Tafamidis Meglumine

The pairing of two Bristol Myers Squibb immunotherapies is now permitted for the first-line treatment of two types of gastrointestinal cancers, after regulatory decisions handed out this past week that convert the accelerated approval status of the drug combination to full FDA approval in both indications. The drugs, Opdivo and Yervoy, belong to the class of medicines called checkpoint inhibitors. On April 8, the FDA approved use of this drug combination for adults and children age 12 and older whose colorectal cancer has metastasized or cannot be removed by surgery. The cancer must also be microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR), references to biomarkers that indicate the stability of DNA in a tumor. The regulatory submission was based on the results of an open-label Phase 3 test that compared the Opdivo/Yervoy combination to either Opdivo alone or chemotherapy. Results showed the immunotherapy combination led to a 38% reduction in the risk of disease progression or death compared to Opdivo monotherapy, and by 79% compared to chemo, both in the first-line setting. The study is ongoing to assess secondary measures, including overall survival. BMS said it will continue working with study investigators to present these data and longer-term follow-up results in the future. Sponsored Post Changes in Nurse Staffing Answer Clinician Demands The ongoing nursing shortage facilitates high turnover rates since nurses know they won’t have difficulties finding new jobs. In order to retain and attract staff, it’s in a facility’s best interest to understand what nurses want. By Tomasz Rezik On April 11, the FDA approved the combination of Opdivo and Yervoy for treating advanced cases of hepatocellular carcinoma. In the pivotal test in this indication, the drug combination led to median overall survival of 23.7 months. The comparator arm, in which patients received standard of care drugs sorafenib or levantinib, median overall survival was 20.6 months. At three years, the drug combination showed an overall survival rate of 38% versus 24% in the comparator arm. FDA approval of Opdivo plus Yervoy in hepatocellular carcinoma follows the European Commission’s early March approval of the drug combination in this indication. There’s plenty of news on the regulatory front. Here’s a recap of other recent highlights: New and Expanded Regulatory Approvals —Argenx drug Vyvgart Hytrulo received an additional approval for dosing of the product via a prefilled syringe. The drug, supplied in vials, previously received FDA approvals for the autoimmune conditions chronic inflammatory demyelinating polyneuropathy and generalized myasthenia gravis. Analysts say prefilled syringes bring patients a more convenient dosing option while giving Argenx the opportunity to get more market share by penetrating into earlier lines of therapy. Sponsored Post Understanding EGPA: The Role of Eosinophils and Advancements in Treatment Options FASENRA® (benralizumab) injection, for subcutaneous use, 30 mg is indicated for the treatment of adult patients with eosinophilic granulomatosis with polyangiitis (EGPA). FASENRA provides a treatment option for HCPs to consider when managing this challenging disease. By FASENRA, Sponsored Content —Amgen’s Uplizna expanded its label to include the treatment of immunoglobulin G4-related disease (IgG4-RD), an immune-mediated inflammatory disorder that can affect multiple organs. The regulatory decision makes the intravenously infused antibody the first FDA-approved treatment for this rare and chronic disease. Uplizna was first approved in 2020 as a treatment for neuromyelitis optica spectrum disorder. —Bayer’s Viktrakvi now has full FDA approval for the treatment of NTRK gene fusion-positive solid tumors in adults and children without a known acquired resistance mutation. The drug received accelerated approval in this indication in 2018. —AstraZeneca and Daiichi Sankyo welcomed European Union approval of Datroway as a treatment for metastatic breast cancer that is HR positive and HER2 negative. Datroway received FDA approval in the same indication in January. The drug is an antibody drug conjugate designed to target the cancer protein TROP2. —Novartis drug atrasentan, brand name Vanrafia, was awarded accelerated approval as a new treatment for the kidney disorder immunoglobulin A nephropathy (IgAN). The drug is an oral small molecule designed to block