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For many people, the end of the year is a mad rush to wrap things up before the holidays, and so it was for the FDA. Notable regulatory decisions include the first drug approval for a prevalent chronic condition and a novel regenerative medicine approach to help trauma patients. In one case, a new drug approval comes as its developer takes on a new identity in the new year. Here’s a look back at some highlights from a busy regulatory month: presented by Health IT Transforming Patient Care: The Role of AI-Powered Assistants The progress in artificial intelligence (AI) is reshaping patient care, across various dimensions, from facilitating faster discharge to curating treatment plans and suggesting lifestyle changes. By IT Medical Notable Firsts —The prevalent sleeping disorder obstructive sleep apnea has historically been managed with a medical device that helps breathing. Eli Lilly’s Zepbound is now the first FDA-approved drug treatment for the chronic condition. Obesity is a risk factor for sleep apnea and clinical trial results showed that weight reductions from treatment with Zepbound were accompanied by breathing improvement. Approval in obstructive sleep apnea adds another potential blockbuster indication for a metabolic medication that has fast become one of Lilly’s top-selling products. —Patients who have the rare inherited metabolic disorder familial chylomicronemia syndrome lack the ability to break down triglycerides, a type of fat from food. The only way to avoid potentially fatal complications to the pancreas is by maintaining an extremely restrictive diet. Approval of Ionis Pharmaceuticals’ olezarsen gives patients a therapeutic option. The once-monthly injected genetic medicine, which is designed to block the body’s production of a liver protein that regulates triglyceride metabolism, will be marketed under the brand name Tryngolza. Ionis previously brought drugs through late-stage development and commercialization under partnerships with larger companies. Tryngolza will be the first product Ionis commercializes on its own. —When trauma to an arm or leg requires replacement of a blood vessel, the standard treatment is grafting a vein from the patient or implanting a synthetic vein. Now there’s a new regenerative medicine option. Humacyte won FDA approval for Symvess, a bioengineered blood vessel for restoring blood flow to avoid the loss of a limb when grafting a vein from the patient is not feasible. Here’s more about the biotech’s regenerative technology. Sponsored Post The Funding Model for Cancer Innovation is Broken — We Can Fix It Closing cancer health