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 ...

Core Insights - The latest national medical insurance drug list will be implemented starting January 1, 2026, adding 114 new drugs, including 50 global innovative drugs, marking a record high in both proportion and quantity [1] - The inclusion of innovative drugs, particularly in the field of cancer treatment, is expected to significantly improve patient access to previously unaffordable medications [1][4] Group 1: Drug Inclusion and Impact - The updated medical insurance list includes a significant number of innovative drugs, especially for breast cancer treatment, which is the most common malignant tumor among women globally [1] - In 2022, over 350,000 new breast cancer cases were reported in China, resulting in approximately 75,000 deaths, highlighting the critical need for effective treatments [1] - The most common molecular subtype of breast cancer, HR+/HER2-, accounts for about 80% of all breast cancer patients [1] Group 2: Treatment Advancements - Advances in screening and treatment have led to a significant improvement in the prognosis of early-stage HR+/HER2- breast cancer, with a five-year survival rate exceeding 90% [3] - Despite treatment advancements, some patients still face disease progression or drug resistance, with about 57% of late-stage HR+/HER2- breast cancer patients in China carrying specific genetic alterations [3] Group 3: Targeted Therapies - The inclusion of targeted therapies in the updated insurance list addresses the clinical treatment gap for late-stage HR+/HER2- breast cancer patients with PIK3CA, AKT1, or PTEN mutations [3] - Notable drugs like Abemaciclib and Ribociclib have expanded their insurance coverage to include all indications for early breast cancer, effective from January 1, 2026 [3][4] Group 4: Shift in Treatment Paradigm - The entry of targeted drugs into the insurance framework signifies a shift from traditional chemotherapy to more precise, individualized treatment approaches for breast cancer [4] - The price reduction for these new drugs can exceed 90%, making previously unaffordable targeted therapies accessible to patients [4]

Key Points Summary of 九州药业 Conference Call Company Overview - 九州药业 is transitioning from generic drugs to innovative drug services, established in 1973 and entered the pharmaceutical sector in 1985. The company began its CDMO (Contract Development and Manufacturing Organization) business in 2008 and went public in 2014 [3][4]. Industry and Business Structure - The company has achieved synergy in domestic and international production through acquisitions and self-built capabilities, including the establishment of an overseas management headquarters in Singapore and R&D platforms in Japan and Germany [2][3]. - The core revenue source is the CDMO business, which generated revenue of 22.91 billion yuan in the first half of 2025, representing a year-on-year growth of 16.27% with a gross margin of 41% [2][3]. Project Pipeline and Clientele - The project pipeline has increased from 378 in 2019 to 1,214 as of the first half of 2025, including 38 commercialized products [2][3]. - Major clients include multinational corporations (MNCs) such as Novartis, Roche, and Pfizer, with Novartis accounting for over 60% of the revenue [2][5]. Financial Performance - In the first half of 2025, the raw material drug business generated revenue of 5.23 billion yuan, maintaining a gross margin of 23% despite industry competition and price declines [2][5]. - The company expects a net profit growth of around 15% over the next three years, with a revenue increase of 4.92% and a net profit increase of 18.51% year-on-year in the first three quarters of the current year [4][5]. New Business Initiatives - The establishment of the Prince Division aims to develop new molecular types such as peptides, conjugated drugs, and small nucleic acids, creating a second growth curve [2][5]. - The company is advancing commercial production capacity for these new initiatives, including three peptide conjugation platforms and capabilities for small nucleic acid research in both China and the U.S. [5]. Market Outlook and Valuation - The company is projected to maintain a net profit growth rate of approximately 15% over the next three years, with a target price of 27 yuan based on a 21x P/E ratio for 2026 [4][5]. - The current stock price reflects a P/E ratio of about 12x for 2027, indicating a favorable investment opportunity considering the rebound in raw material prices and healthy development in small molecule CDMO [5].

