Pulmonary Embolism

来源：企业公告   编辑：wangxinglai2004近日，3药企分别发布了产品海外上市进展公告，具体为亿帆生物艾贝格司亭α注射液获欧盟批准上市、海普瑞依诺肝素钠注射液获泰国药品注册批件、兄弟医药API获得印度注册证书。亿帆生物艾贝格司亭α注射液获欧盟批准上市一、药物基本情况  药品通用名：艾贝格司亭α注射液 中文商品名：亿立舒® 英文商品名：Ryzneuta® 申请事项：人用药品上市许可申请 注册编号：EU/1/24/1793上市许可有效期：5年，自批准之日起。 二、药物其他相情况 公司于2021年9月30日收到欧洲药品管理局（EMA）签发的受理函，于2024年1月通过欧洲集中程序获欧洲药品管理局（EMA）人用药品委员会（CHMP）积极审查意见，推荐批准Ryzneuta®上市，并于2024年3月22日获EC正式批准上市。本次Ryzneuta®上市许可申请获EC批准，是继其在中国、美国获批上市后又 一全球权威药监机构获得的批准，再次验证了公司具有独立完成创新生物药全 球研发、临床、注册和生产的综合能力，也充分证明了公司执行贯彻“创新、 国际化”战略的能力。Ryzneuta®是目前全球G-CSF治疗药品中唯一一款既与长效原研产品，也与短效原研产品进行头对头临床研究对比，达到预设目标，并实现中国、美国、欧洲获批上市的药品，其作为非PEG修饰的长效G-CSF，主要优势为工艺创新、 疗效更优，安全性好，更早给药节省费用等。为加快产品在欧洲的商业化，截至本报告披露日，公司已分别与KALTEQ  S.A., 、Apogepha Arzneimittel GmbH, 签订《独家许可协议》，将希腊、塞浦路斯的独家经销权许可给KALTEQ  S.A.,，将德国、瑞士、奥地利的独家经销权许可给Apogepha Arzneimittel GmbH,。海普瑞依诺肝素钠注射液获泰国药品注册批件 一、药物基本情况 （一）产品名称：依诺肝素钠注射液 （二）剂型：注射液 （三）规格：0.2ml:20mg, 0.4ml:40mg, 0.6ml:60mg, 0.8ml:80mg（四）适应症：1. 0.2ml:20mg 和 0.4ml:40mg 注射液：预防静脉血栓栓塞性疾病（预防静脉血栓形成），特别是与骨科或普通外科相 关的血栓形成。2. 0.6ml:60mg 和 0.8ml:80mg 注射液：治疗深静脉血栓，伴有或不伴有肺栓塞，临床症状不严重，但肺栓塞需 要手术或溶栓治疗除外。与阿司匹林联合治疗不稳定型心绞痛和非Q波型心 肌梗死，预防血液透析时体外循环血栓形成。联合溶栓剂或经皮冠状动脉介入 治疗（PCI）治疗急性ST段抬高型心肌梗死。（五）许可有效期：七年二、药物其他相情况 本次获批，代表着海普瑞的依诺肝素钠制剂可以在泰国市场进行销售，将进一 步提升公司依诺肝素钠制剂在全球的市场占有率。截至本公告日，公司依诺肝素钠制剂在全球超过40个国家及地区获批上市，其中重要获批市场包括中国、美国、欧盟、英国、瑞士、波兰、巴西、哥伦比亚、智利、加拿大、沙特、阿联酋、马来西亚、澳大利亚等。兄弟医药API获得印度注册证书一、药物基本情况  1、品种名称：碘帕醇 2、产品剂型：原料药 3、注册证书编号：RC/BD-0026564、有效期 ：2024年3月21日至2026年2月9日 5、生产企业：江西兄弟医药有限公司 6、生产地址：江西省九江市彭泽县矶山工业园二、药物其他相情况 本次公司碘帕醇原料药产品获得印度注册证书，标志着碘帕醇原料药产品可以正式在印度市场进行销售，为公司碘造影剂原料药项目的产能释放及进一步拓展国际市场带来积极影响。

近日，国家药监局公布的信息显示，江西施美药业股份有限公司的利伐沙班颗粒获批上市。截至目前，国内仅施美药业获得利伐沙班颗粒剂的药品注册证书（国药准字H20243387），原研拜耳尚未进口，施美药业按照化药3类申报生产，为首家获批并视同通过一致性评价的企业，填补了国内空白。01350亿抗凝血药市场迎来改良型利伐沙班颗粒新剂型伴随着中国社会老龄化和城市化进程的加快，居民患心脑血管疾病的危险因素逐渐增多，从而引发患病的比例和人数快速增长。据《中国心血管健康与疾病报告2022》，心血管病患者近3.3亿，在居民疾病死因构成中占比高达 44%，高于肿瘤及其他疾病，居于首位。全国每年因其死亡的人数超过 100 万，存活的患者中有 75%以上留有不同程度的残疾。而在心血管疾病防治方面，中国虽然目前已取得初步成效，但仍面临严峻挑战，心血管疾病患病率及死亡率仍处于上升阶段 。血栓性疾病作为心脑血管病的主要形式之一，具有高发病率、高复发率、高致残率、高死亡率的特征。除了血栓性疾病，还有许多其他疾病与血栓相关，例如心肌梗死、肺栓塞、心房颤动、脑梗死等，尤其是以栓塞和梗塞为主要诱因的心梗和脑梗是居民主要的死亡原因。目前临床上常用的抗血栓药物共分为三类：抗血小板药物、抗凝血药物以及溶栓药物。在中国市场，抗血栓药市场规模保持稳步快速增长的趋势。沙利文数据显示，未来五年，抗凝血药的销量增长仍将成为驱动行业市场规模扩张的首要原因。另据华经市场研究中心数据显示，近年来，国内抗凝血药物市场规模增长迅速，5年复合增长率达到16.19%。2018年样本医院抗凝血药物销售额为292亿元，占抗血栓药物销售额的41.35%。此外，抗血小板药及溶栓药市场规模也将获得快速成长。在此背景下，整体抗血栓药行业市场规模将以 13.1%的年复合增长率持续增长，到2023年，院内抗凝药市场规模有望上升至 350 亿元人民币。可以预见，今后全球市场对抗血栓药物的需求将会不断释放，整个抗血栓药行业有望迎来扩张，而这种扩张主要依靠抗凝血药市场的迅速增长。02利伐沙班颗粒剂尤其适合吞咽困难的老人、儿童和青少年患者利伐沙班是一种选择性凝血因子Xa抑制剂，通过竞争性抑制游离和结合的Xa因子，中断凝血级联反应的内源性和外源性途径，抑制凝血酶的产生，发挥抗血栓作用。该原研药由拜耳和强生子公司西安杨森联合开发，2008年9月获EMA批准，2011年11月获FDA批准，2009年3月获NMPA批准用于成人择期全髋或全膝关节置换术后静脉血栓的预防，商品名拜瑞妥，随后又于2015年5月被NMPA批准用于治疗和预防深静脉血栓构成并预防肺栓塞以及房颤患者的卒中预防。利伐沙班片为2022年版国家医保乙类药品，同时已列入2018年版国家基药目录。根据新思界产业研究中心发布的《2022-2027年利伐沙班行业市场深度调研及投资前景预测分析报告》显示，利伐沙班是全球最畅销药物之一，2020年，利伐沙班全球销售额达到78亿美元以上；国内销售额也稳步增长，2020院内市场达到30亿元以上，2021年院内市场更是突破了38亿元。利伐沙班也是拜耳销售额最高的药品，根据拜耳公布数据显示，2020年，拜耳利伐沙班销售额达到45.15亿美元。利伐沙班无疑已经成为是全球最畅销的药物之一。