equity gaps require medical breakthroughs made possible by new funding approaches. By Eve McDavid - CEO of Mission-Driven Tech —Mesoblast’s regulatory approval was a long time coming. In 2020 and 2023, the FDA turned down the Australian company’s application for remestemcel, an allogeneic cell therapy for graft versus host disease, an immune response that develops when donor T cells attack the recipient’s cells following a transplant procedure. Remestemcel, brand name Ryoncil, is a made from mesenchymal stromal cells sourced from the bone marrow of healthy donors. The product’s approval covers acute graft versus host disease that is refractory to treatment with steroids in patients age 2 months and older. It’s the first affirmative regulatory decision for a cell therapy made from mesenchymal stromal cells. Approvals in Immunology —Vtama, an Organon drug acquired from Roivant Sciences earlier this year, received FDA approval as a treatment for atopic dermatitis in adults and children age 2 and older. The drug is topical cream that was initially approved in 2022 as a treatment for plaque psoriasis. Approval in atopic dermatitis brings the product to a much larger dermatologic indication, albeit one served my many branded and generic medications. —In other atopic dermatitis news, Galderma landed FDA approval for nemolizumab, brand name Nemluvio. The drug is an antibody designed to block IL-31, a signaling protein associated with the itch and inflammation of the chronic skin disorder. FDA approval of Nemluvio covers use of the drug in patients age 12 and older who have moderate-to-severe atopic dermatitis. It’s the second approval in the past year for Nemluvio, which was first approved over the summer as a treatment for prurigo nodularis. Rare Disease Regulatory Decisions —Neurocrine Biosciences received approval for Crenessity, a treatment for the rare inherited hormone disorder congenital adrenal hyperplasia. The small molecule helps bring the hormone imbalance back to more normal levels. The FDA approved a pill formulation for adults and an oral solution for pediatric patients. Analysts project Crenessity could achieve blockbuster sales, pending regulatory approvals in other countries. —Novo Nordisk is best known for metabolic medicines, but its rare disease portfolio is getting a boost with FDA approval of Alhemo, a drug that reduces bleeding episodes in patients with either hemophilia A or B. Alhemo, known in development as concizumab, is an antibody designed to bind to TFPI, preventing that protein from blocking factor Xa, a different protein that plays a role in blood clotting. That’s the same mechanism of action as