the endothelin A receptor. It’s one of two IgAN nephropathy drugs that came via the $3.2 billion acquisition of Chinook Therapeutics in 2023. Novartis’s internal IgAN research yielded the complement inhibitor Fabhalta, which received accelerated approval in this indication last summer. —Speaking of Fabhalta, that Novartis drug expanded its label to include C3 glomerulopathy, making the twice-daily pill the first approved treatment for this rare kidney disorder. Fabhalta is a small molecule designed to block the complement protein C3. It was first approved in 2023 as a treatment for the rare blood disorder paroxysmal nocturnal hemoglobinuria. —Sanofi received FDA approval for fitusiran, brand name Qfitlia, as a treatment for both hemophilia A and B. The drug leverages RNA interference to lower levels of AT, a protein that inhibits blood clotting. The approval covers the treatment of adults and children age 12 and older with or without inhibitors, which are antibodies that can develop in hemophilia patients, making it more difficult to control bleeding. By contrast, new Pfizer drug Hympavzi is approved only for hemophilia A and B patients without inhibitors while Sanofi’s Alhemo is approved only for hemophilia A and B patients with inhibitors. —Blockbuster AstraZeneca cancer immunotherapy Imfinzi landed European Union approval for the treatment of limited-stage small cell lung cancer. This new approval is based on Phase 3 results showing a 27% reduction in the risk of death compared to a placebo. The FDA approved Imfinzi for this indication last December. —Imfinzi also added perioperative treatment of muscle invasive bladder cancer to its list of FDA-approved indications. The newest FDA nod covers use of the drug in combination with chemotherapy before surgery to remove the bladder, followed by Imfinzi as an adjuvant monotherapy. In the pivotal study supporting this new indication, results showed a 32% reduction in the risk of cancer recurrence and a 25% reduction in the risk of death compared to neoadjuvant chemotherapy alone. —The European Commission approved Pfizer vaccine Abrysvo for protection against respiratory syncytial virus (RSV). The regulatory decision expands the vaccine’s approval to adults ages 18 to 59. European regulators initially approved the vaccine in 2023 for preventing lower respiratory tract disease in adults age 60 and older. Abrysvo won FDA approval the same year. —The FDA expanded approval of Novartis’s Pluvicto to include use as an earlier line of treatment — after anti-androgen drugs and before chemotherapy — for PSMA-positive metastatic castration-resistant prostate cancer (mCRPC). Pluvicto was initially approved in 2022 for PSMA-positive mCRPC that has already been treated with an anti-androgen drug and chemotherapy. Novartis said this expansion is important because about half of patients do not live long enough to receive a second-line treatment. —Exelixis cancer drug Cabometyx expanded its FDA-approved uses to advanced cases of pancreatic neuroendocrine tumors (pNET) and extra-pancreatic neuroendocrine tumors (epNET). The approval covers adults and children age 12 and older. Cabometyx is a small molecule designed to block enzymes that support cancer growth. The drug was first approved in 2016 for advanced renal cell carcinoma. —GSK landed FDA approval for Blujepa, an oral antibiotic developed to treat uncomplicated urinary tract infections. While drugs are already available for these infections, they can become resistant to treatment. Blujepa is designed to block two enzyme targets key to bacterial DNA replication, an approach intended to lower the potential for drug resistance. —The European Commission approved Capvaxive, a pneumococcal vaccine developed by Merck to protect against the 21 Streptococcus pneumoniae serotypes that lead to the majority of cases of invasive pneumococcal disease including eight that are not covered by any other available pneumococcal vaccine. The European regulatory decision covers adults age 18 and older. Capvaxive received FDA approval last July. —Alynlam Pharmaceuticals drug Amvuttra received FDA approval for treating cardiomyopathy caused by transthyretin amyloidosis (ATTR). The RNA interference drug was first approved in 2022 for the treatment of polyneuropathy caused by ATTR. In the cardiomyopathy indication, it will compete with Pfizer’s Vyndaqel and Vyndamax, which are established as the standard of care, as