此次施美药业作为国内首家获批上市的利伐沙班颗粒，为单剂量独立复合膜袋包装，装量准确，服用方便，依从性好。适应症为择期髋关节或膝关节置换术成年患者，以预防静脉血栓的形成（VTE）；同时用于治疗和预防深静脉血栓形成（DVT）肺栓塞（PE）以及非瓣膜性房颤患者，以降低卒中和体循环栓塞的风险。尤为值得一提的是，除了用于成人患者外，还专门批准了儿科和青少年人群的新适应症--用于18岁以下且体重为30kg~50kg及50kg以上的儿童和青少年静脉血栓栓塞症（VTE）患者经过初始非口服抗凝治疗至少5天后的静脉血栓栓塞症（VTE）治疗及预防VTE复发。截至目前，该药是中国唯一拥有成人、儿童和青少年患者三大人群静脉血栓栓塞症治疗及预防复发适应症的抗凝药物。颗粒剂剂型尤其适合吞咽困难的老人、儿童和青少年患者。此次施美药业利伐沙班改良型颗粒剂剂型的获批上市，将为利伐沙班在抗凝血药市场的持续发力保驾护航。03为数百万儿童及青少年静脉血栓栓塞症患者带来新的希望据上海交通大学附属儿童医院重症医学科专家张育才在《中国小儿急救医学》撰文介绍：ICU患者存在DVT（下肢静脉血栓）和PE（肺栓塞）高风险，成人ICU患者DVT的发生率可高达10%，PE的发生率可达2%～4%。脓毒症和脓毒性休克有可能增加这种并发症的风险。儿童从生理角度来看处于相对低凝状态，VTE（静脉血栓栓塞症）发生率较成人低。国外报道，儿童总体VTE总体发生率约0.07/10 000～0.14/10000，但在住院儿童患者中，VTE发生率可增加100～1000倍，高达58/10 000人。其中住院创伤儿童和(或)ICU患儿中DVT（下肢静脉血栓）发生率可达0.2%～6.2%，留置中心静脉导管、心脏疾病及感染患儿中DVT（下肢静脉血栓）发生率更高，Manlhiot等报告先天性心脏病和(或)心脏手术的患者，DVT发生率可达11%。此外，由于儿童VTE（静脉血栓栓塞症）大多早期临床表现不典型，容易造成漏诊和误诊，因此实际发生率应该更高。全美最佳10大儿童医院--约翰·霍普金斯儿童医院（JOHNS HOPKINS）官网刊登出的文章明确指出：总的来说，每10000个孩子中就有1个会出现这些问题。然而，它们在住院儿童中更为常见，大约每200名住院儿童中就有1人出现血栓。根据我国第七次人口普查公报及2021年我国卫生健康事业发展统计公报，我国18岁以下青少年及儿童近4亿，每年18岁以下青少年及儿童住院人次在0.8-1亿左右，由此可推算，儿童及青少年静脉血栓栓塞症的患者人群数量在百万左右。此次施美药业利伐沙班改良型颗粒剂的首家获批上市，将为数百万儿童及青少年静脉血栓栓塞症患者带来新的希望。关于施美药业改良型创新药和首仿药江西施美药业股份有限公司近年来在改良型创新药和填补国内空白市场的首仿药方面持续发力。根据CDE网站显示：2023年以来施美药业已经申报2类改良型创新药7个，其中2.3类改良型创新药5个，目前已经获得药物临床试验批准通知书4个。在首仿药方面，除本次获批上市的首仿药利伐沙班颗粒外，此前苯磺酸左氨氯地平片（2.5mg）首家通过一致性评价、培哚普利叔丁胺片（8mg）已经作为首仿获批上市，目前在CDE技术审评的厄贝沙坦氨氯地平片、替米沙坦氨氯地平片、依折麦布阿托伐他汀钙片、坎地沙坦酯氨氯地平片、塞来昔布片等品种均为首家申报，可望陆续拿下首仿。

Posters to be presented highlighting research investigating pegtibatinase as the first potential disease-modifying treatment for classical homocystinuriaSAN DIEGO, April 04, 2024 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc., (Nasdaq: TVTX) today announced that the Company will present eight posters in classical homocystinuria (HCU) at Society for Inherited Metabolic Disorders (SIMD) in Charlotte, North Carolina from April 14-17, 2024, and Genetic Metabolic Dieticians International (GMDI) in Charlotte, North Carolina from April 17-20, 2024. At SIMD and GMDI, the Company will present the trial design of the pivotal Phase 3 HARMONY Study of pegtibatinase, a novel investigational enzyme replacement therapy for the treatment of classical HCU. Additionally, the Company will share insights on the development of an innovative tool used for dietary management and monitoring in the Phase 3 HARMONY Study and open-label extension ENSEMBLE Study, and the positive results from cohort 6 in the placebo-controlled Phase 1/2 COMPOSE Study of pegtibatinase in classical HCU. “Classical HCU is an isolating and devastating rare disorder with very limited treatment options, including adherence to highly restrictive diets, leaving patients and their caregivers with immense challenges,” said Jula Inrig, M.D., chief medical officer of Travere Therapeutics. “We look forward to participating at both of these prestigious metabolic medical meetings, and we are excited about our