Pfizer’s Hympavzi, which won its FDA approval in October. Both drugs are subcutaneous injections that provide alternatives to intravenously infused hemophilia therapies. But Pfizer’s once-weekly Hmypavzi has a dosing edge over Alhemo, which must be injected once daily. —Vertex Pharmaceuticals is adding a new cystic fibrosis (CF) drug to its portfolio with FDA approval of Alyftrek, which combines three compounds in a single therapy. Like Vertex’s other CF therapies, Alyftrek is a modulator of the CFTR a protein that regulates the movement of chloride ions into and out of cells. Alyftrek’s approval is based on clinical data comparing the once-daily therapy to Trikafta, a Vertex triple combination drug initially approved in 2019 as a twice-daily CF treatment. Results showed Alyftrek was non-inferior to Trikafta on a key measure of lung function and was superior in reducing sweat chloride levels, which is a surrogate indicator of the function of CFTR proteins. Besides the dosing advantage, Alyftrek addresses 31 additional mutations that are not addressable by other CFTR modulators. The Dec. 20 approval of Alyftrek came nearly two weeks ahead of the Jan. 2 target date for a regulatory decision. Developments in Cancer Drugs —The FDA awarded accelerated approval to Merus Therapeutics drug zenacutuzumab as a treatment for advanced cases of two types of cancer: non-small cell lung cancer and pancreatic adenocarcinoma. It’s the first approval for a drug that addresses a genetic signature called an NRG1 gene fusion. Netherlands-based Merus will market the bispecific antibody under the brand name Bizengri. In a deal struck days prior to the approval announcement, Partner Therapeutics licensed U.S. commercialization rights to Bizengri. —The blockbuster AstraZeneca drug Imfinzi expanded its FDA-approved uses to include limited-stage small cell lung cancer that has not progressed following concurrent platinum-based chemotherapy and radiation therapy. The checkpoint inhibitor was first approved in 2017 for bladder cancer and added extensive-stage lung cancer as a new indication in 2020. The drug’s latest approval is based on Phase 3 results showing a 27% reduction in the risk of death compared to a placebo. AstraZeneca said the FDA decision makes Imfinzi the first immunotherapy approved for limited-stage small cell lung cancer. —Xcovery Holdings drug ensartinib, brand name Ensacove, was approved to treat adults with advanced cases of non-small cell lung cancer that is positive for ALK mutations. Patients prescribed the once-daily pill must not have previously received an ALK inhibitor. —Pfizer cancer drug Braftovi landed accelerated approval as