Eplerenone

2024 年医保目录现场谈判竞价已经于昨日（10月27日） 上午 8:30 在北京正式开始，本次谈判共持续4天，将于本周三结束，由全国各地遴选的25名医保部门的谈判专家分成5个小组进行，共有 162 个药品参加现场谈判和竞价，最终结果拟于11月公布。 今天是医保谈判的第二天，这两天有哪些品种参与了谈判，情况又如何呢？ 国谈首日现场直击：抗病毒药打头阵，多家药企亮相！ 据界面新闻报道，国谈首日上午有信达生物、人福医药、正大天晴、金赛药业、罗氏制药、默沙东等多家企业的代表出席。涉及的谈判药品以抗病毒、降压药为主。 早上8点半左右，第一批企业代表入场开谈。接近11点，信达生物的企业代表结束谈判，但并未透露更多信息。 据公众号赛柏蓝现场报道，今日上午率先启动谈判的是抗病毒相关产品，从出场企业的情况观察，大部分谈判结果比较理想。 9点左右就已有企业代表结束谈判，有代表对界面新闻记者表示：“（谈判）很简单很简单”。更一企业代表一出门，没等记者开口，就主动问记者：“我是第一个出来的吗？”上午9点半左右，已经陆陆续续有四五家企业代表谈判结束，但表情管理都做得很好，面色凝重，一言不发。10点左右，有一家从深圳来的企业代表表示，“基本在预期之内，专家态度挺好。”另一家企业代表表示他们谈的是创新药，下午还来。 综合媒体报道来看，下午的谈判较上午胶着，各家药企离场间隔时间明显被拉长。 据《科创板日报》记者现场报道，下午场首家被点名的企业是信立泰，随后被点名的企业包括恒瑞医药、百奥泰、宜昌人福、成都倍特、武汉远大、信达生物、云顶新曜、石家庄四药、北京韩美、广州兆科、上海琅钰健康等。与此同时，也有多家外企进场，包括拜耳、赛诺菲、欧加隆、诺华等。 《科创板日报》表示，下午场不论是第一家出来的企业，还是紧随其后离开的企业，在整体耗时方面明显多于上午场的企业。而且下午离场的企业，似乎都达成一种默契，在面对询问时都选择闭口不谈。 根据《科创板日报》记者现场获取的资料，参加下午场谈判的部分药品包括——信立泰的阿利沙坦酯氨氯地平片、北京韩美的氨氯地平氯沙坦钾片（II）、成都倍特的呋塞米口服溶液、武汉远大的依普利酮片、信达生物的托莱西单抗注射液，以及石家庄四药的司替戊醇干混悬剂、诺华制药的奥法妥木单抗注射液和英克司兰钠注射液、广州兆科的利鲁唑口服混悬液、欧加隆的依折麦布阿托伐他汀钙片（II）。 其中，司替戊醇干混悬剂、奥法妥木单抗注射液、利鲁唑口服混悬液和依折麦布阿托伐他汀钙片（II）等均属于罕见病用药，对应分别用于Dravet综合征、成人复发型多发性硬化（RMS）、肌萎缩侧索硬化（ALS）、高胆固醇血症。 据罕见病信息网整理，在此前医保局公布的初审名单罕见病药物方面，至少有40个药品通过初审。 如替度格鲁肽（短肠综合征）、醋酸氟氢可的松片（先天性肾上腺皮质增生症和Addison病）、氯苯唑酸葡胺软胶囊（转甲状腺素蛋白淀粉样变性多发性神经病）、卡谷氨酸分散片&苯丁酸甘油酯（尿素循环障碍）、注射用培妥罗凝血素α（血友病）等。 其中，值得关注的是，12个罕见病有超过两款以上的适应症内药物，包括进行性肌营养不良、肺动脉高压、先天性肾上腺皮质增生症(CAH)、神经母细胞瘤、婴儿痉挛症、地中海贫血、转甲状腺素蛋白淀粉样变性多发性神经病（ATTR-PN)、视神经脊髓炎谱系疾病（NMOSD）、 阵发性睡眠性血红蛋白尿症（PNH）、原发免疫性血小板减少症(ITP)和肝豆状核变性。 此外，国谈首日下午场谈判药品中诺华制药的英克司兰钠注射液也值得特别关注，它是一款超长效降脂药物，目前市场价格为9988元/针，该注射液的给药方式是单次皮下注射，首次给药后，患者需要在3个月和每6个月时分别进行再次给药。因此，一般患者每年可能需要接受两到三针的治疗，每年的费用大约在2万到3万元人民币之间。 今年参加国谈的还有同样去年8月获批上市国内药企信达的创新药托莱西单抗，同样是一款长效降脂药，该药物的推荐剂量为150mg，每2周一次；450mg，每4周一次；或600mg，每6周一次。在不考虑慈善赠药的情况下，患者年用药费用大约为3.75万元至5.3万元。 而在此之前，两款进口PCSK9抑制剂类药，即安进的依洛尤单抗、赛诺菲的阿利西尤单抗已经进入医保目录。通过医保谈判，这两个产品的年治疗费用降低到7000元，患者自付只需要一两千元。而信达生物的托莱西单抗和诺华英克司兰钠注射液均高于此价格，通过谈判后，他们是否能够纳入医保目录，又能降价多少值得关注。 更值得关注的是，君实生物、康方生物在2024年３季度各有一款降脂单抗上市，行业普遍认为他们参与明年的国谈基本是板上钉钉。前有追兵，后有来者，信达和诺华的本次的两款产品在此次谈判中将面临较大幅度的降价压力。 国谈第二日现场直击（10月28日） 据财联社报道，国谈第二日上午以糖尿病药物和肿瘤药物为主，阿斯利康、康方生物、罗氏制药、信立泰、海思科、江苏恒瑞、石药集团、武田、先声药业、南京百鑫愉医药、勃林格殷格翰等多家企业的代表出席。 今日上午，糖尿病药物密集谈判。据财联社记者现场报道，上午，首先进场的企业代表和谈判品种分别是信立泰的苯甲酸福格列汀片、海思科的考格列汀片和江苏恒瑞三款药物——脯氨酸加格列净片、恒格列净二甲双胍缓释片（I）/恒格列净二甲双胍缓释片（II）、武汉久安药物研究院的盐酸二甲双胍缓释片（Ⅳ）。 石药集团（01093.HK）恩必普的丁苯酞氯化钠注射液、武田的马立巴韦片、先声药业（02096.HK）的依达拉奉右莰醇注射用浓溶液、南京百鑫愉医药的依达拉奉舌下片、勃林格殷格翰的注射用阿替普酶等也在上午谈判规划中。 上午完成谈判的第一家企业代表出现在9时10分左右，但谈判代表不苟言笑，一行三人排开围观的媒体记者，然后径直离开。 但完成谈判的第二家企业代表心情比较好，对在场记者感叹“心脏都要跳出来了！”不过，当问及医保局今年对企业态度等问题时，他赶紧表示：“都不让说，都不让说。” 10时30分左右，一位企业代表从会场出来，边走边对在场媒体表示“还行”，在问及是否是创新药时，对方则回应，“创新药，今年谈完了。” 此外，值得一提的是，公众号赛柏蓝捕捉到一个有趣的细节，9点半左右，有等候在外面的人通过大门给参与谈判的代表递上计算器，紧张的价格拉锯跃然纸上。 今日上午单抗、双抗为主的肿瘤药也开始了谈判。 