Gepotidacin

除此之外，声明：因水平有限，错误不可避免，或有些信息非最及时，欢迎留言指出。本文仅作医疗健康相关药物介绍，非治疗方案推荐（若涉及）;本文不构成任何投资建议。据统计，截至6月30日，今年上半年共有16款创新药获FDA批准上市，其中包括9款化药新药和7款生物药新药。从适应症来看，肿瘤药物仍占据获批数量之首，共有7款，涵盖肺癌、乳腺癌、鼻咽癌、卵巢癌等领域。此外，还有4款罕见病药物获批上市，适应症分别为遗传性血管水肿、血友病、神经纤维瘤、腱鞘巨细胞瘤。整理如下：其中，有一款来自中国，即康方生物自主研发的PD-1单抗药物派安普利单抗注射液(商品名：安尼可®)在今年4月获美国FDA批准上市，用于治疗复发或转移性鼻咽癌(NPC)的一线治疗和以铂类为基础的至少一线化疗治疗进展后的2项适应症。派安普利单抗是由康方生物自主研发，并由康方生物与正大天晴负责后续开发和商业化。该产品是采用IgG1亚型并进行Fc段改造的PD-1单抗。其Fc段改造通过消除与FcγR的结合，避免了抗体依赖性细胞吞噬(ADCP)和细胞因子释放(ADCR)，减少效应T细胞的非特异性耗竭，从而提升抗肿瘤活性并降低全身免疫毒性。值得注意的是，派安普利单抗不是第一个获FDA批准的国产抗PD-1单抗药物。2023年10月，君实生物的Toripalimab获得FDA批准用于鼻咽癌治疗；2024年3月，百济神州的tislelizumab也获FDA批准用于食管鳞状细胞癌。派安普利单抗是第三款获FDA批准的国产PD-1单抗。除此之外，上半年获FDA批准的产品还包括第一三共和阿斯利康的Datroway、艾伯维的 Emrelis两款ADC药物，以及Vertex Pharmaceuticals的Journavx、GSK的Blujepa、赛诺菲和Alnylam的Qfitlia、强生的Imaavy 、CSL Behring的Andembry等创新药。DatrowayDatroway是一款由人源化、靶向TROP2的单克隆抗体与创新DNA拓朴异构酶Ⅰ抑制剂(Dxd)通过可裂解的四肽连接子偶联生成的ADC药物获批适应症为HR阳性、HER2阴性乳腺癌。EmrelisEmrelis由c-Met结合抗体、可裂解连接体和微管蛋白抑制剂MMAE有效载荷组成，以特异性和高亲和力结合靶向表达c-Met的肿瘤细胞，获批适应症为c-Met高表达非小细胞肺癌。是首个批准用于治疗c-Met过度表达的经治疗晚期NSCLC的疗法。QfitliaQfitlia是一种可治疗所有血友病类型的siRNA疗法。3月28日，FDA批准赛诺菲和Alnylam联合开发的siRNA疗法Qfitlia上市，用于预防或减少12岁以上血友病A和血友病B患者的出血事件。Qfitlia通过降低抗凝血酶水平，从而促进凝血酶生成，重新平衡止血功能并预防出血。其中在美上市的药物哪些有望登陆中国市场？据不完全统计，在已经公开国内上市进展的药物中，上述跨国药企的新药中至少有4款已进入我国药品审评阶段。其中，多款新药被我国纳入优先审评。强生的尼卡利单抗Imaavy（用于治疗全身型重症肌无力）已被国家药品监督管理局药品审评中心纳入优先审评，有望加速获批；诺华的IgA肾病新药阿曲生坦，已于2024年11月递交上市申请，并被纳入优先审评。此外，进展较快的还有赛诺菲及Alnylam共同研发的血友病新疗法Qfitlia，国家药品监督管理局药品审评中心已在2024年受理其上市申请，预计在中国的审批决定将在2025年下半年公布。参考资料：公开资料-END-扫码报名参会

GSK recently scored a regulatory success against complicated UTIs when the FDA approved Blujepa.\n GSK’s decision to swoop in and save Spero Therapeutics’ antibiotic three years ago appears to have paid off based on a fresh phase 3 readout.The trial of 1,690 hospitalized patients with complicated urinary tract infections (cUTIs) has been halted early after it looked certain that the candidate, called tebipenem HBr, would hit the study’s primary endpoint of showing its non-inferiority to intravenous treatment with the antibiotic imipenem/cilastatin.Armed with the new phase 3 data, GSK is planning its own push for a potential FDA review this year. If it gets the nod from the regulator, tebipenem HBr would become the first oral carbapenem antibiotic available for U.S. patients with cUTIs, GSK pointed out in a May 28 release.“Complicated UTIs can have a profound impact on patients and carry a high risk of clinical complications, including sepsis and septic shock,” GSK Chief Scientific Officer Tony Wood, Ph.D., said in the release.“Currently, many need hospital-based intravenous treatment due to limited oral options for drug-resistant infections, contributing to over $6 billion per year in U.S. healthcare costs,” Wood added. “These positive results add to our growing anti-infectives portfolio and reinforce the potential of tebipenem HBr as an effective oral alternative taken at home.”GSK recently scored a regulatory success against uncomplicated UTIs when the FDA approved its antibiotic Blujepa, also known as gepotidacin, in March. The Big Pharma saw similar promise in tebipenem HBr back in 2022, despite the FDA having at the time recently rejected Spero’s approval application. The biotech used the $66 million upfront payment it received from GSK as part of the licensing deal to fund the phase 3 trial that read out today.“Achieving the primary endpoint in the PIVOT-PO trial marks a significant milestone for tebipenem HBr,” Spero’s CEO Esther Rajavelu said in this morning’s release. “If approved, we believe tebipenem HBr is well-positioned to change the treatment landscape for patients diagnosed with cUTI, including pyelonephritis.”The late-stage win for tebipenem HBr is a welcome boost for Spero, which has spent the past year navigating layoffs and stripping back its pipeline. This included halting work on the oral antibiotic SPR720 after it failed to beat placebo and showed signs of liver toxicity in a phase 2 trial for non-tuberculous mycobacterial pulmonary disease.The low return on investment means many of the biggest biopharmas have given up working on new antifungals or antibiotics in recent years—although GSK in particular has continued to sign deals and post encouraging clinical results against infections like gonorrhea.