a first-line treatment for metastatic colorectal cancer driven by the BRAF V600E mutation. The approval covers use of the drug in combination with Eli Lilly’s Erbitux and the chemotherapy regimen referred to as FOLFOX, both standard colorectal cancer therapies. Braftovi, a small molecule inhibitor of BRAF V600E, was initially approved in 2018 for advanced cases of melanoma. The drug came from Pfizer’s 2019 acquisition of Array Biopharma. —Tevimbra, a cancer immunotherapy developed by BeiGene, received FDA approval as a first-line treatment for gastric and gastroesophageal junction cancers when used in combination with chemotherapy. It’s the second FDA approval for the checkpoint inhibitor, which was approved last March for treating advanced or metastatic esophageal squamous cell carcinoma after prior treatment with chemotherapy. The new year means a new identity for BeiGene. The cancer drug developer is changing its name to BeOne Medicines. Starting Jan. 2, the company’s stock symbol on the Nasdaq will be “ONC.” Rejections, Warnings & More Bad News in Biotech —Astellas Pharma’s bid to bring patients less-frequent eye injections of its drug Izervay was rejected by the FDA. Izervay, approved for treating geographic atrophy in 2023, is administered monthly to slow progression of the vision-loss disorder. Astellas sought approval for every-other-month dosing based on two-year Phase 3 data. According to Astellas, no safety benefit/risk issues were cited but the FDA took issue with a statistical matter related to labelling language proposed by the company. The rejection will limit Izervay’s ability to compete against Apellis Pharmaceuticals’ geographic atrophy drug Syfovre, which is approved for both monthly and every-other-month dosing. —In other Astellas news, the label for menopause drug Veozah now sports a black box warning for the risk of serious liver injury. The warning follows a FDA safety communication issued in September after a postmarketing report of a patient who developed signs and symptoms of liver injury after taking the once-daily pill for about 40 days. Veozah won FDA approval in 2023 as the first in a new class of therapies for menopause. —Applied Therapeutics received a double dose of bad regulatory news. First the FDA rejected the biotech’s application for govorestat, a drug developed as a treatment for the rare metabolic disease galactosemia. According to Applied, the agency cited “deficiencies in the clinical application.” Days after the FDA complete response letter, the FDA sent Applied a warning letter. The trial and the drug are redacted in the public version of the letter, but in a regulatory filing, Applied