开启临床应用。它是全球首款应用脑机接口技术的可充电、可感知闭环脑起搏器，于2025年11月在第八届中国国际进口博览会上首次亮相中国，并已获国家药品监督管理局批准用于帕金森病、特发性震颤、肌张力障碍和药物难治性癫痫等神经系统疾病的治疗。 康泰生物研发的重组乙型肝炎疫苗（酿酒酵母）（60μg）新增适用人群项目获得国家药品监督管理局出具的《药物临床试验批准通知书》。经审查，本品符合药品注册的有关要求，批准本品在慢性乙型肝炎功能性（临床）治愈人群中开展预防HBsAg复阳的临床试验。 国家药品监督管理局正式批准齐鲁制药神经节苷脂钠注射液（商品名：申捷）用于治疗急性缺血性脑卒中（AIS）的新适应症。 联系美通社 +86-10-5953 9500 info@prnasia.com