Odevixibat

Total sales growth of 11.6% at CER1, or 11.7% as reported, driven by all three therapeutic areas and including an increasing contribution from Iqirvo and Bylvay. Tovorafenib regulatory submission to EMA2 for pediatric low-grade glioma. Confirmation of full-year 2025 financial guidance. PARIS, FRANCE, 16 April 2025 – Ipsen (Euronext: IPN; ADR: IPSEY), a global specialty-care biopharmaceutical company, today presents sales for the first quarter of 2025. “Ipsen has delivered a strong start to 2025, building further momentum in the transformation of our company,” commented David Loew, Chief Executive Officer, Ipsen. “We continued to execute on our strategy with strong top-line growth and pipeline progression. I am pleased to see the rapid build-up of our Rare Cholestatic Liver disease franchise with two innovate medicines for five indications. 2025 is set to be an important year for Ipsen, with multiple launches underway and several milestones expected across our portfolio.” Full-year guidance Ipsen is confirming financial guidance for full-year 2025: Total sales growth greater than 5.0%, at constant currency. Based on the average level of exchange rates in March 2025, a limited effect on total sales from currencies is expected. Core operating margin greater than 30.0% of total sales, which includes additional R&D expenses from anticipated early and mid-stage external-innovation opportunities. Guidance includes expected negative impact on Somatuline sales due to increased generic competition in the U.S. and Europe. It excludes any impact from potential late-stage (Phase III clinical development or later) business development transactions. Upcoming Milestones Ipsen anticipates several key milestones across its portfolio in 2025, including: Cabometyx (CABINET trial) – Regulatory decision in the European Union for advanced pancreatic (pNETs) and extra-pancreatic (epNETs) neuroendocrine tumors (NETs). fidrisertib (FALKON trial) – Readout of the pivotal Phase IIb trial in fibrodysplasia ossificans progressiva (FOP). LANT3 (LANTIC trial) – Proof-of-concept data readout, evaluating its potential in aesthetics. Q1 pipeline progress The regulatory filing for tovorafenib was accepted by EMA for review in the European Union, marking an important step forward in the development of this potential treatment for pediatric low-grade glioma and reinforcing Ipsen’s commitment to innovation in rare and difficult-to-treat cancers. Ipsen also initiated the entry in Phase I of IPN01195, a RAF inhibitor, complementing IPN01194, an ERK inhibitor, and tovorafenib, two other assets targeting the MAPK pathway. Group refinancing Ipsen announced on March 19th the successful completion of its inaugural Rated Public Bond of €500 million with a coupon of 3.875%, maturing in March 2032. Following the disclosure of the Investment Grade ratings from S&P and Moody’s, this transaction was very well received and largely oversubscribed by a diversified and solid institutional investor base. This transaction is an important component of Ipsen’s refinancing plan which included the successful renewal of €1,5 billion syndicated Revolving Credit Facility, extending Ipsen’s debt maturity profile. Conference call A conference call and webcast for investors and analysts will begin today at 2pm CET. Participants can access the call and its details by registering here; webcast