Core Viewpoint - The positive results from Roche's oral SERD drug giredestrant in the lidERA trial have significantly boosted the stock of Olema Pharmaceuticals, indicating a de-risking effect for the entire SERD sector and positioning Olema's core asset, Palazestrant, as a leading candidate in breast cancer treatment [1][2][21]. Summary by Sections Roche's Giredestrant Trial Results - Roche announced that its oral SERD drug giredestrant achieved positive results in the lidERA study, marking the second Phase III positive data following the evERA trial at ESMO 2025 [2]. - The lidERA study involved over 4,100 patients and demonstrated statistically significant and clinically meaningful improvements in invasive disease-free survival compared to standard endocrine therapy for early-stage ER+/HER2- breast cancer patients [2][3]. Palazestrant's Mechanism and Development - Palazestrant, Olema's leading candidate, is a novel oral complete estrogen receptor antagonist (CERAN) and selective estrogen receptor degrader (SERD) designed to inhibit estrogen receptor signaling and promote cancer cell degradation [4][5]. - Palazestrant's dual mechanism targets the root of resistance in breast cancer, offering a more effective treatment option compared to traditional SERDs [11][12]. Clinical Data and Future Prospects - Clinical data from ESMO 2025 showed that Palazestrant, in combination with ribociclib, achieved a median progression-free survival (PFS) of 15.5 months across all patients, with specific subgroups showing promising results [14][15][16]. - Palazestrant is currently undergoing two critical Phase III trials, with initial results expected in late 2026, which could pave the way for FDA submission and commercialization [8][9]. Industry Implications - The advancements in oral SERD therapies, particularly with Roche and Olema's developments, signify a paradigm shift in breast cancer treatment, moving towards more convenient and effective options for patients [20]. - The market is recognizing the value of Palazestrant, with potential peak sales estimated between $2.5 billion to $5 billion, indicating a significant opportunity for Olema in the competitive landscape of breast cancer therapies [17][21].

Investment Rating - The report maintains a "Buy" rating for the education sector, particularly highlighting New Oriental's performance and potential recovery in overseas education [4]. Core Insights - New Oriental's revenue for Q1 FY26 reached $1.523 billion, a year-on-year increase of 6.1%, with the education segment (including cultural tourism) generating $1.366 billion, up 8.5% year-on-year [9][10]. - The report indicates that the growth rate of overseas education services has bottomed out, with expectations for recovery in the remaining quarters of FY26 [10]. - The company has announced a shareholder return plan, distributing $190 million in cash dividends and repurchasing up to $300 million in stock [9]. Summary by Sections 1. Overseas Education - The overseas education business has shown signs of recovery, with Q1 FY26 revenue from overseas exam training and consulting at $328 million, a 1% increase year-on-year, although the growth rate has slowed by 19 percentage points compared to the previous year [10]. - New business segments, including K9 non-academic training and learning machines, have maintained high growth, with Q1 revenue increasing by 15% to $403 million [10][11]. - The number of teaching locations increased to 1,347, representing a 24% year-on-year growth [10]. 2. Profitability Improvement - Despite a slowdown in high-margin study abroad services, the profitability of the literacy business has improved, offsetting the decline [11]. - Q1 FY26 Non-GAAP operating profit was $336 million, up 11.3% year-on-year, with an operating profit margin of 22%, expanding by 1 percentage point [11]. 3. Market Performance - The education index fell by 3.7% during the week, underperforming the Hang Seng Index by 4.3 percentage points, with a year-to-date increase of 19.3% [8]. - The report suggests a focus on Chinese education companies, particularly New Oriental, TAL Education, and others, as they show strong enrollment data for the fall semester [14]. 4. Key Company Updates - MGM China reported a record EBITDA of HKD 2.37 billion for Q3 2025, with net revenue of HKD 8.5 billion, a 17% year-on-year increase [16]. - The company continues to focus on high-end gaming and has seen significant growth in its market share [16].

Core Viewpoint - The ESMO annual meeting serves as a significant platform for innovative drug companies to disclose critical clinical data, which often catalyzes stock price increases for the companies involved [1] Group 1: Clinical Data and Market Reaction - XuanZhu Biotech presented promising Phase III clinical data for Pyrotinib at the ESMO conference, demonstrating efficacy and safety advantages [1] - Following the announcement, XuanZhu Biotech's stock surged by 31.87%, reaching a new high, reflecting market confidence in the clinical results and the company's future [1] Group 2: Clinical Trial Insights - The randomized double-blind trial involved 397 patients across 58 centers in China, showing that the combination of Pyrotinib with Letrozole or Anastrozole significantly outperformed the control group in terms of median progression-free survival (mPFS) and objective response rate (ORR) [2] - The mPFS was not reached at a median follow-up of 20.7 months, while the ORR was 63.5%, 21 percentage points higher than the control group [2] - The risk of disease progression or death was reduced by 47%, with a 64% reduction in liver metastasis patients, indicating clear therapeutic advantages [2] Group 3: Comprehensive Treatment Strategy - Pyrotinib's data represents a key component of its "full-cycle treatment strategy," having received approval for two indications earlier this year [3] - The drug is positioned to cover the entire treatment spectrum from late-line to first-line therapy, enhancing its market potential [3] Group 4: Competitive Advantages - Pyrotinib has established a unique competitive barrier through its efficacy, safety, and broad indications, outperforming competitors in key metrics [5] - The ORR of 63.5% for Pyrotinib in first-line treatment is significantly higher than that of similar products, indicating its long-term benefit potential [6] - Pyrotinib is the first and only CDK4/6 inhibitor approved for single-agent use in China, filling a critical gap in the treatment of difficult-to-treat breast cancer patients [6] Group 5: Market Potential and Evidence Support - The commercial potential of Pyrotinib is bolstered by strong clinical evidence and progress in reimbursement approval [8] - The drug has established a credible evidence base through publications in top journals and inclusion in clinical guidelines, enhancing trust among healthcare providers [9] - Pyrotinib has passed the initial review for inclusion in the national medical insurance directory, which is expected to significantly boost its sales [10] Group 6: Future Outlook - The successful approval of Pyrotinib for first-line treatment and its anticipated inclusion in the insurance directory could lead to substantial revenue growth for XuanZhu Biotech [10] - The product's comprehensive indication coverage and superior efficacy data position it well for market penetration, potentially replicating or exceeding the sales growth seen with similar products [10] - Pyrotinib's presence at ESMO symbolizes a shift for domestic innovative drugs from "catching up" to "leading," with its clinical advancements and reimbursement progress warranting ongoing investor attention [12]