work pioneering the first potential disease-modifying treatment for the thousands of people affected by classical HCU.” SIMD Poster Presentations Latest Results From the COMPOSE Phase 1/2 Trial For the Treatment of Classical Homocystinuria (HCU) Using Pegtibatinase, a Novel Investigational Enzyme Replacement TherapyPoster: 139Abstract Category: Innovative TherapiesSymphony Ballrooms 4-7; April 15, 2024, 8-9 p.m. ET Pegtibatinase, an Investigational Enzyme Replacement Therapy, For The Treatment of Classical Homocystinuria (HCU): Design of the HARMONY Phase 3 Study Poster: 147Abstract Category: Innovative Therapies Symphony Ballrooms 4-7; April 15, 2024, 8-9 p.m. ET Economic Burden of Classical Homocystinuria in the United StatesPoster: 48Abstract Category: Clinical Care/ResearchSymphony Ballrooms 4-7; April 15, 2024, 7-8 p.m. ET Clinical Burden of Classical Homocystinuria in the United States: A Retrospective Claims Analysis Poster: 64Abstract Category: Clinical Care/ResearchSymphony Ballrooms 4-7; April 15, 2024, 7-8 p.m. ET Association Between Homocysteine and Clinical Outcomes in Patients with Classical Homocystinuria: A Systematic Literature ReviewPoster: 68Abstract Category: Clinical Care/ResearchSymphony Ballrooms 4-7; April 15, 2024, 7-8 p.m. ET GMDI Poster Presentations Latest Results from the COMPOSE Phase 1/2 Trial of Pegtibatinase, a Novel Investigational Enzyme Replacement Therapy for Classical Homocystinuria (HCU) Poster: 10April 18, 2024, 4:30-5:15 p.m. ET Novel Tool for Dietary Management and Monitoring in Clinical Trials of Pegtibatinase, an Investigational Enzyme Replacement Therapy for Classical Homocystinuria Poster: 16April 18, 2024, 4:30-5:15 p.m. ET Novel Dietary Management Strategies for Classical Homocystinuria (HCU) in HARMONY/ENSEMBLE Phase 3 Studies of Pegtibatinase, an Investigational Enzyme Replacement Therapy Poster: 33April 18, 2024, 5:15-6 p.m. ET About Classical Homocystinuria Classical homocystinuria (HCU) is a rare genetic metabolic disorder caused by a deficiency in the enzyme cystathionine beta synthase (CBS). CBS is a pivotal enzyme that is essential for the management of methionine and cysteine in the body. Classical HCU leads to toxic levels of homocysteine that can result in life-threatening thrombotic events such as stroke, pulmonary embolism and deep vein thrombosis, ophthalmologic and skeletal complications, as well as developmental delay. Current treatment options are limited to protein-restricted diet and use of vitamin B6 and betaine. About Pegtibatinase Pegtibatinase is an investigational PEGylated, recombinant enzyme replacement therapy designed to address the underlying cause of classical homocystinuria (HCU). In preclinical studies, pegtibatinase has demonstrated an ability to reduce total homocysteine levels and improve clinical parameters. Pegtibatinase is currently advancing in the ongoing