阿斯利康上午的谈判品种包含两款，一是被称为“ADC神药”的DS-8201（注射用德曲妥珠单抗）和布地格福吸入气雾剂。 据相关资料显示，DS-8201是由第一三共和阿斯利康联合开发的ADC药物，在中国已获批用于治疗既往接受过一种或一种以上抗HER2药物治疗的不可切除或转移性HER2阳性成人乳腺癌患者。 DS-8201并非首次参加国谈，去年，阿斯利康中国区副总裁黄彬就携该品种参加了国谈，但遗憾的是，DS-8201并未被纳入医保。此外，今年6月，有行业媒体消息称，经过企业主动申报降价，自2024年6月20日起，DS-8201规格为100mg/瓶的定价将从8860元降至6912元，降幅为22%。当时业内普遍认为，DS-8201此举就是为了被纳入医保目录而来。 降价需求进入医保不是只有阿斯利康，今天上午康方生物（09926.HK）的依沃西单抗注射液、卡度尼利单抗注射液也在谈判进程中，今年6月康方生物卡度尼利单抗价格下调，单支价格从原先的13220元降至6166元，降幅53.4%。 据悉，康方生物和阿斯利康企业代表离场时间远超昨日上午企业代表，且均匆匆离开未透露谈判结果。康方生物此次由董事长夏瑜亲自带队谈判，接近下午一点才离场而去；阿斯利康DS-8201（注射用德曲妥珠单抗）上午开始谈判，直到下午一点半左右，阿斯利康中国区副总裁黄彬等才走出会场。 今日上午谈判的肿瘤药物还有罗氏制药的帕妥珠曲妥珠单抗注射液（皮下注射）和法瑞西单抗注射液、先声药业的西妥昔单抗β注射液、石药集团（01093.HK）的恩朗苏拜单抗注射液、上海科州药物的妥拉美替尼胶囊等。 此外，据财联社最新消息，下午2点医保局点名诺和诺德、杨森、亿帆医药、三生制药、豪森药业、默克、京新药业等企业和相关药品进场谈判，其中京新药业的地达西尼胶囊是一款专门用于治疗失眠的一类创新药。据相关资料显示，地达西尼(EVT201)是由我国京新医药研发的一款治疗成人失眠症的新药，地达西尼上市，打破了国内16年无失眠药物获批的尴尬局面。 来源：趣学术 投稿/内容沟通：华籍美人（Ww_150525） 近期精华文章

October 1, 2024 – Santhera Pharmaceuticals (SIX: SANN) announces the positive outcome of the LIONHEART study, confirming vamorolone’s distinctive action also as a mineralocorticoid receptor antagonist, setting it apart from other corticosteroids. The LIONHEART study, an open-label randomized, placebo- and eplerenone-controlled study involving 30 healthy adult male subjects, met its primary endpoint. It demonstrated a statistically significant increase in the urinary sodium/potassium ratio in the vamorolone arm compared to placebo (p<0.0001) following a fludrocortisone challenge. This increased ratio in urine provides clinical evidence for vamorolone’s unique mode of action also as a mineralocorticoid receptor antagonist (MRA) in humans. Combined with its known properties as a dissociative glucocorticoid receptor agonist, these findings further differentiate vamorolone’s pharmacological profile, distinguishing it from other corticosteroids. Cardiac complications such as cardiomyopathy are a leading cause of morbidity and mortality in boys with Duchenne muscular dystrophy (DMD). While treatment with corticosteroids (and ACE inhibitors) has demonstrated a delay in the onset of cardiomyopathy, the addition of MRAs including eplerenone to standard of care has also shown an improvement in left ventricular systolic dysfunction, the benefit of which increases with earlier initiation [1-3]. “Mineralocorticoid receptor antagonists are strongly recommended but late when cardiac function is already reduced and tend to be used in the presence of myocardial fibrosis as detected in magnetic resonance imaging,” explained Prof Karim Wahbi, PhD, MD, Cardiologist at the APHP Hospital Cochin, Paris, France. “What is intriguing about this mechanistic study