2025全球罕见病行业发展报告： 政策演进、市场趋势与领先企业布局 摩熵咨询 2026年1月 生命科学领域全球领先的数据系统与咨询服务提供商 摩熵数科 www.bcpmdata.com 2 1 罕见病行业概览 中、美、日、欧罕见病政策分析 3 全球五大热门研发罕见病概述 4 全球罕见病领域五大药物研发企业 中美日欧罕见病的定义 中美日欧罕见病影响人数 中国罕见病用药可及性对比 国内罕见病有药可用疾病列举 中美日欧罕见病管理格局：各国对罕见病的定义有差异，中国按照目录管理，已有207种疾病纳入罕见病目录 ü 中美日欧对罕见病的定义： • 中美日欧各国对于罕见病的定义并非是统一的医学概念，而是政策导向的结果。 美国以"患者人数"为核心，日本以"医保可负担性"为核心，欧盟强调"跨 国协同"，中国起步较晚仍处于"目录建设+登记体系完善"的阶段。 表1. 中美日欧对罕见病的定义标准及相关法律/政策依据 • 中国通过国家罕见病目录确定纳入疾病并由政府主导更新；美国没有统一 目录，而是通过 GARD公开数据库管理相关疾病和药物信息；日本以难病 法为基础建立难病名录，由学会和政府共同评估并纳入医保补助；欧盟未 设统一目录，但 ...

业绩减亏因公司拓展市场，阿达木单抗、托珠单抗销售额提升，乌司奴单抗在美国上市，授权许可及销 售收入增长。 近日，百奥泰发布公告称，预计 2025 年度实现归属于母公司所有者的净利润与上年同期相比，将减少 亏损，实现归属于母公司所有者的净利润为-28,000.00 万元到-39,000.00 万元，减少亏损 12,030.38 万元 到 23,030.38 万元。 （百奥泰公告） ...

百奥泰公告称，2025年1 - 12月，公司预计实现营收9 - 9.7亿元，较上年增加1.57 - 2.27亿元。归属于母 公司所有者的净利润为-2.8 - -3.9亿元，将减少亏损1.20 - 2.30亿元；扣非净利润为-3.8 - -4.9亿元，将减 少亏损0.66 - 1.76亿元。业绩减亏因公司拓展市场，阿达木单抗、托珠单抗销售额提升，乌司奴单抗在 美国上市，授权许可及销售收入增长。 ...

Group 1: Product Performance and Market Strategy - The patent protection for the injectable form of Esomeprazole will last until after 2035, while the tablet form has faced generic competition in China, yet the company has maintained stable sales growth in the first three quarters of this year due to its strong presence in the gastrointestinal sector [2] - The P-CAB product JP-1366 is expected to participate in the 2027 medical insurance negotiations, with sales anticipated to ramp up significantly after its market entry [2][3] - The domestic sales of P-CAB products reached approximately CNY 1.45 billion in the first three quarters of 2025, representing a year-on-year growth of 65% [3] Group 2: Competitive Landscape in Mental Health Treatments - The average usage rate of long-acting antipsychotic injections in Europe and the U.S. is between 25%-30%, while the rate in China is currently below 1% [3] - The new guidelines for schizophrenia treatment emphasize the importance of long-acting therapies, which is expected to increase the market penetration and sales of the company's long-acting antipsychotic products [3][4] - The company strategically focuses on developing third-generation antipsychotic drugs for long-acting formulations to avoid safety issues associated with first- and second-generation drugs [4] Group 3: Pipeline and Future Developments - The IL-17A/F monoclonal antibody (LZM012) has been submitted for regulatory approval and is expected to participate in the 2027 national medical insurance negotiations [5] - The recombinant human follicle-stimulating hormone (r-FSH) is set to launch in 2026, enhancing the company's product matrix in assisted reproduction [5][6] - The company has significantly reduced losses this year and anticipates entering a product harvest phase with the upcoming launches of r-FSH and LZM012 [6]