details can be found here. Calendar Ipsen intends to publish its half-year results on July 31st, 2025. Notes All financial figures are in € millions (€m). The performance shown covers the three-month period to 31 March 2025 (Q1 2025, the quarter), compared to the three-month period to 31 March 2024 (Q1 2024), unless stated otherwise. About Ipsen We are a global biopharmaceutical company with a focus on bringing transformative medicines to patients in three therapeutic areas: Oncology, Rare Disease and Neuroscience. Our pipeline is fueled by internal and external innovation and supported by nearly 100 years of development experience and global hubs in the U.S., France and the U.K. Our teams in more than 40 countries and our partnerships around the world enable us to bring medicines to patients in more than 100 countries. Ipsen is listed in Paris (Euronext: IPN) and in the U.S. through a Sponsored Level I American Depositary Receipt program (ADR: IPSEY). For more information, visit ipsen.com. Ipsen contacts InvestorsKhalid Deojee +33 6 69 09 12 96 MediaSally Bain +1 857 320 0517Anne Liontas +33 7 67 34 72 96 Disclaimers and/or forward-looking statements The forward-looking statements, objectives and targets contained herein are based on Ipsen’s management strategy, current views and assumptions. Such statements involve known and unknown risks and uncertainties that may cause actual results, performance or events to differ materially from those anticipated herein. All of the above risks could affect Ipsen’s future ability to achieve its financial targets, which were set assuming reasonable macroeconomic conditions based on the information available today. Use of the words ‘believes’, ‘anticipates’ and ‘expects’ and similar expressions are intended to identify forward-looking statements, including Ipsen’s expectations regarding future events, including regulatory filings and determinations. Moreover, the targets described in this document were prepared without taking into account external-growth assumptions and potential future acquisitions, which may alter these parameters. These objectives are based on data and assumptions regarded as reasonable by Ipsen. These targets depend on conditions or facts likely to happen in the future, and not exclusively on historical data. Actual results may depart significantly from these targets given the occurrence of certain risks and uncertainties, notably the fact that a promising medicine in early development phase or clinical trial may end up never being launched on the market or reaching its commercial targets, notably for regulatory or competition reasons. Ipsen must face or might face competition from generic medicine that might translate into a loss of market share. Furthermore, the research and development process involves several stages each of which involves the substantial risk that Ipsen may fail to achieve its objectives and be forced to abandon its efforts with regards to a medicine in which it has invested significant sums. Therefore, Ipsen cannot be certain that favorable results obtained during preclinical trials will be confirmed subsequently during clinical trials, or that the results of clinical trials will be sufficient to demonstrate the safe and effective nature of the medicine concerned. There can be no guarantees a medicine will receive the necessary regulatory approvals