Core Viewpoint - Tian Tan Bio's application for a hemophilia drug was withdrawn after 10 months of acceptance due to the need for additional clinical trial data for children under 12 years old, highlighting regulatory scrutiny in the rare disease market [2][11]. Company Summary - Tian Tan Bio's subsidiary Chengdu Rongsheng Pharmaceutical withdrew the registration application for "Recombinant Human Coagulation Factor VIIa Injection," which had a research investment of 266 million yuan [2][5]. - The drug's indication was aligned with that of imported products, focusing on adult and adolescent patients, but lacked data for younger patients, which is now a regulatory requirement [4][11]. - The company had previously completed a Phase III clinical trial, but the latest announcement did not specify age groups for the patient population [4][5]. Industry Summary - The hemophilia drug market in China is dominated by foreign companies, with over 90% market share, and there is a growing demand for domestic alternatives [12][13]. - The recent approval of a similar drug by Zhengda Tianqing, which does not cover children under 12, indicates a gap in the market that domestic companies are trying to fill [8][11]. - Regulatory bodies are pushing for comprehensive data that includes pediatric populations, reflecting a shift towards more inclusive treatment options for rare diseases [11][12]. - The domestic pharmaceutical industry is accelerating its efforts in rare disease drug development, with 21 rare disease drugs approved in the first half of 2025, indicating a positive trend towards addressing unmet medical needs [12][13].

ANI Pharmaceuticals Conference Call Summary Company Overview - ANI Pharmaceuticals is a growing, profitable biopharmaceutical company focused on rare diseases with three business units: rare disease, generics, and brands [2][3] Core Business Segments - **Rare Disease Business**: - Primary growth driver with two key assets: Purified Cortrophin Gel and ILUVIEN - Purified Cortrophin Gel is used for autoimmune diseases across multiple therapeutic areas [2][3] - ILUVIEN targets retinal diseases, specifically diabetic macular edema and non-infectious uveitis [2] - **Generics Business**: - Strong growth over the past four to five years, supported by R&D and U.S.-based manufacturing [3] Financial Guidance for 2025 - Total company revenues projected at $854 million to $873 million, reflecting a 39%-42% year-over-year growth [4] - Adjusted non-GAAP EBITDA expected to be $221 million to $228 million, indicating a 42%-46% year-over-year growth [4] - Adjusted non-GAAP EPS forecasted at $7.37 to $7.64, also showing a 42%-46% year-over-year growth [4] Growth Strategy - Transition to focus on rare diseases initiated by the current CEO, with significant growth in the rare disease segment [5] - Purified Cortrophin Gel revenue guidance for 2025 is $347 million to $354 million, representing a 75%-78% growth from the previous year [5][6] Market Potential and Demand - Addressable patient population for Purified Cortrophin Gel is substantial, with specific numbers cited for conditions like acute gouty arthritis [14][15] - The company has expanded its sales force to reach more prescribers, resulting in significant growth [17] Competitive Landscape - Both ANI and competitor Mallinckrodt have seen strong growth, validating the market for ACTH therapies [18][24] - The previous peak sales for the category were around $1.2 billion, and current projections suggest potential for surpassing this figure [24][25] Access and Reimbursement - The IRA Part D redesign provided a moderate tailwind for access, lowering out-of-pocket costs for patients [33] - Ongoing efforts to improve access through partnerships with payers and patient assistance programs [26][28] ILUVIEN Update - 2025 is viewed as a reset year for ILUVIEN, with expectations for growth driven by a larger addressable patient population and improved sales force engagement [36][37] - Anticipated revenue for ILUVIEN in 2025 is $73 million to $77 million, with growth expected in subsequent years [37][39] Conclusion - ANI Pharmaceuticals is positioned for significant growth in the rare disease market, with strong financial projections and a strategic focus on expanding its product offerings and market access initiatives [4][25][39]

