Vusolimogene oderparepvec

编者按：2025年，美国FDA旗下的药物评价和研究中心（CDER）批准了46款创新药。作为创新赋能者、客户信赖的合作伙伴以及全球医药及生命科学行业的贡献者，药明康德将持续通过独特的“CRDMO”业务模式，助力更多合作伙伴，为全球病患带来突破性创新疗法。根据PDUFA的目标日期，预计2026年4月，美国FDA将对4款创新药物是否获批做出监管决定，本文将对这些疗法进行相关介绍。 ▲4月美国FDA可能批准的新药（点击可见大图） 活性成分：Orca-T 适应症：血液恶性肿瘤 公司名称：Orca Bio Orca-T由富集的CD34+干细胞，以及通过高精度平台纯化、取材自单倍体匹配供体的特定T细胞亚群组合而成。Orca-T有潜力改善患者的预后，并降低移植物抗宿主病风险，并且无需对无法找到人类白细胞抗原（HLA）匹配的患者进行移植后环磷酰胺（PTCy）治疗。 美国FDA已授予Orca-T的生物制品许可申请（BLA）优先审评资格，用于治疗包括急性髓系白血病（AML）、急性淋巴细胞白血病（ALL）和骨髓增生异常综合征（MDS）在内的血液恶性肿瘤，该申请的PDUFA日期为2026年4月6日。BLA的提交主要基于关键3期临床试验Precision-T的积极结果。 Precision-T是一项随机、开放标签、多中心临床试验，旨在评估Orca-T与传统同种异体造血干细胞移植（alloHSCT）在上述疾病患者中的安全性和有效性。结果显示，试验达到主要终点，Orca-T在无中重度慢性移植物抗宿主病（cGvHD）生存率方面相较传统移植显示出统计学显著且具有临床意义的改善。一年时，Orca-T组无中重度cGvHD生存率为78%，而alloHSCT组为38%（HR=0.26，p<0.00001）；总生存率方面，Orca-T组达94%，alloHSCT组则为83%；中重度cGvHD累积发生率分别为13%与44%。 活性成分：Vusolimogene oderparepvec（RP1） 适应症：晚期黑色素瘤 公司名称：Replimune RP1是一款基于单纯疱疹病毒（HSV）的溶瘤病毒免疫疗法。它同时表达GALV-GP R-蛋白和粒细胞巨噬细胞集落刺激因子（GM-CSF），旨在增强肿瘤杀伤效力，提高癌细胞死亡的免疫原性，并激活全身性的抗肿瘤免疫反应。RP1通过注射至浅表和/或深层及内脏肿瘤病灶来治疗患者。它曾经获得FDA授予的突破性疗法认定。 美国FDA已接受RP1联合PD-1抑制剂nivolumab用于治疗在抗PD-1方案下出现疾病进展的晚期黑色素瘤患者的生物制品许可申请。该申请的PDUFA日期定为2026年4月10日。 该BLA的支持数据来源于IGNYTE试验的主要分析结果，该试验评估了RP1联合nivolumab治疗黑色素瘤患者的疗效。这些患者曾接受过PD-1靶向疗法治疗后疾病出现进展。在2024年第39届癌症免疫治疗学会年会（SITC 2024）中公布的结果显示，约三分之一的患者获得确认缓解。根据FDA要求的RECIST v1.1标准评估，患者的总缓解率（ORR）为32.9%。根据修正RECIST评估方案（mRECIST）v1.1评估的完全缓解（CR）率为15%。在既往接受过抗PD-1和抗CTLA-4疗法的患者中，ORR为27.7%；而在对抗PD-1初始耐药的患者中，ORR为35.9%。患者的中位缓解持续时间（DOR）为21.6个月。 活性成分：Orforglipron 适应症：肥胖或超重 公司名称：礼来 Orforglipron是一种在研、每日一次的小分子口服胰高血糖素样肽-1（GLP-1）受体激动剂，可在一天中的任何时间服用，不受进食和饮水限制。礼来（Eli Lilly and Company）公司在2025年12月宣布，已向美国FDA递交orforglipron用于治疗成人肥胖或超重的新药申请。 2025年8月公布的3期临床试验ATTAIN-2的结果显示，针对肥胖或超重并伴有2型糖尿病的成年人，orforglipron的三种剂量均达到了主要和所有关键次要终点。在为期72周的治疗中，药物带来了显著的体重下降、糖化血红蛋白水平有意义的降低，并改善了心脏代谢风险因素。12月公布的3期临床试验ATTAIN-MAINTAIN的积极顶线结果显示，orforglipron在维持体重减轻方面也达到临床试验的主要和关键次要终点。 活性成分：Doravirine/islatravir 适应症：HIV-1感染 公司名称：默沙东 Doravirine/islatravir（DOR/ISL）是默沙东（MSD）开发的复方口服药物。其中doravirine是一种口服创新非核苷逆转录酶抑制剂。它通过与HIV-1病毒的逆转录酶结合，防止HIV-1病毒将RNA转化为DNA，从而阻断HIV-1病毒复制。它已在美国获批用于治疗未接受过抗病毒疗法的成年HIV-1感染患者。 Islatravir则是一种核苷类逆转录酶易位抑制剂（NRTTI），具有转录酶和易位抑制作用（可防止核苷酸结合和掺入DNA链，导致链立即终止）和延迟链终止作用（即使在易位的情况下也能防止核苷酸掺入）。 美国FDA已受理DOR/ISL的新药申请，用于治疗病毒学抑制的HIV-1感染成人患者。该申请的PDUFA日期为2026年4月28日。 这一新药申请基于两项关键3期临床试验（MK-8591A-051和MK-8591A-052）第48周的数据。在MK-8591A-051试验中，DOR/ISL在开放标签设计下展现出不劣于基线抗病毒治疗的疗效；而在随机双盲对照的MK-8591A-052试验中，该方案亦被证实不劣于三联复方治疗方案克替拉韦/恩曲他滨/替诺福韦艾拉酚胺（BIC/FTC/TAF）。两项研究中，DOR/ISL的安全性整体与对照治疗方案相当，支持其作为一种简化治疗的新选项，有望助力HIV治疗方案进一步优化。 参考资料： [1] U.S. FDA Accepts New Drug Application for Merck’s Doravirine/Islatravir, an Investigational, Once-Daily, Oral, Two-Drug Regimen for Treatment of Adults with Virologically Suppressed HIV-1 Infection. Retrieved March 30, 2026, from https://www.merck.com/news/u-s-fda-accepts-new-drug-application-for-mercks-doravirine-islatravir-an-investigational-once-daily-oral-two-drug-regimen-for-treatment-of-adults-with-virologically-suppressed-hiv-1-infe/ [2] Exclusive: US FDA delays two drug reviews in new voucher program after safety, efficacy concerns. Retrieved March 30, 2026, from https://www.reuters.com/sustainability/boards-policy-regulation/us-fda-delays-two-drug-reviews-new-voucher-program-after-safety-efficacy-2026-01-15/ [3] Orca Bio Presents New Data at the 2026 Tandem Meetings of ASTCT® and CIBMTR® Reinforcing Orca-T® as a Durable, High-Precision Cell Therapy for Hematological Malignancies. Retrieved March 30, 2026, from https://orcabio.com/orca-bio-presents-new-data-at-the-2026-tandem-meetings-of-astct-and-cibmtr-reinforcing-orca-t-as-a-durable-high-precision-cell-therapy-for-hematological-malignancies/ 免责声明：本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。 版权说明：欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。 分享，点赞，在看，聚焦全球生物医药健康创新

