在北京友谊医院顺义院区，患者刘丽（化名）拿到了中国内地开出的第一盒司拉德帕。刘丽已经确诊原 发性胆汁性胆管炎多年，选择目前一线治疗药物后，相关指标没有显著好转，经充分沟通后，决定试用 罕见病创新药司拉德帕。"希望这款创新药能够改善我的情况，同时希望此类药物早日纳入医保，惠及 更多患者。"刘丽说。 原发性胆汁性胆管炎是一种慢性胆汁淤积性肝病，以肝内小胆管的进行性破坏为特征。这类患者疾病进 展风险高，可逐渐进展至肝纤维化、肝硬化，并出现消化道出血、腹水、肝性脑病等并发症。目前，约 30%至40%患者对目前该领域一线用药疗效欠佳，亟需更有效的创新治疗方案。 新京报讯 据"北京顺义"微信公众号消息，1月6日上午，北京友谊医院顺义院区开出罕见病创新药司拉 德帕（Seladelpar）的中国内地首张处方，这款药物专治原发性胆汁性胆管炎。这不仅标志着我国罕见 病治疗领域迈出重要一步，也为更多创新药物的引进提供了可借鉴范式。 在北京市临床急需药品临时进口政策东风以及科园信海（北京）医疗用品贸易有限公司的高效协作下， 司拉德帕仅历时两个多月便快速获批，为罕见病患者带来福音。 2025年9月，48盒司拉德帕已经在天竺综保区内保税 ...

Analyst Ratings - Weiss Ratings restated a "hold (c-)" rating on Pharming Group shares [1] - Zacks Research upgraded Pharming Group from "hold" to "strong-buy" [1] - Wall Street Zen also upgraded Pharming Group to a "strong-buy" rating [1] - The average rating for Pharming Group is "Buy" with a consensus target price of $38.00 [1] Financial Performance - Pharming Group has a market capitalization of $1.21 billion and a P/E ratio of -1,765.23 [2][3] - The company reported earnings of $0.10 per share, exceeding analysts' expectations of $0.05 by $0.05 [4] - Revenue for the quarter was $97.30 million, slightly below the consensus estimate of $98.22 million [4] - The company has a quick ratio of 2.39, a current ratio of 3.16, and a debt-to-equity ratio of 0.35 [2][3] Stock Performance - Pharming Group's share price increased by 4.4% to $17.55, with a trading volume of approximately 5,296 shares [8] - The stock had previously closed at $16.81 and traded as high as $17.32 during the day [8] Institutional Activity - EverSource Wealth Advisors LLC purchased 2,979 shares of Pharming Group, valued at approximately $32,000 [5] - Institutional investors currently own 0.03% of Pharming Group's stock [5] Company Overview - Pharming Group N.V. is a clinical-stage biopharmaceutical company based in Leiden, Netherlands, focusing on innovative protein replacement therapies for rare diseases [6] - The company's lead product, RUCONEST, is approved for treating acute hereditary angioedema (HAE) attacks in multiple markets [7]

编者按：寡核苷酸疗法正成为罕见病治疗的重要突破方向。当前，全球范围内已有十多款寡核苷酸疗法获监管机构批准治疗罕见病，此外还有上百项寡核苷 酸疗法管线在针对罕见病适应症进行临床研究，未来有望造福更多患者。为帮助合作伙伴更高效地推动寡核苷酸药物从实验室走向临床，药明康德化学业务 旗下WuXi TIDES平台围绕寡核苷酸、多肽及其相关化学偶联药物建立了一体化解决方案，覆盖定制合成、共价偶联、工艺开发和CMC等关键环节，赋能创 新项目加速进入临床阶段。本文将回顾2025年寡核苷酸新药在罕见病领域取得的部分新进展。 再如，3月，赛诺菲（Sanofi）与Alnylam联合开发的siRNA疗法Qfitlia（fitusiran）获FDA批准上市，用于预防或减少12岁以上血友病A和血友病B患者的出血 事件，无论患者体内是否含有凝血因子VIII或IX的抑制物。该药通过降低抗凝血酶水平，从而促进凝血酶生成，重新平衡止血功能并预防出血，为血友病患 者提供了长效预防新选择。 寡核苷酸药物是全球新药开发的重要热点，正为众多曾被视为"不治之症"的罕见病带来新的治疗希望。 近年来，该领域研发进展持续加速。公开资料显示，全球已有十多款罕见病 ...