Phase 1/2 COMPOSE Study to assess its safety, tolerability, pharmacokinetics, pharmacodynamics and clinical effects in patients with classical HCU. In May 2023, the Company announced that data from four patients treated with the highest dose of pegtibatinase showed a clinically meaningful 67.1% mean relative reduction in total homocysteine from baseline and was generally well-tolerated after 12 weeks of treatment. To date, the pegtibatinase program has been granted Breakthrough Therapy designation, Rare Pediatric Disease and Fast Track designations by the FDA, as well as Orphan Drug designation in the U.S. and Europe. About Travere Therapeutics At Travere Therapeutics, we are in rare for life. We are a biopharmaceutical company that comes together every day to help patients, families and caregivers of all backgrounds as they navigate life with a rare disease. On this path, we know the need for treatment options is urgent – that is why our global team works with the rare disease community to identify, develop and deliver life-changing therapies. In pursuit of this mission, we continuously seek to understand the diverse perspectives of rare patients and to courageously forge new paths to make a difference in their lives and provide hope – today and tomorrow. For more information, visit travere.com Forward Looking Statements This press release contains “forward-looking statements” as that term is defined in the Private Securities Litigation Reform Act of 1995. Without limiting the foregoing, these statements are often identified by the words “anticipate,” “believe,” “expect,” “intend,” “may,” “might,” “objective,” “plan,” “will” or similar expressions. In addition, expressions of our strategies, intentions or plans are also forward-looking statements. Such forward-looking statements include, but are not limited to, statements regarding pegtibatinase as the first potential disease-modifying treatment for classical homocystinuria, references to the HARMONY study and the other studies described herein, including statements regarding expectations related thereto, and statements regarding the development of an innovative tool used for dietary management and monitoring. Such forward-looking statements are based on current expectations and involve inherent risks and uncertainties, including factors that could delay, divert or change any of them, and could cause actual outcomes and results to differ materially from current expectations. No forward-looking statement can be guaranteed. Among the factors that could cause actual results to differ materially from those indicated in the forward-looking statements are risks related to the timing and outcome of the HARMONY study and the other studies described herein; risks and uncertainties associated with the regulatory review and approval process, risks associated with enrollment of clinical trials for rare