or that the medicine will prove to be commercially successful. If underlying assumptions prove inaccurate or risks or uncertainties materialize, actual results may differ materially from those set forth in the forward-looking statements. Other risks and uncertainties include but are not limited to, general industry conditions and competition; general economic factors, including interest rate and currency exchange rate fluctuations; the impact of pharmaceutical industry regulation and healthcare legislation; global trends toward healthcare cost containment; technological advances, new medicine and patents attained by competitors; challenges inherent in new-medicine development, including obtaining regulatory approval; Ipsen’s ability to accurately predict future market conditions; manufacturing difficulties or delays; financial instability of international economies and sovereign risk; dependence on the effectiveness of Ipsen’s patents and other protections for innovative medicines; and the exposure to litigation, including patent litigation, and/or regulatory actions. Ipsen also depends on third parties to develop and market some of its medicines which could potentially generate substantial royalties; these partners could behave in such ways which could cause damage to Ipsen’s activities and financial results. Ipsen cannot be certain that its partners will fulfil their obligations. It might be unable to obtain any benefit from those agreements. A default by any of Ipsen’s partners could generate lower revenues than expected. Such situations could have a negative impact on Ipsen’s business, financial position or performance. Ipsen expressly disclaims any obligation or undertaking to update or revise any forward-looking statements, targets or estimates contained in this press release to reflect any change in events, conditions, assumptions or circumstances on which any such statements are based, unless so required by applicable law. Ipsen’s business is subject to the risk factors outlined in its registration documents filed with the French Autorité des Marchés Financiers. The risks and uncertainties set out are not exhaustive and the reader is advised to refer to Ipsen’s latest Universal Registration Document, available on ipsen.com. 1 At constant exchange rates (CER), which exclude any foreign-exchange impact by recalculating the performance for the relevant period by applying the exchange rates used for the prior period.2 EMA: European Medicines Agency 3 Long-acting neurotoxin Attachment Ipsen PR_Q1 2025_15042025 Total sales growth of 11.6% at CER1, or 11.7% as reported, driven by all three therapeutic areas and including an increasing contribution from Iqirvo and Bylvay. Tovorafenib regulatory submission to EMA2 for pediatric low-grade glioma. Confirmation of full-year 2025 financial guidance. PARIS, FRANCE, 16 April 2025 – Ipsen (Euronext: IPN; ADR: IPSEY), a global specialty-care biopharmaceutical company, today presents sales for the first quarter of 2025. “Ipsen has delivered a strong start to 2025, building further momentum in the transformation of our company,” commented David Loew, Chief Executive Officer, Ipsen. “We continued to execute on our strategy with strong top-line growth and pipeline progression. I am pleased to see the rapid build-up of our Rare Cholestatic Liver disease franchise with two innovate medicines for five indications. 