well as Attruby, a BridgeBio Pharma drug that received FDA approval last fall. —Bristol Myers Squibb’s Breyanzi received European Commission approval for relapsed or refractory follicular lymphoma. The CAR T-therapy received accelerated FDA approval in this indication last May. —Geron Corporation’s Rytelo received European Commission approval for the treatment of transfusion-dependent anemia caused by lower-risk myelodysplastic syndrome, a progressive type of blood cancer. The intravenously infused drug is the first telomerase inhibitor to reach the market. Rytelo landed FDA approval last June. —Neffy, an epinephrine nasal spray that ARS Pharmaceuticals developed to treat allergic reactions to insect bites, medications, and food, expanded its FDA approval to include patients weighing 15 to 30 kilograms (about 33 to 60 pounds). The product’s initial approval last year covered its use in patients weighing 30 kg or more. —Roche subsidiary Genentech received FDA approval for tenecteplase, brand name TNKase, as a treatment for acute ischemic stroke in adults. Genentech said TNKase is the first stroke medicine approved by the FDA in nearly 30 years. It’s the second approved indication for the clot-busting drug. The FDA approved it in 2000 for treating acute myocardial infarction. —A Neurotech Pharmaceuticals cell and gene therapy administered via a surgical implant received FDA approval for the treatment of macular telangectasia type 2. The therapy, brand name Encelto, is the first approved treatment for this rare vision-loss disorder. Regulatory Setbacks —The FDA missed the April 1 regulatory decision target date for Novavax’s Covid-19 vaccine. Politico reported the holdup was due to the intervention of Sara Brenner, the FDA’s principal deputy commissioner, who asked for more data. Novavax said it has responded to all of the FDA’s information requests and believes the application is ready for approval based on the submitted Phase 3 data. Novavax’s protein-based vaccine initially received FDA emergency use authorization in 2022, a status that it retained under amended authorizations in 2023 and last year, both times for new formulas that covered the prevalent variants at the time. Full FDA approval would put the vaccine on par with the approved messenger RNA vaccines marketed by Pfizer and Moderna. Novavax’s Covid-19 vaccine will be co-commercialized with Sanofi under a partnership announced last year. —Roche paused European clinical tests of the Duchenne muscular dystrophy gene therapy Elevidys, the pharmaceutical giant said in a letter to the World Duchenne Organization. The move follows Sarepta Therapeutics’ recent report of a liver failure-associated patient death. Roche said the European Medicines Agency asked for a clinical hold on four studies until the inquiry into the cause of death is complete. Under a 2019 deal with Sarepta, Roche holds rights to Elevidys outside of the U.S. —For the second time, the FDA has rejected Aldeyra Therapeutics drug reproxalap as a treatment for dry eye disease. The agency turned down an application for the drug in 2023. According to Aldeyra, the FDA’s latest complete response letter said the new drug application failed to demonstrate efficacy. The regulator asked the company to run at least one more clinical trial. Among the concerns raised by the FDA is differences in baseline scores, which may have affected interpretation of trial results. Aldeyra said results from two ongoing studies are expected later in the current quarter, potentially paving the way for a mid-year resubmission of the application. —The European Medicines Agency refused marketing authorization for Eli Lilly Alzheimer’s disease drug Kisunla. Safety was the main reason cited, particularly the brain inflammation and bleeding that is a known complication risk associated with all drugs in the same class of Alzheimer’s medicines. The agency said the drug’s benefits were not enough to outweigh the potentially fatal safety risks. The FDA approved Kisunla last July. —In other Alzheimer’s drug news, Eisai again failed to persuade Australia’s drugs regulator to register Leqembi. The pharma company had asked the Therapeutics Goods Administration (TGA) to reconsider its October 2024 decision to not register the drug in Australia. Eisai’s request for reconsideration covered a narrower indication, but Eisai said the TGA did not agree that safety has been established for these patients.