12月22日，盐野义宣布计划收购田边制药的肌萎缩侧索硬化症（ALS，俗称"渐冻症"）药物业务板块，其中核心资产包括口服 混悬剂Radicava ORS及其静脉注射剂型，同时涵盖相关商业化运营能力。 盐野义方面表示，预计该交易将于2026年4月1日或之后完成交割，交易完成后每年将为公司新增约7亿美元的全球销售额。 根据官方披露，盐野义将以25亿美元的一次性付款方式收购Radicava相关业务，同时约定在"特定条件"下支付潜在的未来特许 权使用费。交易交割后，Radicava业务单元将成为盐野义的全资子公司。 盐野义强调，此次收购将助力公司在美国市场搭建"强大的罕见病商业化平台"，并为其计划推出的脆性X综合征、乔丹综合征 及庞贝病等适应症药物提供支持。 值得一提的是，Radicava静脉注射剂型早在2017年就获得美国FDA批准，成为数十年来首款获批的新型ALS治疗药物；时隔5 年，其口服剂型也顺利通过美国监管机构审批，为患者提供了更便捷的治疗选择。 近期盐野义在并购市场动作频频：本月刚从日本烟草公司收购了多项"早期阶段罕见病资产"，此前还与瑞士生物技术公司 BioVersys达成合作，获得一款临床前抗生素项目的 ...

渤健制药（BMRN）于周五宣布，将以约 48 亿美元的价格收购阿米库斯治疗公司（FOLD），此举旨 在扩大其在罕见代谢性疾病领域的业务版图。 渤健将以每股 14.50 美元的价格收购阿米库斯治疗公司股份，较该公司最新收盘价溢价 33.1%。受此消 息影响，阿米库斯治疗公司股价暴涨 30%，渤健股价在盘前交易中也上涨了近 5%。 通过这笔交易，渤健的产品组合将得到进一步充实，获得阿米库斯治疗公司已获批的多款遗传病治疗药 物。其中包括治疗法布里病的口服药加拉福，法布里病是由基因缺陷引发的疾病，会导致脂肪类物质在 细胞内堆积。 责任编辑：郭明煜 渤健制药（BMRN）于周五宣布，将以约 48 亿美元的价格收购阿米库斯治疗公司（FOLD），此举旨 在扩大其在罕见代谢性疾病领域的业务版图。 渤健将以每股 14.50 美元的价格收购阿米库斯治疗公司股份，较该公司最新收盘价溢价 33.1%。受此消 息影响，阿米库斯治疗公司股价暴涨 30%，渤健股价在盘前交易中也上涨了近 5%。 通过这笔交易，渤健的产品组合将得到进一步充实，获得阿米库斯治疗公司已获批的多款遗传病治疗药 物。其中包括治疗法布里病的口服药加拉福，法布里病是由基因 ...