2025 is set to be an important year for Ipsen, with multiple launches underway and several milestones expected across our portfolio.” Full-year guidance Ipsen is confirming financial guidance for full-year 2025: Total sales growth greater than 5.0%, at constant currency. Based on the average level of exchange rates in March 2025, a limited effect on total sales from currencies is expected. Core operating margin greater than 30.0% of total sales, which includes additional R&D expenses from anticipated early and mid-stage external-innovation opportunities. Guidance includes expected negative impact on Somatuline sales due to increased generic competition in the U.S. and Europe. It excludes any impact from potential late-stage (Phase III clinical development or later) business development transactions. Upcoming Milestones Ipsen anticipates several key milestones across its portfolio in 2025, including: Cabometyx (CABINET trial) – Regulatory decision in the European Union for advanced pancreatic (pNETs) and extra-pancreatic (epNETs) neuroendocrine tumors (NETs). fidrisertib (FALKON trial) – Readout of the pivotal Phase IIb trial in fibrodysplasia ossificans progressiva (FOP). LANT3 (LANTIC trial) – Proof-of-concept data readout, evaluating its potential in aesthetics. Q1 pipeline progress The regulatory filing for tovorafenib was accepted by EMA for review in the European Union, marking an important step forward in the development of this potential treatment for pediatric low-grade glioma and reinforcing Ipsen’s commitment to innovation in rare and difficult-to-treat cancers. Ipsen also initiated the entry in Phase I of IPN01195, a RAF inhibitor, complementing IPN01194, an ERK inhibitor, and tovorafenib, two other assets targeting the MAPK pathway. Group refinancing Ipsen announced on March 19th the successful completion of its inaugural Rated Public Bond of €500 million with a coupon of 3.875%, maturing in March 2032. Following the disclosure of the Investment Grade ratings from S&P and Moody’s, this transaction was very well received and largely oversubscribed by a diversified and solid institutional investor base. This transaction is an important component of Ipsen’s refinancing plan which included the successful renewal of €1,5 billion syndicated Revolving Credit Facility, extending Ipsen’s debt maturity profile. Conference call A conference call and webcast for investors and analysts will begin today at 2pm CET. Participants can access the call and its details by registering here; webcast details can be found here. Calendar Ipsen intends to publish its half-year results on July 31st, 2025. Notes All financial figures are in € millions (€m). The performance shown covers the three-month period to 31 March 2025 (Q1 2025, the quarter), compared to the three-month period to 31 March 2024 (Q1 2024), unless stated otherwise. About Ipsen We are a global biopharmaceutical company with a focus on bringing transformative medicines to patients in three therapeutic areas: Oncology, Rare Disease and Neuroscience. Our pipeline is fueled by internal and external innovation and supported by nearly 100 years of development experience and global hubs in the U.S., France and the U.K. Our teams in more than 40 countries and our partnerships around the world enable us to bring medicines to patients in more than 100 countries. Ipsen