精彩内容罕见病“用药短缺”备受关注。近年来国家密集出台激励政策，加快新药审评审批速度，为企业提供罕见病药研发支持、提升其市场准入效率。据不完全统计，2024年至少有55个罕见病药获批上市，覆盖发作性睡病、转甲状腺素蛋白淀粉样变性等34个罕见病病种，涉及南京正大天晴、齐鲁等知名药企。日前，国家发文提及2025国谈时间或前移，华东、鲁南......49个独家罕见病药（含剂型独家，下同）蓄势待发，最终医保准入情况如何，我们拭目以待。激励政策密集出台，罕见病药市场冲刺1800亿“无药可用”是罕见病患者面临的最大困难之一。由于患者人数少、市场规模小、研发成本高，制药企业对该领域投入的积极性不高，经济回报难以实现。据统计，目前全球共发现7000多种罕见病，有药可治的比例不足10%。我国有2000多万罕见病患者，每年新增患者超20万。对此，国家近年来密集发布系列激励政策，不断加码罕见病赛道，加快新药审评审批速度，为企业提供罕见病药研发支持、提升其市场准入效率。比如：今年1月，国务院办公厅发布的《关于全面深化药品医疗器械监管改革促进医药产业高质量发展的意见》中明确，对符合条件的罕见病用创新药减免临床试验；将罕见病药注册检验批次由3批减为1批；探索由特定医疗机构先行进口未在境内注册上市的临床急需罕见病药；对符合条件的罕见病药给予一定的市场独占期等。来源：国务院官网不止于此，2024年9月，CDE发布了《以患者为中心的罕见疾病药物研发试点工作计划（“关爱计划”）》，旨在推动以患者为中心的罕见疾病药物研发和上市，截至目前已有10余款罕见病药被纳入“关爱计划”；2023年9月，国家卫健委等六部门联合制定了《第二批罕见病目录》，涉及86个罕见病种，标志着中国罕见病目录完成首次扩容；2023年1月，国家药监局印发《进一步加强中药科学监管促进中药传承创新发展若干措施的通知》，鼓励并扶持用于重大疾病、罕见病，或者儿童用中药新药的研制，对符合规定情形的相关注册申请实行优先审评审批......近年来国家发布的部分鼓励罕见病药研发和生产的相关政策来源：国家药监局、国家卫健委等，米内网整理伴随临床用药需求的推动，以及激励创新研发政策的出台，罕见病药物市场持续升温。弗若斯特沙利文指出，中国罕见病药市场于2016年及2020年分别占全球罕见病市场的0.4%及1%，预计2030年将激增至259亿美元，约合1886亿元（按实时汇率换算），年均复合增长率达34.5%。150余个罕见病药来袭，南京正大天晴、齐鲁、三生亮眼市场增速显著、前景广阔，让越来越多企业瞄准中国市场，加快罕见病领域的布局，近年来，我国罕见病用药上市数量和速度实现了“双提升”。据不完全统计，2018-2024年，国内已有150余个罕见病药（按罕见病适应症获批时间统计，下同）获批上市，其中2022年约有14个，2023年约有27个；2024年则至少有55个，覆盖发作性睡病、转甲状腺素蛋白淀粉样变性、血栓性血小板减少性紫癜等34种罕见病。2024年部分获批上市的罕见病药来源：米内网综合数据库、国家卫健委官网2024年获批上市的罕见病药中，涉及第一批和第二批《罕见病目录》的品种数占比约为1:1，集中在特发性肺动脉高压、肝豆状核变性等病种；按治疗大类统计，抗肿瘤和免疫调节剂为主力军，有11个产品在列；从企业层面上看，国产企业在获批产品数上力压外资企业，不乏齐鲁制药、华东医药、南京正大天晴制药、上海医药、三生制药等知名药企。地舒单抗注射液、注射用罗普司亭N01均为齐鲁制药2024年获批的生物药新品，分别用于治疗骨巨细胞瘤和血栓性血小板减少性紫癜。米内网数据显示，地舒单抗注射液近年来在中国三大终端六大市场（统计范围详见本文末）销售额逐年走高，2023年突破13亿元，同比增长54.3%。注射用罗普司亭是全球首个长效TPO受体激动剂，目前国内有2家企业拥有该药生产批文，其中齐鲁制药为国产首家获批。近年来中国三大终端六大市场地舒单抗注射液销售趋势（单位：万元）来源：米内网格局数据库氯苯唑酸葡胺软胶囊用于转甲状腺素蛋白淀粉样变性，齐鲁制药、南京正大天晴制药先后于2024年6月拿下首仿+首家过评、国产第2家。马昔腾坦片用于治疗特发性肺动脉高压，米内网数据显示，目前已有8家企业以新分类提交了该药上市申请，南京泽恒医药、上海医药、远大健康、齐鲁制药于2024年相继获批上市、视同过评。