is whether there is a synergistic benefit of the anti-inflammatory and MRA effects of vamorolone on the evolution of cardiac disease in children who started treatment early or if vamorolone could be of benefit to those who are already experiencing cardiac symptoms and wish to remain on a corticosteroid” “The LIONHEART study is an important milestone to establish the proof of concept for a cardioprotective potential of vamorolone,” stated Shabir Hasham, MD, Chief Medical Officer of Santhera. “We continue to collect data from patients who have been on vamorolone for up to seven years allowing us to better characterize long-term outcomes including any beneficial impact on cardiac complications in DMD.” AGAMREE is a novel drug with a mode of action based on binding to the same receptor as glucocorticoids but modifying its downstream activity. Moreover, it is not a substrate for the 11-β-hydroxysteroid dehydrogenase (11β-HSD) enzymes that may be responsible for local drug amplification and corticosteroid-associated toxicity in local tissues [4-7]. This mechanism has shown the potential to ‘dissociate’ efficacy from steroid safety concerns and therefore AGAMREE is positioned as a dissociative anti-inflammatory drug and an alternative to existing corticosteroids, the current standard of care in children and adolescent patients with DMD [4-7]. In the pivotal VISION-DMD study, AGAMREE met the primary endpoint Time to Stand (TTSTAND) velocity versus placebo (p=0.002) at 24 weeks of treatment and showed a good safety and tolerability profile [4]. The most commonly reported side effects were cushingoid features, vomiting, weight increase and irritability. Side effects were generally of mild to moderate severity. Currently available data show that AGAMREE, unlike corticosteroids, has been shown to be a dual mineralocorticoid antagonist and a dissociative glucocorticoid agonist based on animal experiments [7]. It has no restriction of growth [8] and no negative effects on bone metabolism as demonstrated by normal bone formation and bone resorption serum markers [9]. AGAMREE (vamorolone), an orphan medicinal product, is approved for use in the United States (Prescribing Information), the European Union (Summary of Product Characteristics) and the United Kingdom for the treatment of DMD. The LIONHEART study (SNT-I-VAM-026) is an open label randomized, 3-arm, parallel-group, placebo and eplerenone controlled study to evaluate the mineralocorticoid receptor antagonism (MRA) effect of vamorolone in 30 healthy adult male subjects after challenge with fludrocortisone, a known mineralocorticoid receptor agonist. The primary endpoint was defined as the ratio of sodium to potassium (Na/K) and the corresponding logarithm of the ratio (log10(10*Na/K) in urine at different time-points. Further analysis of the data is ongoing and will be presented at medical conferences. References: [1] Birnkrant et al. (2018) Lancet Neurol. 