acknowledged the FDA’s concerns are about the govorestat galactosemia trial. Applied said the warning letter identified issues with electronic data capture and a dosing error in the dose-escalation portion of the study — both of which the company believed it had already addressed with the agency. Applied said it would respond to the FDA warning letter. —Bad news keeps stacking up for Intercept Pharmaceuticals and its drug, Ocaliva, a treatment for the rare liver disease primary biliary cholangitis (PBC). On Dec. 12, the FDA issued a safety alert, warning of the risk of serious liver injury in patients without advanced liver cirrhosis. The alert came one month after the FDA turned down Intercept’s application seeking full approval for Ocaliva, which won its accelerated approval in PBC in 2016. The FDA’s rejection letter for the drug cited safety concerns. When Ocaliva first reached the market, it was the only FDA-approved second-line treatment for PBC. In the past year, drugs from Ipsen and Gilead Sciences have each won accelerated approvals as new second-line therapies for the rare disease. Two weeks after the FDA turned down full approval of Ocaliva, the European Commission revoked the conditional marketing authorization for the drug. Advanz Pharma holds rights to Ocaliva in Europe. The company said the commission decision is subject to an ongoing annulment procedure in the EU’s General Court and a ruling is expected in 2025. —The FDA turned down Zealand Pharma’s glepaglutide as a treatment for short bowel syndrome, a rare disorder that develops when the small intestine is damaged or shortened, making it difficult for the organ to absorb nutrients. The drug is a long-acting GLP-2 analog intended to enhance the intestine’s ability to absorb nutrients, reducing patients’ dependence on intravenous feeding. According to Zealand, the FDA said the application needed more evidence of efficacy and safety. The company said it would discuss the letter with the FDA and proceed with plans to seek European approval in 2025. —Lexicon Pharmaceuticals came up short in its bid to expand approval of its drug, sotagliflozin (brand name Zynquista), to include the treatment of adults with type 1 diabetes and chronic kidney disease. The FDA’s Dec. 20 rejection of the drug followed a negative FDA advisory committee vote in October. The FDA approved sotagliflozin last year as a treatment for heart failure; it’s marketed in this indication under the brand name Inpefa. —Johnson & Johnson received a complete response letter for its injectable version of Rybrevant, a drug that treats cancer driven by EGFR mutations. Intravenously infused Rybrevant was approved in 2021 as a treatment for non-small cell lung cancer. The injectable version is made with technology from Halozyme. J&J said the FDA letter flagged manufacturing issues and did not cite any concerns about the new formulation or its safety and efficacy.