Core Insights - The 2025 European Society for Medical Oncology (ESMO) annual meeting will take place in Germany from October 17 to 21, where several significant oral reports from domestic innovative pharmaceutical companies are expected to be disclosed [1] - China National Pharmaceutical Group is set to present key data on its CDK2/4/6 inhibitor, Kumosili, and HER2 dual-target ADC, TQB2102, highlighting its comprehensive coverage in the breast cancer treatment landscape [1] Industry Overview - Breast cancer is the second most common cancer globally, following lung cancer, and has the highest incidence and mortality rates among women [1] - The breast cancer market is highly competitive, particularly in the HR+/HER2- subtype, which accounts for approximately 65%-70% of all breast cancer cases [2] - The global market for CDK4/6 inhibitors is projected to approach $13 billion by 2024, with ongoing growth driven by products from Pfizer, Eli Lilly, and Novartis [2] Company Developments - Kumosili capsules, a new generation CDK2/4/6 inhibitor, are expected to receive approval for second-line treatment of HR+/HER2- locally advanced or metastatic breast cancer by the end of this year [3] - The company is also advancing its clinical trials for Kumosili in first-line and adjuvant settings, aiming for comprehensive coverage across treatment lines [3] - TQB2102, another product from China National Pharmaceutical Group, will present phase Ib clinical data for HER2-positive recurrent/metastatic breast cancer at ESMO [3] Competitive Landscape - The ADC market for HER2-positive breast cancer is evolving, with significant sales from existing products like DS-8201, which reached $3.8 billion last year and is projected to peak at $10 billion [4] - TQB2102 has been optimized in terms of target binding, toxin load, and payload compared to DS-8201, and has been recognized as a breakthrough therapy for multiple indications [4] - The company has a diverse pipeline covering various cancer types, including breast, colorectal, lung, bile duct, and gastric cancers, with several indications entering phase III clinical trials [4]

Core Insights - The global pharmaceutical sales ranking for the first half of 2025 has been released, highlighting the dominance of GLP-1 drugs in the market, with the top three drugs surpassing $10 billion in sales, driving industry growth [2][5] - The competition among pharmaceutical giants is intensifying, particularly in the GLP-1 category, with Novo Nordisk and Eli Lilly leading the charge [5][6] Group 1: GLP-1 Drug Market - GLP-1 drugs are the main drivers of market growth, contributing nearly 30% to the global biopharmaceutical market [3][5] - Semaglutide from Novo Nordisk topped the sales chart with $16.632 billion, while Eli Lilly's tirzepatide followed closely with $14.734 billion, marking a significant year-on-year growth of 121.3% [5][6] - The competitive landscape is shifting, with semaglutide's various formulations (injection, oral, and weight loss) ending Keytruda's long-standing dominance [5][6] Group 2: Emerging Therapies - New therapies such as bispecific antibodies, antibody-drug conjugates (ADC), and fusion proteins are gaining traction, accounting for over 15% of the top-selling drugs [3][10] - ADC drug Enhertu made its debut in the top rankings with $3.9 billion in sales, indicating a growing interest in innovative therapies [3][10] Group 3: CDK4/6 Inhibitors - The CDK4/6 inhibitor market is experiencing a reshuffle, with Eli Lilly's Abemaciclib leading at $2.648 billion, while Novartis' Ribociclib showed a remarkable growth rate of 58.7% [10] - Pfizer's Palbociclib, once a market leader, has seen a decline, dropping to $2.026 billion in sales [10] Group 4: BTK Inhibitors - The BTK inhibitor market is projected to reach approximately $12.5 billion by 2024, with Ibrutinib, Zebrutinib, and Acalabrutinib dominating the market [11][12] - Zebrutinib from BeiGene has entered the global top 50 list with $1.742 billion in sales, marking a significant achievement for Chinese innovation in the pharmaceutical sector [12][13]