2018 Apr;17(4):347-361. doi: 10.1016/S1474-4422(18)30025-5. [2] Raman et a. (2015) Lancet Neurol 14: 153–161 doi:10.1016/s1474-4422(14)70318-7 [3] Raman et al. (2017) Orphanet J Rare Dis 12: 39. doi:10.1186/s13023-017-0590-8 [4] Dang UJ et al. (2024) Neurology 2024;102:e208112. doi.org/10.1212/WNL.0000000000208112. Link. [5] Guglieri M et al (2022). JAMA Neurol. 2022;79(10):1005-1014. doi:10.1001/jamaneurol.2022.2480. Link. [6] Liu X et al (2020). Proc Natl Acad Sci USA 117:24285-24293 [7] Heier CR et al (2019). Life Science Alliance DOI: 10.26508 [8] Ward et al., WMS 2022, FP.27 - Poster 71. Link. [9] Hasham et al., MDA 2022 Poster presentation. Link. Duchenne muscular dystrophy (DMD) is a rare inherited X-chromosome-linked disease, which almost exclusively affects males. DMD is characterized by inflammation which is present at birth or shortly thereafter. Inflammation leads to fibrosis of muscle and is clinically manifested by progressive muscle degeneration and weakness. Major milestones in the disease are the loss of ambulation, the loss of self-feeding, the start of assisted ventilation, and the development of cardiomyopathy. DMD reduces life expectancy to before the fourth decade due to respiratory and/or cardiac failure. Corticosteroids are the current standard of care for the treatment of DMD. Santhera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical company focused on the development and commercialization of innovative medicines for rare neuromuscular diseases with high unmet medical need. The Company has an exclusive license from ReveraGen for all indications worldwide to AGAMREE® (vamorolone), a dissociative steroid with novel mode of action, which was investigated in a pivotal study in patients with Duchenne muscular dystrophy (DMD) as an alternative to standard corticosteroids. AGAMREE for the treatment of DMD is approved in the U.S. by the Food and Drug Administration (FDA), in the EU by the European Medicines Agency (EMA), and in the UK by the Medicines and Healthcare products Regulatory Agency (MHRA). Santhera has out-licensed rights to AGAMREE for North America to Catalyst Pharmaceuticals and for China to Sperogenix Therapeutics. For further information, please visit www.santhera.com. The content above comes from the network. if any infringement, please contact us to modify.