富马酸二甲酯肠溶胶囊用于治疗多发性硬化，2023年以来，已有四川医产集团、齐鲁制药和人福医药拿下该产品生产批文，另有厦门力卓药业提交的仿制4类上市申请在审。富马酸二甲酯肠溶胶囊以新分类报产的仿制药来源：米内网中国申报进度（MED）数据库重组人血小板生成素注射液为三生制药的独家1类创新药，用于治疗血栓性血小板减少性紫癜等。在中国三大终端六大市场，该产品近年来销售额稳步提升，2023年拿下超48亿元，同比增长19.21%；2024上半年收获超26亿元，高居止血药产品TOP1之位。2024年8月，重组人血小板生成素注射液拟用于接受择期侵入性手术的慢性肝病相关血小板减少的上市申请获受理。若该适应症成功获批，将进一步扩大其在肝病治疗领域的竞争力。2024H1中国三大终端六大市场止血药产品TOP10来源：米内网格局数据库注：增长率为负数用*表示注射用利纳西普是华东医药与Kiniksa合作开发的一款重组二聚体融合蛋白，2024年11月和12月，该药先后在国内获批用于治疗成人和12岁及以上青少年冷吡啉相关周期性综合征（CAPS）和复发性心包炎（RP）。目前，冷吡啉相关周期性综合征、复发性心包炎均已被纳入我国《第二批罕见病目录》。此外，据NMPA发布的《2024年度药品审评报告》，20个罕见病药通过优先审评审批程序得以加快上市。其中，伯瑞替尼肠溶胶囊为北京浦润奥生物的1类创新药，是全球首个脑胶质瘤MET靶向药，目前已纳入医保乙类药目录；亿帆医药的二氮嗪口服混悬液可用于先天性高胰岛素性低血糖血症（CHI），该新品的获批，将为CHI患儿的治疗带来了新希望......56款罕见病药剑指新国谈，华东、鲁南、万邦德备战为进一步提高患者的用药可及性，加快推进罕见病药准入医保已然成为国家的重要任务之一。今年1月，国家医保局召开“保障人民健康 赋能经济发展”新闻发布会，公布2025年有关医保工作安排。据国家医保局相关负责人介绍，2025年医保谈判时间或会适当提前，并引入“预申报”的过渡措施，预计从4月开始申报，争取9月结束医保谈判、发布新版医保目录；而预计能在今年5月底前获批的新药，相关企业也可预申报。来源：国家医保局官网据不完全统计，目前至少有56款罕见病药有望参与2025年国谈，其中进行性肌营养不良、黏多糖贮积症、肝豆状核变性、血友病等罕见病的治疗药较多；剂型上以注射剂、片剂为主，分别占34个和9个席位。从治疗大类上看，抗肿瘤和免疫调节剂、消化系统及代谢药、神经系统药物均以9个及以上产品在数量上领跑。米内网数据显示，上述品类2023年在中国三大终端六大市场合计销售规模分别超过2200亿元、2100亿元和1000亿元。部分有机会参与2025国谈的罕见病药来源：米内网中国申报进度（MED）数据库56个有望参与新一轮国谈的罕见病药中，独家品种有49个，占比超过八成，包括鲁南制药的盐酸沙丙蝶呤片（高苯丙氨酸血症）、华东医药的注射用利纳西普（冷吡啉相关周期性综合征、复发性心包炎）、兆科药业的苯丁酸钠颗粒（尿素循环障碍）、万邦德制药的石杉碱甲注射液（重症肌无力）、复星医药的注射用人干扰素γ（慢性肉芽肿）等，以及达妥昔单抗β注射液（神经母细胞瘤）、注射用维拉苷酶α（戈谢病）、聚乙二醇重组人生长激素注射液（特纳综合征）、布罗索尤单抗注射液（X连锁低磷血症）、注射用阿糖苷酶α（庞贝病）等2023-2024H1在中国三大终端六大市场销售额增速均达两位数及以上的潜力品种。根据国家医保局新闻发布会的消息，2025国谈时间前移或已势在必行。四月已至，距离新药申报截至时间大约有2个月（申报时间：2025年4月1日-2025年5月底），届时，哪些品种能踏上国谈申报的“末班车”，并最终成功入选，我们拭目以待！来源：米内网数据库、国家卫健委官网等注：米内网《中国三大终端六大市场药品竞争格局》，统计范围是：城市公立医院和县级公立医院、城市社区中心和乡镇卫生院、城市实体药店和网上药店，不含民营医院、私人诊所、村卫生室，不含县乡村药店；上述销售额以产品在终端的平均零售价计算。数据统计截至4月3日，如有疏漏，欢迎指正！免责声明：本文仅作医药信息传播分享，并不构成投资或决策建议。本文为原创稿件，转载文章或引用数据请注明来源和作者，否则将追究侵权责任。投稿及报料请发邮件到872470254@qq.com稿件要求详询米内微信首页菜单栏商务及内容合作可联系QQ：412539092【分享、点赞、在看】点一点不失联哦