摘要 目的：对2023年美国FDA批准的新药进行分析，并与我国2023年新药审批相关情况进行对比分析，以供业界参考。 方法：通过查阅美国FDA CDER发布的《2023年新药审批总结报告》、我国药品审评中心发布的《2023年度药品审评报告》、Drugs@FDA数据库、国家药品监督管理局发布的药品批件及药品审评中心公开的上市药品信息，结合相关文献，收集并统计分析2023年FDA批准的新药信息，并与我国的相关情况做对比分析。 结果与结论：2023年，FDA批准了55个新药，涵盖肿瘤、遗传/罕见病、神经系统疾病等多个疾病领域。51%的新药获得了孤儿药认定，36%的新药被认定为“first-in-class”，65%的新药审批至少使用了一种加速审评程序。获批新药的企业涵盖广泛，从大型制药巨头到中小型生物技术公司均有不同数量的新药获批。我国批准新药74个，疾病领域广泛性和多样性与FDA不相上下，但新药创新性及罕见病药物的开发有待提升。38%的新药审批至少使用了一种加速上市程序。新药审评用时比2022年减少，但一次性通过率降低，反映了监管的趋严性。药企需提升申报资料质量。监管部门需完善药品监管法规，加强药品监管人员的培训和教育，提升其专业水平和国际视野。 _ 正文 _ 在当今快速发展的医药行业中，新药的批准和上市不仅是科研创新的成果展示，更是对患者健康需求的直接响应。美国作为医药强国，其新药审批对业界有重要借鉴意义。本文基于美国食品药品管理局（Food and Drug Administration，FDA）发布的《2023年新药审批总结报告》、我国国家药品监督管理局（National Medical Products Administration，NMPA）药品审评中心（Center for Drug Evaluation，CDE）发布的《2023年度药品审评报告》、Drugs@FDA数据库、NMPA发布的药品批件及CDE公开的上市药品信息，结合相关文献对2023年FDA批准的新药进行统计与分析，探讨其治疗领域分布、创新性、加速审批途径的应用以及获批企业的情况，并与我国2023年新药审批相关情况进行对比分析，供行业从业者参考。 1 获批新药的数量与治疗领域 1.1 获批新药的数量 2023年FDA药品评价和研究中心（Center for Drug Evaluation and Research，CDER）共批准了55个新药，包括38个（69.1%）根据新药申请（New Drug Application，NDA）的新分子实体（New Molecular Entitys，NMEs）和17个（30.9%）根据生物制品许可申请（Biologic License Application，BLA）的新生物制品，不包括生物制品评估和研究中心（Center for Biologics Evaluation and Research，CBER）审评的疫苗、过敏性产品、CHINESE PHARMACEUTICAL AFFAIRS血液和血液制品、细胞和基因治疗产品。2023年获批新药数量相较于2022年批准的37个增长了近50%，并高于2014-2023年的平均批准数量46个（图1）。 我国CDE2023年共批准了74个新药，包括新分子实体45个，治疗用生物制品23个和中药6个，不包括疫苗、生物类似物、新适应证和新剂型。我国2023年的新药获批数量约是FDA的1.3倍。 1.2 获批新药的治疗领域 按治疗领域分类，肿瘤依然占据了两国获批新药数量的最大比例。2023年CDER共批准了13个肿瘤新药，占所有获批新药的24%。遗传/罕见病领域仅次于肿瘤领域，获得了12个新药的批准，占比达到22%。排在第三的是神经系统疾病领域，有6个新药获得批准，占所有获批新药的11%。其他治疗领域还包括血液病5个；眼病3个；代谢/内分泌疾病、肾病、传染病、皮肤病、精神疾病和胃肠疾病各2个；生殖系统疾病、肺病、心血管疾病和影像各1个（图2）。我国批准肿瘤新药24个（32%）；其次是皮肤病领域9个（12%）；遗传/罕见病领域7个（9%）；传染病、代谢/内分泌疾病、胃肠疾病、肺病、神经系统疾病、精神疾病、免疫系统疾病、肾病、眼病和血液病等领域均有不同数量的新药获批。中美两国获批新药的疾病覆盖领域均很丰富。 1.3 中美获批新药的共同品种 值得一提的是，2023年中美获批新药中有3个共同品种，即格菲妥单抗（Columvi）、利特昔替尼（Litfulo）和艾贝格司亭α（Ryzneuta）。罗氏的格菲妥单抗与辉瑞的利特昔替尼分别在美国获批145天、118天后在中国获批上市。格菲妥单抗在中美均获得了加速审评，其在美国获得了快速通道、优先审批、加速批准资格，审批时长为227天；在我国获得突破性治疗药物、附条件批准、优先审评审批资格，审批时长为270天，略长于FDA的审批时限。利特昔替尼在FDA的审批无加速途径，审批时长为364天；其在我国获得了突破性治疗药物、优先审评审批资格，审批时长270天，相较于FDA缩短了四分之一。艾贝格司亭α在中美的审批均无加速途径，其于2021年3月30日在美国提交了生物制品许可申请，但直至2023年11月16日才获批，历时918天；其在我国的上市申请承办于2022年2月25日，于2023年5月9日早于美国获批上市，历时374天，远远低于FDA的审批时长。由此可见，我国的新药审评审批已与国际接轨，甚至部分品种审批速度快于美国，极大地提升了创新药的可及性。 2 罕见疾病治疗药物的审批 罕见疾病，又称为孤儿病，通常影响着相对较少的人群，但由于疾病种类繁多，全球患者总数依然庞大，这些疾病往往缺乏有效的治疗手段。因此，罕见疾病药物的审批对于满足患者迫切的治疗需求具有重大意义。