Core Insights - The global pharmaceutical sales ranking for the first half of 2025 has been released, highlighting the dominance of GLP-1 drugs in the market, with the top three drugs surpassing $10 billion in sales, driving industry growth [1][6][13] - The competition among global pharmaceutical giants is intensifying, particularly in the metabolic drug sector, with companies like Novo Nordisk and Eli Lilly expected to expand their advantages [2][4] Group 1: GLP-1 Drug Market - GLP-1 drugs are leading the market, with Novo Nordisk's semaglutide family generating $16.632 billion in sales, marking its first position in the ranking [6] - Eli Lilly's tirzepatide follows closely with $14.734 billion in sales, showing a remarkable year-on-year growth of 121.3% [6] - The sales dynamics indicate that semaglutide's injection version holds a 61% market share, while the oral version accounts for 29% [6][7] Group 2: Emerging Therapies - New therapies such as bispecific antibodies, antibody-drug conjugates (ADC), and fusion proteins are gaining traction, accounting for over 15% of the market [1][13] - ADC drug Enhertu has entered the top rankings with $3.9 billion in sales [1] - mRNA vaccines have collectively contributed $9.4 billion, showcasing the impact of innovative therapies in the pharmaceutical landscape [1] Group 3: CDK4/6 Inhibitors - The CDK4/6 inhibitor market is experiencing a reshuffle, with Eli Lilly's Abemaciclib leading at $2.648 billion, while Novartis's Ribociclib has surged with a 58.7% growth [10][11] - Pfizer's Palbociclib has seen a decline, dropping to $2.026 billion, marking a significant shift in market dynamics [11] Group 4: Chinese Pharmaceutical Innovations - The entry of Chinese innovation, specifically BeiGene's Zebrutinib, into the global top 50 with $1.742 billion in sales signifies a breakthrough for domestic drugs [12] - Zebrutinib's success reflects the potential for Chinese pharmaceuticals to transition from thematic investments to performance-driven investments in the global market [12]

Core Insights - The global pharmaceutical sales ranking for the first half of 2025 has been released, highlighting the dominance of GLP-1 drugs, with the top three drugs surpassing $10 billion in sales, marking them as key growth drivers in the industry [1][2][3] - The competition among global pharmaceutical giants is intensifying, particularly in the metabolic drug sector, with companies like Novo Nordisk and Eli Lilly expected to further strengthen their market positions [2][3] GLP-1 Drug Market Dynamics - Novo Nordisk's GLP-1 family, particularly semaglutide, achieved sales of $16.632 billion, securing the top position, while Eli Lilly's tirzepatide followed closely with $14.734 billion, reflecting a year-on-year growth of 121.3% [3][4] - The sales of GLP-1 drugs are reshaping the "king of drugs" competition, with semaglutide and tirzepatide leading the market, and the latter's rapid growth could potentially position it as the top-selling drug for the year [3][4] Emerging Drug Categories - New therapeutic modalities, including bispecific antibodies, antibody-drug conjugates (ADC), and fusion proteins, are gaining traction, accounting for over 15% of the top-selling drugs, with ADC drug Enhertu entering the list with $3.9 billion in sales [1][8] - The mRNA vaccine category also contributed significantly, with three vaccines generating a total of $9.4 billion in sales [1] CDK4/6 Inhibitor Market - The CDK4/6 inhibitor market is experiencing a reshuffle, with Eli Lilly's Abemaciclib leading at $2.648 billion, followed by Novartis' Ribociclib, which saw a remarkable growth rate of 58.7% [6] - Pfizer's Palbociclib, once a market leader, has seen a decline, with sales dropping to $2.026 billion, marking a significant shift in market dynamics [6] BTK Inhibitor Landscape - The global BTK inhibitor market is projected to reach approximately $12.5 billion by 2024, with Ibrutinib, Acalabrutinib, and Zanubrutinib dominating over 97% of the market share [7] - Chinese innovation is making strides, with BeiGene's Zanubrutinib entering the global top 50 list for the first time, achieving sales of $1.742 billion [7][8] Future Outlook - The GLP-1 market is expected to maintain high growth, potentially securing two of the top three positions in the overall sales ranking for 2025, indicating a significant shift in the weight loss drug market [8] - The rise of new molecular entities such as bispecific antibodies and ADCs suggests that the next wave of major innovations will stem from modality innovations, further transforming the pharmaceutical landscape [8]