iStock/ Grandbrothers The FDA has some big verdicts lined up in the second quarter, including one for a closely watched obesity drug that many anticipate will further intensify competition in weight loss. The second quarter is looking particularly front-loaded for the FDA, which in April is expected to make a handful of highly consequential decisions. One is for a rare disease with high unmet need, while another, for a melanoma treatment, could be a bellwether for how things might change at the agency after the impending departure of a controversial leader. Another key verdict in April is for Eli Lilly’s orforglipron, a weight loss pill that many expect will give Novo Nordisk’s oral Wegovy a run for its money. Here, BioSpace looks at some of the biggest upcoming regulatory decisions expected in the next three months. Biogen Hopes To Bounce Back from High-Dose Spinraza Rejection Following a surprise rejection in September last year, Biogen is once again awaiting the FDA’s word on its high-dose formulation of the antisense oligonucleotide Spinraza, being proposed for the treatment of spinal muscular atrophy (SMA). The agency is expected to release a decision on or before April 3. Biogen is supporting its application with data from the Phase 2/3 DEVOTE study, which in September 2024 demonstrated that higher-dose Spinraza—comprising two 50 mg loading doses 14 days apart and a maintenance regimen of 28 mg every 4 months—led to better improvements in motor skills versus sham control. The higher-dose formulation of the drug likewise outperformed the standard-dose version in this regard, though the company at the time did not release its statistical analysis for this comparison. At the recently concluded 2026 meeting of the Muscular Dystrophy Association, Biogen posted updated data from DEVOTE—as well as from an open-label Phase 3 study called ONWARD—confirming that the higher-dose formulation of Spinraza is significantly better than sham at improving motor skills. Higher-dose Spinraza likewise outperformed sham in terms of secondary endpoints such as neurofilament light chain levels and event-free survival. The FDA’s rejection of Spinraza in September 2025 was not due to the drug’s data package but was instead linked to manufacturing issues . Moment of Truth for Lilly’s Closely Watched Weight Loss Pill In what could be its most consequential verdict for the weight loss space this year, the FDA will render a decision on Eli Lilly’s oral obesity medicine orforglipron on or before April 10. Analysts expect orforglipron to help Lilly’s trizepatide franchise—consisting of the diabetes drug Mounjaro and the weight management brand Zepbound—to exceed $100 billion in peak revenue. If approved, orforglipron will challenge Novo Nordisk’s oral Wegovy, which has a three-month headstart after winning approval late last year . Despite Novo’s first-to-market advantage, Lilly might have an efficacy edge. Data from the Phase 3 ACHIEVE-3 study released last month showed that orforglipron elicited greater weight loss and better glycemic control than oral semaglutide—marketed as Rybelsus for diabetes—in patients with type 2 diabetes whose condition is inadequately controlled by metformin. The FDA awarded orforglipron its Commissioner’s National Priority Voucher