Summary of BioMarin Pharmaceutical's Acquisition of Amicus Therapeutics Conference Call Company and Industry - **Company**: BioMarin Pharmaceutical (NasdaqGS: BMRN) - **Acquisition Target**: Amicus Therapeutics - **Industry**: Rare diseases and biopharmaceuticals Core Points and Arguments 1. **Strategic Fit**: The acquisition of Amicus Therapeutics is seen as an exceptional strategic fit for BioMarin, enhancing its position in the rare diseases market with innovative therapies like Galafold and POMOP [4][6][17] 2. **Immediate Revenue Growth**: The deal is expected to accelerate BioMarin's revenue growth immediately upon closing, with both Galafold and POMOP projected to reach peak sales of $1 billion each [5][21][53] 3. **Financial Outlook**: The transaction is anticipated to be accretive to non-GAAP diluted EPS within the first 12 months and substantially accretive starting in 2027. The combined company is expected to generate additional cash flow, allowing for continued investment in innovation [5][8][14][76] 4. **Transaction Details**: BioMarin will acquire Amicus for $14.50 per share, valuing the deal at $4.8 billion, financed through cash and approximately $3.7 billion of non-convertible debt [7][8] 5. **Market Expansion Opportunities**: There are significant opportunities for expanding the reach of Galafold and POMOP in existing and new markets, with a focus on increasing diagnosis and treatment rates for underdiagnosed conditions like Fabry and Pompe diseases [11][24][73] Additional Important Content 1. **Intellectual Property Settlements**: Amicus has settled ongoing litigation related to Galafold's IP, preventing competitors from entering the U.S. market before 2037, which supports the growth outlook for Galafold [12][69] 2. **Underdiagnosed Conditions**: Both Fabry and Pompe diseases are considered underdiagnosed, with estimates suggesting a higher prevalence than currently diagnosed patients. This presents a significant opportunity for BioMarin to increase market penetration [23][24] 3. **Synergies and Integration**: The integration of Amicus is expected to yield operational synergies, leveraging BioMarin's scale to enhance the growth of both products. The focus will be on maintaining Amicus's capabilities while integrating operations [9][36][58] 4. **Long-term Growth Potential**: The combined business is projected to grow at a higher rate through the rest of the decade, with both products expected to contribute significantly to revenues by 2027 [39][53] 5. **Competitive Landscape**: BioMarin aims to differentiate Galafold and POMOP from existing therapies through unique mechanisms and real-world evidence supporting their efficacy, particularly in driving patient switches from traditional enzyme replacement therapies [42][54] This summary encapsulates the key points discussed during the conference call regarding BioMarin's acquisition of Amicus Therapeutics, highlighting the strategic, financial, and operational implications of the deal.

Acquisition Overview - BioMarin will acquire Amicus Therapeutics in an all-cash transaction for $14.50 per share, valuing Amicus' equity at approximately $4.8 billion[14] - The purchase price represents a 33% premium to Amicus' closing stock price on December 18, 2025[14] - The transaction is expected to close in Q2 2026, subject to regulatory clearance and approval by Amicus' stockholders[14] Financial Impact - The acquisition is expected to increase BioMarin's long-term revenue CAGR through 2030 and beyond[12] - It is anticipated to be accretive to Non-GAAP Diluted Earnings Per Share (EPS) in the first 12 months after close and substantially accretive beginning in 2027[12, 28] - BioMarin is committed to deleveraging, targeting gross leverage < 2.5x within two years after close[12, 28] - The transaction will be financed through a combination of cash on hand and approximately $3.7 billion of non-convertible debt financing[14] Strategic Rationale - The acquisition expands BioMarin's position as a leader in rare diseases by adding two marketed, high-growth products[4, 12, 28] - BioMarin's global scale and manufacturing capabilities will enable more patients to benefit from Galafold and Pombiliti + Opfolda[12, 28] - The deal diversifies BioMarin's revenue mix and strengthens its commercial portfolio[5, 12, 15] Product Portfolio - Galafold, an oral therapy for Fabry disease, is projected to generate $458 million in revenue in FY'25, with a growth rate of +10-15%[19] - Pombiliti + Opfolda, a two-component therapy for Pompe disease, is projected to generate $70 million in revenue in FY'25, with a growth rate of +50-65%[24]

12月14日晚间，一品红（300723）发布公告，公司参股公司Arthrosi收到Sobi美国发出的相关协议条 款，Sobi美国拟以9.5亿美元首付款(折合人民币约67.13亿元)，以及最高达5.5亿美元(折合人民币约38.87 亿元)的临床、注册和销售里程碑付款收购Arthrosi100%股权，总价值为15亿美元(折合人民币约106亿 元)。 根据公告，此次交易的收购方Sobi美国系瑞典医药巨头Swedish Orphan Biovitrum AB(简称"Sobi"，斯德 哥尔摩证券交易所股票代码：SOBI.ST)的全资子公司。Sobi是一家专注于罕见病领域的国际生物医药公 司，主要治疗领域包括血液学、免疫学和专科护理。 核心管线涉及难治性痛风 已提前布局生产环节 值得一提的是，为了AR882能够早日上市，一品红在生产环节已经提前作了充分的布局准备。 2024年10月，一品红子公司瑞石制药的创新原料药基地在韶关翁源举行试投产仪式。该基地按照美国 FDA和欧盟GMP等国际标准设计建设，一期占地110亩，已建成面积超过5万平方米，建有多条先进生 产线，具备大规模的仿制药、创新药原料药供应生产能力。 2025年5 ...