美国FDA通过孤儿药认定等方式为罕见疾病药物的研发提供激励，包括市场独占权、税收优惠、研发资助等，以促进罕见疾病药物的研发和上市。这不仅提高了患者获得治疗的可能性，也推动了医学研究的深入和医药科技的进步。 2023年，FDA对罕见疾病治疗药物的审批表现活跃。2023年批准的新药中有28个（51%）获得了孤儿药认定（表1），表明FDA对罕见疾病治疗药物的重视。这些新药针对的罕见疾病包括但不限于Friedreich共济失调症、念珠菌病和侵袭性念珠菌病、Rett综合征、CD55缺陷型蛋白丢失性肠病（CHAPLE病）、阵发性夜间血红蛋白尿症以及活化磷脂酰肌醇3-激酶δ综合征等。2023年，CDER还批准了许多治疗罕见癌症或肿瘤的疗法，包括套细胞淋巴瘤、鼻咽癌、大B细胞淋巴瘤和筛状肿瘤。这些新药的批准，为罕见疾病患者提供了更多的治疗选择。 我国2023年批准罕见病新药8个（11%）（表2），包括阿那白滞素（家族性地中海热）、艾夫糖苷酶α（庞贝病）、纳鲁索拜单抗（骨巨细胞瘤）、卡谷氨酸（高氨血症）、苯丁酸甘油酯（尿素循环障碍）、氯马昔巴特（Alagille综合征）、硫酸氢司美替尼（丛状神经纤维瘤）和尼替西农（酪氨酸血症I型）。 虽然2023年度我国罕见病用药批准数量大幅提升，但是我国罕见病药物占批准新药的比例远低于美国。再者，我国获批的罕见病用药中仅纳鲁索拜单抗为国产新药，其余均为进口新药，我国罕见病新药的开发还有待提升。 3 获批新药的创新性 从创新角度看，CDER在2023年批准的55个新药中，有20个（占36%）被认定为首创新药（“first-in-class”新药），这些药物采用了与现有疗法不同的作用机制。它们涵盖了癌症、神经系统疾病、传染病、肾病、眼病以及罕见病等多个领域。值得关注的“first-in-class”新药包括： （1）Acadia制药公司开发了用于治疗2岁及以上儿童和成人Rett综合征的Daybue（Trofinetide），其是目前第一种也是唯一一种被批准用于治疗Rett综合征的药物。Rett综合征是一种罕见的影响大脑发育的遗传性神经系统疾病。 （2）葛兰素史克公司开发了用于治疗至少接受4个月透析治疗的慢性肾病贫血成人患者的Jesduvroq（Daprodustat），该药物是一款缺氧诱导因子脯氨酰羟化酶抑制剂，其是FDA批准的治疗慢性肾病贫血患者群体的首款口服疗法的药物。 （3）博士伦公司开发了用于治疗干眼症的体征和症状的Miebo（Perfluorohexyloctane），其是首款直接针对泪液蒸发的处方滴眼液。 （4）辉瑞公司开发了用于治疗轻度至中度COVID-19（Corona Virus Disease2019）的Paxlovid（Nirmatrelvir Tablets；Ritonavir Tablets），是奈玛特韦（150mg）和利托那韦（100mg）的组合包装，适用于有发展为重度COVID-19（包括住院或死亡）高风险的成人患者。该药物是首个获准用于治疗成人COVID-19的口服抗病毒药。 （5）Reata制药公司开发了用于治疗成人和16岁及以上青少年Friedreich共济失调症的Skyclarys（Omaveloxolone）。这是FDA批准的首款针对Friedreich共济失调症的药物。Friedreich共济失调是一种罕见的遗传性退行性疾病，会损害神经系统，表现为协调能力受损和行走困难。 （6）杨森生物技术公司开发了用于治疗复发性或难治性多发性骨髓瘤成人患者的Talvey（Talquetamab-tgvs），该药物是一款GPRC5D/CD3双特异性T细胞结合抗体，可结合T细胞表面表达的CD3受体和GPRC5D。 （7）安斯泰来公司开发了用于治疗因更年期引起的中度至重度血管舒缩症或潮热（Vasomotor Symptoms，VMS）的Veozah（Fezolinetant），该药物选择性靶向神经激肽3（Neurokinin3，NK3）受体，从而阻断NK3与基斯细胞素/神经激肽/强啡肽（Kisspeptin/Neurokinin/Dynorphin，KNDy）神经元的结合，来调节大脑体温调节中枢（下丘脑）的神经元活动，降低更年期相关的中重度VMS的发生频率和严重程度。Veozah既是FDA批准的首个用于更年期VMS的NK3受体拮抗剂，也是全球首款VMS非激素治疗药物。 （8）Tarsus公司开发了用于治疗蠕形螨睑缘炎的Xdemvy（Lotilaner），该药物是美国FDA批准的首个且唯一一个直接靶向蠕形螨睑缘炎疾病根源——蠕形螨的治疗药物。 我国2023年获批的74个新药中仅有1个“first in-class”药物——埃普奈明，作为全球首个批准上市的DR4/DR5激动剂，由武汉海特生物制药自主研发，用于治疗多发性骨髓瘤。我国药企源头创新有所突破，但是和美国相比仍存在显著差距。 4 加速审批途径的应用 CDER通过一系列快速开发和审查途径，显著提升了新药审评的效率，加速了新药的上市进程。这些加速审评途径包括快速通道（Fast Track）、突破性疗法（Breakthrough Therapy）、优先审评（Priority Review）和加速批准（Accelerated Approval），旨在为严重疾病患者提供更快的治疗选择。2023年CDER授予了25种新药快速通道资格，占当年55个新药的45%，这一途径通过增加FDA与药物开发者之间的沟通，使CDER能够滚动审查药物申请的部分内容，从而加快了药物的开发和审查过程。同时，CDER还指定了9个（16%）新药为突破性疗法，这些药物在开发过程中获得了FDA的密集指导和支持。此外，31个（56%）新药获得了优先审评资格，这意味着CDER在6个月内对这些药物的申请采取行动，相比标准审查的10个月目标日期大大缩短。此外，9个（16%）新药通过加速批准途径获批，这一途径允许基于替代终点或中间临床终点的安全性和有效性决定，为治疗严重疾病且提供显著优势的新药开放了快速上市的通道。整体来看，2023年65%的新药审批至少使用了一种加速审评程序，详见表3。 为了尽快满足临床需求，加速新药惠及患者，我国亦采取了多种加速新药审评的措施，包括纳入突破性治疗药物程序、附条件批准程序、优先审评审批程序和特别审批程序。