diseases, and risks that ongoing or planned clinical trials may not succeed or may be delayed for safety, regulatory or other reasons. The Company faces risks related to its business and finances in general; risks related to the commercial launch of a new product; risks associated with market acceptance of FILSPARI and other current and future products, including efficacy, safety, price, reimbursement and benefit over competing therapies; and the risk that the Company’s clinical candidates will not be found to be safe or effective and that current or anticipated future clinical trials will not proceed as planned. Specifically, the Company faces risks related to the timing and potential outcome of the FDA’s potential acceptance for filing and review of the sNDA submission for full approval of FILSPARI in IgAN, and the timing and potential outcome of the European Commission’s decision regarding conditional marketing authorization of sparsentan for IgAN. There is no guarantee that the FDA will accept the sNDA submission for filing, that the European Commission will grant conditional marketing authorization of sparsentan for IgAN, or that regulators will grant full approval of sparsentan for IgAN or FSGS. The Company also faces the risk that it will be unable to raise additional funding that may be required to complete development of any or all of its product candidates, including as a result of macroeconomic conditions; risks relating to the Company's dependence on contractors for clinical drug supply and commercial manufacturing; uncertainties relating to patent protection and exclusivity periods and intellectual property rights of third parties; risks associated with regulatory interactions; and risks and uncertainties relating to competitive products, including current and potential future generic competition with certain of the Company's products, and technological changes that may limit demand for the Company's products. The Company also faces additional risks associated with global and macroeconomic conditions, including health epidemics and pandemics, including risks related to potential disruptions to clinical trials, commercialization activity, supply chain, and manufacturing operations. You are cautioned not to place undue reliance on these forward-looking statements as there are important factors that could cause actual results to differ materially from those in forward-looking statements, many of which are beyond our control. The Company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events, or otherwise. Investors are referred to the full discussion of risks and uncertainties, including under the heading “Risk Factors”, as included in the Company’s most recent Form 10-K, Form 10-Q and other filings with the Securities and Exchange Commission. Contact Info Media:888-969-7879 mediarelations@travere.comInvestors:888-969-7879 IR@travere.com