Core Insights - Rezolute, Inc. faced a significant setback with its Phase 3 sunRIZE study for treating congenital hyperinsulinism, leading to a dramatic decline in stock performance [1][5] Group 1: Study Results - The sunRIZE study did not meet its primary endpoint, with the highest dose of ersodetug achieving only a 45% reduction in hypoglycemia events, which was not statistically significant compared to the placebo group's 40% reduction [2][5] - The trial also failed to meet its key secondary endpoint, showing a 25% reduction in average daily percent time in hypoglycemia for the top dose, while the placebo group experienced a 5% increase [3][5] - Despite these failures, the company reported that target drug concentrations were achieved across all age groups, and safety observations were generally favorable, although two participants experienced serious hypersensitivity reactions [3] Group 2: Stock Performance - Following the disappointing trial results, Wedbush set a price target of $1 for RZLT, indicating a potential decrease of approximately -19.68% from its trading price of $1.245 [2][5] - The stock has seen a significant decline of 88.21% from its previous value, currently priced at $1.29, with fluctuations between a low of $1.07 and a high of $1.41 [4][5] - Over the past year, the stock reached a high of $11.46 and a low of $1.07, with a current market capitalization of approximately $119.62 million and a trading volume of 91.55 million shares [4]

Savara (NasdaqGS:SVRA) 2025 Conference Summary Company Overview - Savara is a single-asset rare disease company focused on the orphan pulmonary respiratory space, specifically targeting autoimmune pulmonary alveolar proteinosis (aPAP) with its product Molrivi (molgramostim inhalation solution) [4][5] Key Points Product Development and Milestones - Positive phase three data for Molrivi was reported in summer 2024 for the treatment of autoimmune PAP [4] - The company plans to submit a Biologics License Application (BLA) next month and Marketing Authorization Applications (MAAs) in the EU and U.K. in the first quarter of 2025 [4] - The U.S. launch is anticipated around August or September 2025 [5] Market Opportunity - The estimated diagnosed prevalence of aPAP in the U.S. ranges from 6-26 per million, with Savara's analysis suggesting approximately 5,500 patients diagnosed with aPAP [6][8] - The pricing corridor for Molrivi is expected to be between $400,000-$500,000 per patient per year, reflecting strong pricing power due to clinical data [12] Patient Population Insights - The analysis identified over 6,100 patients with a PAP diagnosis, with a focus on ensuring unique patient counts and accurate diagnoses [7][9] - There is potential for a significant number of undiagnosed patients, with estimates suggesting the total could reach 9,000-10,000 based on literature [14] Compliance and Adherence - High compliance is expected due to the favorable safety profile of Molrivi, with 159 out of 164 patients completing the IMPALA-2 trial [15][16] - The once-daily dosing and ease of use of the nebulizer are anticipated to enhance patient adherence [17] Regulatory Confidence - The company is confident in addressing previous regulatory issues related to manufacturing, having transitioned to Fujifilm for production [18][20] - The BLA resubmission is on track for December, with expectations for FDA acceptance in February 2025 [19] Market Preparedness - Savara plans to launch independently in the U.S., Europe, and U.K., leveraging existing relationships with key opinion leaders and reference centers [22][23] - The company is building a market development team ahead of the launch to ensure readiness [24] Financial Position - Savara currently has approximately $264 million on its balance sheet, providing a strong financial foundation for its upcoming initiatives [4] Intellectual Property and Market Exclusivity - Molrivi is expected to receive 12 years of regulatory exclusivity in the U.S. and 10 years in Europe, with additional patents in place for the drug-device combination and formulation [31][32] Additional Insights - The company emphasizes the importance of early intervention in aPAP treatment to prevent severe complications, such as lung lavages [29] - There is a commitment to educating payers about the disease and the clinical benefits of Molrivi to facilitate reimbursement [12][21] This summary encapsulates the critical aspects of Savara's conference call, highlighting the company's strategic direction, market potential, and operational readiness for the upcoming product launch.