Core Viewpoint - 百奥泰 reported a revenue of 684 million yuan for the first three quarters of 2025, representing a year-on-year growth of 17.57%, while the net loss narrowed to 224 million yuan, indicating improved financial performance [1] Group 1: Financial Performance - The company achieved a revenue of 684 million yuan in the first three quarters, marking a 17.57% increase year-on-year [1] - The net loss for the same period was 224 million yuan, showing a reduction in losses compared to the previous year [1] Group 2: Product Development and Approvals - Several products have been approved for sale, including Adalimumab and Tocilizumab in China and other regions, and Bevacizumab in multiple countries [1] - BAT1806 (Tocilizumab) and BAT2206 (Ustekinumab) have been approved in Europe, with sales managed by partners [2][3] - The company is advancing its innovative drug pipeline, with BAT5906 and BAT4406F expected to submit applications for market approval soon [2][3] Group 3: Market Expansion and Strategy - The company is preparing for participation in centralized procurement, optimizing production capacity and supply chain to enhance competitiveness [2] - New guidelines in the EU and the US for biosimilars are expected to lower R&D costs, providing a competitive advantage for the company [2] Group 4: Clinical Trials and Research - BAT6026 is currently in Phase II clinical trials for atopic dermatitis, while BAT8008 is set to begin Phase III trials for cervical cancer and HER2-negative breast cancer in mid-2026 [3] - Ongoing clinical research for BAT8008 in combination with BAT1308 has shown positive efficacy signals, leading to an expansion of the sample size [3] - BAT7111 is in Phase I dose escalation studies, progressing smoothly with three doses explored [3]

Core Insights - IL-6 has emerged as a significant target in autoimmune diseases, with four drugs targeting IL-6/IL-6R approved globally, including tocilizumab, which reached sales of $3.96 billion in 2021 due to its first-mover advantage and multiple indications [1][3] - The expiration of tocilizumab's patent and the entry of biosimilars have intensified competition, prompting pharmaceutical companies to explore new indications beyond rheumatic diseases [3][6] - Recent acquisitions, such as Novartis's $1.4 billion purchase of Tourmaline Bio, indicate a strategic focus on differentiating therapies targeting IL-6 in cardiovascular diseases [4][6] Group 1: IL-6's Role and Market Dynamics - IL-6 is a multifunctional cytokine involved in various physiological and pathological processes, playing a crucial role in inflammation and immune response [2] - The complexity of IL-6's functions makes it challenging to develop drugs that inhibit its harmful effects while preserving its beneficial roles [2][8] - The cardiovascular field is becoming a rapidly advancing area for IL-6 monoclonal antibodies, with Novartis and Novo Nordisk actively pursuing this market [5][7] Group 2: Clinical Developments and Challenges - Novartis's acquisition of Tourmaline Bio was driven by promising Phase II clinical results of pacibekitug, which significantly reduced hs-CRP levels, indicating anti-inflammatory effects [6][8] - The ongoing clinical trials, such as ZEUS, will be critical in validating the efficacy of IL-6 inhibitors in improving hard endpoints like myocardial infarction and stroke [8] - Potential risks associated with long-term IL-6 suppression, including infection and liver toxicity, necessitate careful monitoring in clinical settings [8][9] Group 3: Future Directions and Research - The exploration of IL-6's role in chronic kidney disease and ocular diseases is gaining momentum, with ongoing studies indicating its potential in these areas [9][10] - Innovative approaches, such as dual-targeting antibodies and personalized medicine, are being developed to enhance treatment efficacy for conditions like rheumatoid arthritis [10] - The quest to understand the underlying mechanisms of elevated IL-6 levels and its varied effects across diseases remains a critical area for future research [10]

Core Viewpoint - Baotai Biopharmaceutical Co., Ltd. has received approval from the European Commission for Usymro®, a biosimilar drug for treating moderate to severe plaque psoriasis, active psoriatic arthritis, and moderate to severe active Crohn's disease in adults and children, marking a significant milestone for the company [2][3] Company Overview - Baotai is a global biopharmaceutical company based in Guangzhou, China, focused on developing innovative drugs and biosimilars for various diseases, including cancer and autoimmune disorders [2][3] - The company has successfully launched multiple drugs, including Adalimumab and Tocilizumab, in various markets, establishing itself as a leader in antibody drug development [3] Recent Developments - The approval of Usymro® follows a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) in June 2025, highlighting the company's commitment to advancing its biosimilar pipeline [2] - In October 2024, Baotai signed a licensing and commercialization agreement with Gilead Sciences for BAT2206, another biosimilar, covering markets in the EU, UK, Switzerland, and other regions [2]