is listed in Paris (Euronext: IPN) and in the U.S. through a Sponsored Level I American Depositary Receipt program (ADR: IPSEY). For more information, visit ipsen.com. Ipsen contacts InvestorsKhalid Deojee +33 6 69 09 12 96 MediaSally Bain +1 857 320 0517Anne Liontas +33 7 67 34 72 96 Disclaimers and/or forward-looking statements The forward-looking statements, objectives and targets contained herein are based on Ipsen’s management strategy, current views and assumptions. Such statements involve known and unknown risks and uncertainties that may cause actual results, performance or events to differ materially from those anticipated herein. All of the above risks could affect Ipsen’s future ability to achieve its financial targets, which were set assuming reasonable macroeconomic conditions based on the information available today. Use of the words ‘believes’, ‘anticipates’ and ‘expects’ and similar expressions are intended to identify forward-looking statements, including Ipsen’s expectations regarding future events, including regulatory filings and determinations. Moreover, the targets described in this document were prepared without taking into account external-growth assumptions and potential future acquisitions, which may alter these parameters. These objectives are based on data and assumptions regarded as reasonable by Ipsen. These targets depend on conditions or facts likely to happen in the future, and not exclusively on historical data. Actual results may depart significantly from these targets given the occurrence of certain risks and uncertainties, notably the fact that a promising medicine in early development phase or clinical trial may end up never being launched on the market or reaching its commercial targets, notably for regulatory or competition reasons. Ipsen must face or might face competition from generic medicine that might translate into a loss of market share. Furthermore, the research and development process involves several stages each of which involves the substantial risk that Ipsen may fail to achieve its objectives and be forced to abandon its efforts with regards to a medicine in which it has invested significant sums. Therefore, Ipsen cannot be certain that favorable results obtained during preclinical trials will be confirmed subsequently during clinical trials, or that the results of clinical trials will be sufficient to demonstrate the safe and effective nature of the medicine concerned. There can be no guarantees a medicine will receive the necessary regulatory approvals or that the medicine will prove to be commercially successful. If underlying assumptions prove inaccurate or risks or uncertainties materialize, actual results may differ materially from those set forth in the forward-looking statements. Other risks and uncertainties include but are not limited to, general industry conditions and competition; general economic factors, including interest rate and currency exchange rate fluctuations; the impact of pharmaceutical industry regulation and healthcare legislation; global trends toward healthcare cost containment; technological advances, new medicine and patents attained by competitors; challenges inherent in new-medicine development, including obtaining regulatory approval; Ipsen’s ability to accurately predict future market conditions; manufacturing difficulties or delays; financial