2023年获批新药中纳入优先审评审批程序20个（27%）、附条件批准程序17个（23%）、突破性治疗药物程序10个（14%）和特别审批程序5个（7%）。总体而言，38%的新药审批至少纳入了一种加速上市程序，详见表4。 5 审批效率 2023年，CDER批准新药的89%在《处方药用户费用法案》（Prescription Drug User Fee Act，PDUFA）设定的目标日期内或之前完成审批。尽管COVID-19疫情导致的国际旅行限制阻碍了用户费用审查时限内的现场检查，进而推迟了部分新药的审批进程，但CDER依然保持了较高的审批效率。 在首次审批周期内，CDER批准了84%的新药申请，这反映了FDA在新药审评过程中指导的清晰性和协调的高效性。此外，65%的新药审批使用了快速通道认定、突破性疗法认定、优先审查认定和加速批准4个加速程序中的一个或多个，有效地缩短了新疗法上市的时间，为患者提供了更快速的治疗选择。 值得注意的是，2023年64%的新药首次在美国获得批准，凸显了FDA在新药审批方面的领先地位。与2022年相比，2023年我国新药审评用时中位数减少了20天。然而，一次性通过率比2022年减少了50%，这可能是由于随着我国药品监管与国际接轨，对药品申报资料的要求更为严格和细致。为了适应这一趋势，药企必须提升其申报资料的研究质量和细节处理能力，以提高一次性通过新药审评的概率，从而加速新药的上市进程。此外，47%的新药首次在我国获得批准，该比例虽然低于2023年FDA获批新药中首次在美国获批的新药的比例，但它仍然显示了我国药品监管体系对新药的开放性和包容性。 6 获批新药的企业情况 2023年，辉瑞共有6个新药获批，其获批新药数量是位于其后的凯西制药和优时比的两倍。辉瑞作为全球领先的制药公司，其新产品涵盖了多个治疗领域，包括成人偏头痛急性治疗药物Zavzpret、COVID-19抗病毒药物Paxlovid、严重斑秃治疗药物Litfulo、生长迟缓治疗药物Ngenla、多发性骨髓瘤药物Elrexfio和活动性溃疡性结肠炎治疗药物Velsipity。专注于呼吸系统疾病和罕见病治疗的凯西制药和专注于免疫学和神经学领域的生物制药公司优时比并列第二，各有3个新药获得批准。全球制药行业的巨头礼来、葛兰素史克、诺华、阿斯利康和日本的跨国制药公司安斯泰来各有2个新药获批。其他企业包括罗氏、赛诺菲、强生、卫材、第一三共、Acadia Pharmaceuticals、Incyte、Cidara、Pharming Group、Biogen、博士伦、Genmab、Entasis Therapeutics、Blue Earth Diagnostics、Lexicon Pharmaceuticals、BioLineRx、Fabre-Kral、Amicus Therapeutics、Catalyst Pharmaceuticals、Coherus BioSciences等，各有1种新药获得批准。总体来看，FDA在2023年批准的新药涵盖了从大型制药巨头到中小型生物技术公司的广泛企业，这反映了整个制药行业的创新活力和多样性。 罗氏、诺华和我武生物2023年在我国均获批了3个新药，百时美施贵宝、阿斯利康、贝达药业、恒瑞医药和武田各获批了2个新药，葛兰素史克、拜耳、Argenx BV、Bioprojet Pharma、豪森药业、合源生物、正大天晴、京新药业、齐鲁制药、思济药业、先声药业等企业也分别有1个药物获批。总而言之，不仅有国际知名的大型制药企业例如罗氏、诺华、BMS、阿斯利康等在我国获批新药，还有众多本土企业例如恒瑞医药、我武生物、正大天晴等也取得了显著成绩。这表明我国药品市场正在吸引越来越多的国际和本土企业参与竞争。 7 讨论 2023年，FDA批准新药55个，我国批准新药74个，约是美国FDA批准新药的1.3倍。我国新药审批数量超FDA，中美两国获批新药的疾病覆盖领域均多样化。从治疗领域来看，肿瘤占据中美获批新药数量首位，在遗传/罕见病领域、神经系统疾病、皮肤病领域、传染病、代谢/内分泌疾病、胃肠疾病、肺病、精神疾病、免疫系统疾病、肾病、眼病和血液病等领域，两国分别有不同数量的新药获批。 与美国相比，我国在新药创新性和罕见病药物开发上存在显著差距。2023年FDA批准新药中的36%被认定为具有突破性创新的“first-in-class”药物，51%的新药获得了孤儿药认定。我国批准新药中仅有1个（1.4%）“first-in-class”药物，8个（11%）罕见病新药。我国新药的创新性及罕见病药物的开发与美国相比仍有较大差距。相信随着我国鼓励创新药与罕见病药物研发政策的不断出台、药品审评审批制度的不断完善以及药企研发能力的持续提升，该差距将会逐步缩小。 中美两国均广泛应用加速审批途径，提升新药审评效率。2023年FDA65%的新药审批至少使用了一种加速审评程序，例如快速通道、突破性疗法、优先审评和加速批准等，有效缩短了新疗法上市的时间，也为严重疾病患者提供了更快的治疗选择。为了尽快满足临床需求，加速药物可及性，我国亦采取突破性治疗药物程序、附条件批准程序、优先审评审批程序和特别审批程序等多种加速上市程序，38%的新药审批至少纳入了一种加速上市程序。2023年我国新药审评用时中位数比2022年减少了20天，但一次性通过率比2022年减少了50%，反映出随着我国药品监管与国际接轨，对药品申报资料的要求更为严格和细致。为了适应这一趋势，药企必须提升其申报资料的研究质量和细节处理能力。监管部门需进一步完善药品监管法规，确保与国际标准接轨，同时要加强药品监管人员的培训和教育，提升其专业水平和国际视野。 从企业情况来看，2023年辉瑞以6款新药成功获批，彰显了大型制药公司的创新力和竞争力。相较于美国市场以辉瑞为代表的制药巨头的强劲研发实力，我国药品市场则展现了本土企业与国际企业的竞争活力，不仅有国际巨头例如罗氏、诺华等获批新药，本土企业恒瑞医药、我武生物、正大天晴等也取得了显著成就。 随着我国药品监管体系的不断完善和药企研发能力的持续提升，期待未来能有更多高质量的新药通过审评，为患者带来更多的治疗选择。同时，整个行业需密切关注国际药品监管的动态，不断学习和借鉴先进的监管经验和技术，推动我国药品监管事业的持续发展。 参考文献 详见《中国药事 》2024年11月 第38卷第11期 识别微信二维码，可添加药时空小编 请注明：姓名+研究方向！