in November 2025, promising a 1-2-month review period. However, the agency delayed its decision in January by two weeks, according to Reuters . If approved, Lilly will be ready to launch orforglipron quickly. The pharma has already stockpiled some $1.5 billion worth of the drug, ready for deployment, and has been ramping up production capacity since February 2024. FDA FDA Delays Decision for Lilly’s Obesity Pill Orforglipron, Other Priority Voucher Awardees: Report Target action dates for drugs sponsored by Sanofi, Boehringer Ingelheim and Disc Medicine have also been pushed back despite assurances of swift reviews under the FDA’s new Commissioner’s National Priority Voucher program. January 15, 2026 · 2 min read · Tristan Manalac Read more Replimune Gives Tumor Destroyer Another Try Like Biogen, Replimune is looking to bounce back from a rejection. The FDA is expected to release a decision for the company’s oncolytic immunotherapy RP1 for advanced melanoma by April 10. The regulator rejected RP1 in July last year, claiming that Replimune’s pivotal IGNYTE study was not adequate and well-controlled and therefore could not support an approval. This feedback, Replimine CEO Sushil Patel said at the time, was “not raised by the agency during the mid- and late-cycle reviews.” Analysts at BMO Capital Markets had a more pointed analysis of the rejection, indicating that Vinay Prasad, director of the FDA’s Center for Biologics Evaluation and Research, played a role. “Prasad has previously been critical of the approvability of uncontrolled data, and today we see that opinion underscored,” the firm wrote in a July 22, 2025 note to investors. In October last year, Replimune resubmitted its data package for RP1, beefing it up with additional data and analyses. Also potentially playing in Replimune’s favor is the impending departure of Prasad, who will leave the FDA at the end of April. RP1 makes use of a proprietary and engineered herpes simplex virus that carries a fusion protein. Taken together, this construct seeks out and destroys cancer cells, while also altering the microenvironment and stimulating the body’s antitumor immune response. RP1 is meant to be used in combination with Bristol Myers Squibb’s Opdivo. Immuno-oncology Replimune Plummets Following FDA’s Surprise Rejection of Melanoma Treatment BMO Capital Markets pointed to FDA leadership, and CBER Director Vinay Prasad in particular, as potential factors in the agency’s decision to issue a complete response letter for Replimune’s viral treatment RP1 for advanced melanomas. Shares of the company tumbled 75% on Tuesday. July 22, 2025 · 1 min read · Dan Samorodnitsky Read more Axsome Awaits FDA Action on Alzheimer’s Agitation Drug By April 30, the FDA will release its verdict on Axsome Therapeutics’ oral drug AXS-05 for the treatment of agitation in patients with Alzheimer’s disease. Around 7 million people in the U.S. have Alzheimer’s, according to Axsome, 76% of whom experience agitation characterized by distress, aggressiveness and irritability. AXS-05 combines two drugs—dextromethorphan and bupropion—and works by blocking the NMDA receptor and activating the sigma-1 receptor, while also inhibiting the aminoketone CYP2D6 enzyme. In January 2025, Axsome posted mixed late-stage data for the drug. In the Phase 3 ACCORD-2 study, AXS-05 reduced the risk of relapse of agitation by 3.6-fold compared with placebo. A smaller Phase 3 study dubbed ACCORD-1 likewise met its primary endpoint of delaying time to relapse in 