instability of international economies and sovereign risk; dependence on the effectiveness of Ipsen’s patents and other protections for innovative medicines; and the exposure to litigation, including patent litigation, and/or regulatory actions. Ipsen also depends on third parties to develop and market some of its medicines which could potentially generate substantial royalties; these partners could behave in such ways which could cause damage to Ipsen’s activities and financial results. Ipsen cannot be certain that its partners will fulfil their obligations. It might be unable to obtain any benefit from those agreements. A default by any of Ipsen’s partners could generate lower revenues than expected. Such situations could have a negative impact on Ipsen’s business, financial position or performance. Ipsen expressly disclaims any obligation or undertaking to update or revise any forward-looking statements, targets or estimates contained in this press release to reflect any change in events, conditions, assumptions or circumstances on which any such statements are based, unless so required by applicable law. Ipsen’s business is subject to the risk factors outlined in its registration documents filed with the French Autorité des Marchés Financiers. The risks and uncertainties set out are not exhaustive and the reader is advised to refer to Ipsen’s latest Universal Registration Document, available on ipsen.com. 1 At constant exchange rates (CER), which exclude any foreign-exchange impact by recalculating the performance for the relevant period by applying the exchange rates used for the prior period.2 EMA: European Medicines Agency 3 Long-acting neurotoxin Attachment Ipsen PR_Q1 2025_15042025

▎药明康德内容团队报道 对于罕见病患者，每一款新药的诞生都是一束光！过去一年，在中国又有一批罕见病新药获批上市，为无数患者和家庭带来希望。在罕见病日到来的时节，让我们回顾这些进展，致敬科研与坚持的力量，期待更多生命被治愈。 根据中国国家药监局（NMPA）官方批件信息以及各公司公开资料，2024年全年有至少17款罕见病新药获批上市，覆盖Wilson病、阵发性睡眠性血红蛋白尿症、血友病、“渐冻症”、杜氏肌营养不良等十多种罕见病类型。（本文盘点范围为2024年首次获NMPA批准上市的1类、3.1类和5.1类新药；罕见病参考中国《第一批罕见病目录》及《第二批罕见病目录》） 图片来源：123RF Orphalan公司：盐酸曲恩汀片 作用机制：铜离子络合剂 适应症：Wilson病 2024年1月，Orphalan公司的四盐酸曲恩汀薄膜衣片获NMPA批准上市，用于治疗不耐受D-青霉胺治疗的成人、青少年和≥5岁儿童的Wilson病（威尔逊病，又称肝豆状核变性）。在Wilson病患者中，遗传突变使这种胆汁排泄途径失效，导致过量的铜在肝细胞中累积，并最终超过身体的安全储存能力，引起细胞损伤。曲恩汀（trientine）是一款铜离子络合剂，它的作用类似青霉胺，可以促使铜通过肾脏排泄出体外。 罗氏：可伐利单抗注射液 作用机制：抗C5循环单克隆抗体 适应症：PNH 2024年2月，罗氏（Roche）的可伐利单抗获NMPA批准上市，用于未接受过补体抑制剂治疗的阵发性睡眠性血红蛋白尿症（PNH）成人和青少年（≥12岁）患者。可伐利单抗是一种新型抗C5循环单克隆抗体，患者可进行每四周一次皮下注射。PNH是一种罕见且危及生命的综合征，患者容易出现乏力、骨髓衰竭、血红蛋白尿、贫血、血栓等症状。 诺贝仁医药：醋酸锌片 作用机制：铜吸收抑制剂 适应症：Wilson病（肝豆状核变性） 2024年2月，由诺贝仁制药（Nobelpharma）申报的5.1类新药醋酸锌片获NMPA批准上市， 用于治疗Wilson病。醋酸锌片是一种铜吸收抑制剂。研究表明，锌制剂既能阻止铜在肠道的吸收，也能清除沉积于组织中的铜，可作为该疾病的治疗方式之一。 武田：注射用替度格鲁肽 作用机制：GLP-2类似物 适应症：短肠综合征 2024年2月，武田（Takeda）研发的注射用替度格鲁肽获NMPA批准上市，用于治疗短肠综合征（SBS）成人和1岁及以上儿童患者。替度格鲁肽为一款人胰高血糖素样肽2（GLP-2）类似物。短肠综合征是指因各种原因引起广泛小肠切除后，肠道有效吸收面积减少，残存功能性肠管不能维持患者的营养或生长需求，出现以腹泻、酸碱/水/电解质紊乱、以及营养吸收和代谢功能障碍为主的症候群。研究证实，替度格鲁肽可以增加绒毛高度及隐窝深度，加强肠道上皮屏障，从而减轻局部炎症并改善肠道通透性，促进肠适应。 武田：注射用舒索凝血素α 作用机制：重组猪FⅧ（rpFⅧ）药物 适应症：获得性血友病A 2024年2月，武田注射用舒索凝血素α（susoctocog alfa）在中国获批上市，用于获得性血友病A成人患者按需治疗和出血事件的控制。舒索凝血素α是是一款重组猪FⅧ（rpFⅧ）药物，它与人序列凝血因子Ⅷ结构相似，序列同源，而且不容易受到抗人FⅧ自身抗体的灭活，可以替代人FⅧ发挥明显的止血作用。血友病（hemophilia）是一种X染色体连锁的隐性遗传性出血性疾病，可分为血友病A和血友病B。前者为凝血因子Ⅷ（FⅧ）缺乏，后者为凝血因子Ⅸ（FⅨ）缺乏，均由相应的凝血因子基因突变引起。 诺华：盐酸伊普可泮胶囊 作用机制：补体B因子口服抑制剂 适应症：PNH 2024年4月，诺华（Novartis）创新药物盐酸伊普可泮胶囊获得NMPA批准上市，用于治疗既往未接受过补体抑制剂治疗的阵发性睡眠性血红蛋白尿症（PNH）成人患者。盐酸伊普可泮胶囊是一款特异性补体B因子口服抑制剂，作用于免疫系统的补体旁路途径中的近端通路，可通过控制血管内溶血和血管外溶血，弥补抗C5抗体治疗的缺陷，为PNH提供了一种新型的口服单药靶向治疗选择。 