Core Viewpoint - The article discusses the extension of centralized procurement to biosimilars in China, highlighting the implications for market dynamics and pricing strategies in the pharmaceutical industry [4][6]. Group 1: Centralized Procurement of Biosimilars - The Anhui Provincial Medical Procurement Platform has initiated information reporting for eight monoclonal antibody products, marking the beginning of centralized procurement for biosimilars [4]. - Biosimilars are large molecule biological products that are similar in quality, safety, and efficacy to already approved reference drugs, and their development is typically more costly and time-consuming than small molecule drugs [4][8]. - The centralized procurement process for biosimilars is expected to take longer than previous chemical drug procurements due to the need for comprehensive data collection and rule formulation [6]. Group 2: Market Impact and Dynamics - The total sales of the eight involved biosimilar products are projected to approach 30 billion yuan in 2024, with specific products like Bevacizumab exceeding 10 billion yuan in sales [9]. - The article emphasizes that the centralized procurement of biosimilars will not drastically disrupt existing market structures but will lead to adjustments within the current competitive landscape [9][10]. - The involvement of multiple companies in the production of these biosimilars indicates a competitive environment, with several products having three or more approved manufacturers [8][9]. Group 3: Characteristics of Biosimilar Procurement - Compared to chemical drug procurement, biosimilar procurement is characterized by smaller price reductions, comprehensive reporting requirements, and a focus on high-quality products [13][14]. - The procurement process aims to clarify market demand and supply, allowing companies to better predict future business conditions and adjust their product pipelines accordingly [6][10]. - The article suggests that the pricing adjustments in biosimilar procurement will be more moderate than those seen in previous chemical drug procurements, reflecting the unique nature of biological products [14][15].

Core Viewpoint - The introduction of centralized procurement for biosimilars in China marks a significant shift in the pharmaceutical industry, aiming to enhance drug accessibility and affordability while maintaining quality standards [1][3][6]. Group 1: Centralized Procurement Overview - The centralized procurement initiative has officially extended to biosimilars, with the Anhui Provincial Medical Procurement Platform issuing a notification for companies to report pricing, sales volume, and production capacity for specific monoclonal antibody products [1][3]. - This marks the first instance of centralized procurement for biosimilars in mainland China, with expectations for a longer timeline for information organization and rule formulation compared to previous chemical drug procurements [1][2]. Group 2: Market Impact and Dynamics - The market for the eight involved biosimilar products is projected to reach nearly 30 billion yuan, with specific products like Bevacizumab exceeding 10 billion yuan in sales [4][6]. - The competitive landscape is expected to be influenced but not drastically altered, as the procurement process aims to provide a clearer understanding of market volumes and guide companies in their operational strategies [2][4]. Group 3: Characteristics of Biosimilar Procurement - Compared to chemical drug procurement, biosimilar procurement is characterized by smaller price reductions, comprehensive reporting requirements, and a focus on high-quality products [7][8]. - The procurement process emphasizes a data-driven approach, requiring companies to report detailed sales figures and product specifications to ensure a thorough understanding of the market [7][9]. Group 4: Future Expectations - The impact of the biosimilar procurement is anticipated to manifest in the performance of related companies and product sales starting from 2026, with ongoing adjustments to market dynamics expected [1][2]. - The overall goal of the procurement initiative is to optimize resource allocation in the pharmaceutical market while ensuring that patient interests remain a priority [5][9].

Core Viewpoint - The Anhui Provincial Medical Price and Centralized Procurement Center has initiated information collection for eight monoclonal antibody biological agents, indicating the commencement of a national centralized procurement alliance for biological drugs in China [1][5]. Group 1: Market Overview - The eight biological drugs targeted for information collection have a combined market sales exceeding 28 billion yuan [2]. - The original manufacturers of these drugs include AbbVie, Amgen, and Johnson & Johnson, with Roche holding the original drugs for several others [2]. - The competitive landscape is intense, particularly for Bevacizumab, which has 13 approved products in the domestic market, with sales reaching 10.576 billion yuan in 2023 [2]. Group 2: Sales Trends - Sales of Adalimumab in sample hospitals increased from 1.257 billion yuan in 2022 to 1.488 billion yuan in 2024, while sales in urban pharmacies rose from 743 million yuan to 931 million yuan [3]. - The market share of original drugs is declining due to the introduction of biosimilars, with the original Adalimumab being surpassed in market share by a biosimilar in 2023 [3]. Group 3: Impact of Centralized Procurement - Centralized procurement is expected to intensify market competition, leading to a redistribution of market shares, with original drugs facing greater pressure from price competition [3][7]. - The procurement process is anticipated to enhance drug accessibility for patients, potentially lowering prices and ensuring quality [4]. - Companies must improve production capacity and flexibility to meet the demands of centralized procurement [4]. Group 4: Regulatory Environment - The development of biosimilars in China has accelerated, with a record 104 registration applications for therapeutic biological products submitted in 2024 [5]. - The centralized procurement of biological drugs is not prohibited, and the National Healthcare Security Administration has indicated that it will consider various factors before implementing such measures [5][6]. Group 5: Future Industry Trends - The initiation of national centralized procurement for biological drugs may shorten the profit period for low-quality products, prompting companies to focus on enhancing product quality and R&D capabilities [7]. - Companies may also shift their R&D focus towards more valuable innovative fields and increase investments in overseas markets to mitigate the impact of localized procurement [7].