2022. However, in the Phase 3 ADVANCE-2 trial, which randomized more than 400 patients, Axsome’s drug failed to significantly outperform placebo in terms of easing agitation. The biotech nevertheless said at the time that it would move forward with an application. AXS-05 is already approved in the U.S. under the brand name Auvelity for the treatment of major depressive disorder. After Maintenance Win, Biogen, Eisai Target SubQ Expansion for Leqembi Initiation In September last year, the FDA cleared a subcutaneous formulation of Biogen and Eisai’s anti-amyloid Alzheimer’s disease therapy Leqembi, allowing it to be delivered via an under-the-skin injection rather than IV—but only in patients who have already completed 18 months of treatment with the drug. Now, the partners are looking to expand the subcutaneous injection, branded as Leqembi Iqlik, to the induction setting. The FDA is currently reviewing the proposal and is expected to release a decision by May 24. In their data package, Biogen and Eisai provided data showing that a weekly subcutaneous injection provides bioequivalent drug exposure to an intravenous infusion given once every two weeks. If approved, Leqembi Iqlik would become the first anti-amyloid therapy that patients can inject at home both for induction and maintenance treatment, according to a January 2026 press announcement from the companies. An approval is “going to be also extremely important,” Biogen CEO Christopher Viehbacher told investors at the company’s J.P. Morgan presentation in January. The chief executive noted that Biogen has a competitor in the anti-amyloid antibody space, “and the competitor offers once-monthly infusion versus our every 2-week infusion.” This competitor would be Eli Lilly’s Kisunla. “That advantage is going to go away once we have a subcutaneous formulation,” Viehbacher said. Neurodegenerative disease JPM26: With Leqembi Launched, Biogen Looks To Expand Alzheimer’s Presence Three years after the accelerated approval of its anti-amyloid Alzheimer’s therapy, Biogen—neck and neck in the market with Eli Lilly and its Kisunla offering—is focused on a near-term FDA decision for a subcutaneous induction dose of Leqembi, a presymptomatic readout in 2028 and a clutch of next-generation candidates. January 14, 2026 · 5 min read · Heather McKenzie Read more Review Comes to Close for AstraZeneca’s Lipid-Lowering Pill AstraZeneca is looking to open up a new drug class. The company is awaiting the FDA’s word on its aldosterone synthase inhibitor baxdrostat for patients with uncontrolled or treatment-resistant hypertension, which, if approved, would be the first aldosterone synthase blocker to pass the FDA’s muster, AstraZeneca said in December last year . The pharma hasn’t specified a target action date for the application, only revealing that a decision should come in the second quarter. The pharma is backing baxdrostat with data from the Phase 3 BaxHTN study, in which a 2-mg dose of the drug demonstrated an up to 9.8-mmHg placebo-corrected reduction in systolic blood pressure, according to an August 2025 publication in The New England Journal of Medicine . BaxHTN used baxdrostat on top of standard of care and also aced all of its secondary endpoints. The drug, taken orally, works by inhibiting the aldosterone synthase enzyme, which otherwise produces a steroid that promotes sodium retention, in turn maintaining blood pressure levels. AstraZeneca obtained baxdrostat from its $1.3 billion takeover of CinCor Pharma in 2023.