爱施健：醋酸氟氢可的松片 作用机制：肾上腺皮质激素类产品 适应症：失盐型先天性肾上腺皮质增生症和失盐型原发性慢性肾上腺皮质功能减退症 2024年5月，爱施健（aspen）的醋酸氟氢可的松片上市申请已获得NMPA批准上市，用于治疗失盐型先天性肾上腺皮质增生症（CAH）和失盐型原发性慢性肾上腺皮质功能减退症（Addison病，艾迪生病）。其中，21-羟化酶缺乏症（21-OHD）是先天性肾上腺皮质增生症中最常见的类型，是由于编码21-羟化酶的基因缺陷导致肾上腺皮质类固醇激素合成障碍的一种先天性疾病，患者需要每天甚至终身服药。醋酸氟氢可的松片为肾上腺皮质激素类产品，能抑制结缔组织增生，降低毛细血管和细胞膜的通透性，减少炎性渗出，抑制组胺及其他炎症介质的形成和释放。 诺和诺德：注射用培妥罗凝血素α 作用机制：长效重组凝血因子VIII 适应症：血友病A 2024年7月，诺和诺德（Novo Nordisk）注射用培妥罗凝血素α获NMPA批准上市，用于12岁及以上成人和儿童血友病A患者按需治疗及控制出血事件、围手术期管理、常规预防治疗以防止或减少出血事件的发生。诺和诺德新闻稿指出，这是中国获批的首个长效重组凝血因子VIII（FVIII），开启了血友病长效治疗的新时代。注射用培图罗凝血素α（N8-GP）经创结构修饰后可将半衰期延长1.6倍，以长效治疗帮助血友病患者减少输液次数，减轻治疗负担。 元羿生物/Marinus公司：加那索龙口服混悬剂 作用机制：靶向GABAA受体的阳性别构调节剂 适应症：“希舞综合征” 2024年7月，元羿生物引进自Marinus公司的的加那索龙口服混悬剂在中国获批上市，用于治疗2岁及以上细胞周期蛋白依赖性激酶5（CDKL5）缺乏症患者癫痫发作。CDKL5缺乏症（又称为“希舞综合征”）是由位于X染色体上的CDKL5基因突变引起，具有难以控制的癫痫发作和重度神经发育障碍等特征。加那索龙（ganaxolone）是一款靶向GABAA受体的阳性别构调节剂，具有静脉注射和口服两种给药方式。它作用于神经元突触和突触外GABAA受体，从而达到抗癫痫和抗焦虑活性的效用。 药华医药：罗培干扰素α-2b注射液 作用机制：超长效干扰素 适应症：真性红细胞增多症 2024年7月，药华医药的罗培干扰素获得NMPA批准上市，用于治疗真性红细胞增多症（PV），这是一种造血干细胞恶性肿瘤。罗培干扰素是单一异构体超长效干扰素，可2~4周注射1次。经多项临床研究显示，该产品可快速有效控制血细胞的过度增殖、并显著降低致病基因的突变负荷，从而有效降低血栓栓塞并发症及其所导致的死亡风险。 武田：注射用伏尼凝血素α 作用机制：重组血管性血友病因子 适应症：血管性血友病 2024年8月，武田的注射用vonicog alfa（注射用伏尼凝血素α）在中国获批上市，用于血管性血友病成人患者（18岁及以上）的按需治疗和出血事件控制，以及围手术期出血管理。血管性血友病是常见的遗传性出血性疾病之一，主要是由于血管性血友病因子（VWF）基因突变引起血浆中的血管性血友病因子数量减少或质量异常。根据不同的类型，VWD患者的出血严重程度差异大。其中血管性血友病Ⅲ型已在中国被纳入《第二批罕见病目录》。注射用伏尼凝血素α是一款重组血管性血友病因子。它包含完整的VWF多聚体，含有超大多聚体，半衰期长，可有效实现血管性血友病的替代治疗。 兴科蓉药业/奥克特珐玛：注射用重组人凝血因子Ⅷ 作用机制：重组人凝血因子Ⅷ 适应症：血友病A 2024年8月，奥克特珐玛（Octapharma）和兴科蓉药业共同申报的重组人凝血因子Ⅷ获NMPA批准上市。重组人凝血因子Ⅷ是一种用于血友病A替代治疗的产品。 渤健：托夫生注射液 作用机制：反义寡核苷酸（ASO）药物 适应症：SOD1基因突变的ALS成人患者 2024年10月，由渤健（Biogen）和Ionis Pharmaceuticals联合开发的托夫生注射液在中国获批上市，用于治疗携带超氧化物歧化酶1（SOD1）基因突变的肌萎缩侧索硬化（ALS）成人患者。ALS是一种进行性神经退行性疾病，也被称为“渐冻症”。许多基因被认为与此疾病有关联，其中具有SOD1突变的患者（SOD1-ALS）约占2%。且患者中大部分疾病进展迅速。托夫生注射液是一种反义寡核苷酸（ASO）药物，可通过减少SOD1蛋白合成，减少毒性SOD1蛋白的蓄积，从而减轻运动神经元的损伤，减缓疾病进展。 安进：阿伐可泮胶囊 作用机制：口服选择性补体5a（C5a）受体抑制剂 适应症：ANCA相关血管炎 2024年11月，安进（Amgen）公司的阿伐可泮胶囊获NMPA批准上市，适用于成人严重、活动性抗中性粒细胞胞浆抗体（ANCA）相关性血管炎（肉芽肿性多血管炎[GPA]或显微镜下多血管炎[MPA]）在含糖皮质激素的标准治疗下的辅助治疗。ANCA相关血管炎（AAV）是一种罕见的血管炎症性疾病，其疾病机制是由于C5a补体途径的过度激活，进一步激活中性粒细胞，导致全身炎症反应和小血管破坏，进而可能导致肾脏、肺和其他器官的严重损伤和功能障碍。阿伐可泮是一款口服选择性补体5a（C5a）受体抑制剂，它通过精确阻断位于中性粒细胞等炎症细胞上的C5a受体，阻止这些细胞受到C5a的激活，降低炎症损伤。 华东医药：注射用利纳西普 作用机制：IL-1抑制剂 适应症：CAPS、复发性心包炎 2024年11月，华东医药引进的注射用利纳西普获NMPA批准上市，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），包括家族性寒冷型自身炎症综合征（FCAS）、Muckle-Wells综合征（MWS）。同年12月，该产品获批罕见病新适应症，用于治疗成人和12岁及以上青少年复发性心包炎（RP）以及降低复发风险。注射用利纳西普是一款每周进行皮下注射给药的重组二聚体融合蛋白，可阻断白细胞介素-1α（IL-1α）和IL-1β的信号传导。 CAPS是与NLRP3基因缺陷有关的一组罕见的炎症性疾病。NLRP3基因突变的患者会不断过度产生IL-1β分子，从而引起炎症。该病的临床特征是反复发作的多系统炎症，累及皮肤、肌肉、骨骼、关节、眼、耳以及中枢神经系统等。复发性心包炎是一种罕见的自身炎症性的心血管疾病，患者会有反复的胸痛发作，且伴有危及生命的并发症。 益普生：奥德昔巴特胶囊 作用机制：IBAT抑制剂 适应症：进行性家族性肝内胆汁淤积症 2024年12月，益普生（Ipsen）公司奥德昔巴特胶囊（odevixibat）获NMPA批准上市，用于治疗≥6月龄的进行性家族性肝内胆汁淤积症（PFIC）患者的瘙痒。PFIC是一种罕见的儿童疾病，会导致进行性、危及生命的肝病。在许多情况下，PFIC在患者出生后10年内会发生肝硬化和肝衰竭。PFIC最突出的持续症状是剧烈瘙痒，患者的生活质量严重下降。奥德昔巴特胶囊是一种强效、非体循环回肠胆汁酸转运蛋白（IBAT）抑制剂，可通过抑制回肠远端胆汁酸的再摄取，增加胆汁酸通过结肠的清除，降低血清中胆汁酸浓度，减轻患者瘙痒症状。 曙方医药：伐莫洛龙口服混悬液 作用机制：新型解离性皮质类固醇 适应症：DMD 2024年12月，曙方医药引进自Santhera Pharmaceuticals的伐莫洛龙口服混悬液（vamorolone）在中国获批上市，用于治疗4岁及以上杜氏肌营养不良（DMD）患者。DMD是一种致命的罕见X连锁退行性神经肌肉疾病。伐莫洛龙的作用机制与糖皮质激素结合的受体相同，但改变了其下游活性，且不会造成局部组织扩增和局部组织中皮质类固醇相关毒性。 让罕见病不再“罕治”！我们期待在全球各界的共同携手努力下，罕见病治疗领域可以涌现更多创新成果，为更多患者照亮未来。 本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权或其他合作需求，请联系wuxi_media@wuxiapptec.com。 免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。