超罕见病，是罕见病中患病率极低、确诊人数极少的细分群体。据国家卫健委第二届罕见病诊疗与保障 专家委员会委员谢俊明介绍，如果一种疾病的发病人数在1400人以内，即发病率低于百万分之一，那么 这种疾病就是"罕见病中的罕见病"。戈谢病、庞贝病、法布雷病等均在此列，国内确诊患者仅数百人。 过去几年，罕见病药物断供事件偶有发生，但"超罕"家庭面临的困境更为残酷——无药可用甚至成为常 态。 11月14日，一位遗传性周期性共济失调（EA）患者组织相关人士向《每日经济新闻》记者透露，目前 全球尚无专门针对EA的药物获批，国内仅有的几位患者只能超适应证用药。雪上加霜的是，受仿制药 冲击，他们服用的药物——氨吡啶缓释片的美国原研厂商已于去年宣告破产。 已有90余种罕见病药物纳入医保，"超罕"用药却告急 《Neurology》发表的一项临床研究证实，与安慰剂相比，氨吡啶能使EA患者的发作次数减少63%，且 副作用少于传统药物乙酰唑胺。不过，记者并未查到任何制药公司就氨吡啶针对EA的适应证向监管机 构提交上市申请。因此，EA患者长期依赖该药物"超适应证用药"。 但去年，渤健终止了与氨吡啶缓释片原研厂商——Acorda Therape ...

恒瑞医药(01276)发布公告，近日，江苏恒瑞医药股份有限公司(以下简称"公司")... 阵发性睡眠性血红蛋白尿症(PNH)是一种后天获得性溶血性疾病，其特征为 CD55(衰变加速因子，DAF) 和 CD59(反应性溶解膜抑制物，MIRL)缺乏导致补体介导的血管内溶血，主要临床表现为血管内溶血、 潜在造血功能衰竭及血栓形成倾向。因其发病率/患病率低，PNH 已被纳入国家《第一批罕见病目 录》。 HRS-5965 胶囊是一种补体因子 B 抑制剂，可抑制补体介导的血管内外溶血反应，提升血红蛋白 水平。针对本适应症，同靶点药物目前仅有诺华的盐酸伊普可泮胶囊(Fabhalta)在国外获批上市，经查 询 EvaluatePharma 数据库，2024 年该产品全球销售额约为 1.29 亿美元。截至目前，HRS-5965 胶囊相 关项目累计研发投入约 20,994 万元。追加内容 本文作者可以追加内容哦 ! 恒瑞医药(01276)发布公告，近日，江苏恒瑞医药股份有限公司(以下简称"公司")子公司成都盛迪医药有 限公司的 HRS-5965 胶囊被国家药品监督管理局药品审评中心(以下简称"药审中心")拟纳入优先审评品 种公示 ...

Core Viewpoint - The introduction of the innovative drug FILSUVEZ for Epidermolysis Bullosa (EB) patients in China marks a significant breakthrough in addressing the treatment gap for this rare genetic skin disease, facilitated by the "pilot" policy in Boao Lecheng [1][2][3] Group 1: Drug Introduction and Significance - FILSUVEZ is the first drug approved for treating nutritional deficiency type (DEB) and junctional type (JEB) EB wounds in patients aged six months and older, receiving approvals from EMA and FDA in June 2022 and December 2023 respectively [2] - The drug's availability in Boao Lecheng allows patients to access treatment that was previously unavailable in China, addressing a critical need for EB patients [3][4] - The introduction of FILSUVEZ is expected to create a viable pathway for the registration and market entry of innovative drugs in China, as it provides real-world data for future regulatory processes [3][5] Group 2: Collaboration and Policy Impact - The collaboration between the Cassi Group and Boao Lecheng was initiated to expedite the introduction of urgently needed innovative drugs, with both parties sharing a common goal [2][3] - The "pilot" policy in Boao Lecheng allows for the early use of foreign-approved drugs, which helps patients receive treatment sooner while also aiding regulatory assessments based on real-world data [4][5] Group 3: Market Dynamics and Future Strategy - The changing landscape of the Chinese pharmaceutical industry is prompting multinational companies to adapt, focusing on innovation and addressing unmet medical needs [6][8] - The Cassi Group aims to enhance its presence in China, with a growth strategy that includes local market needs and potential future product introductions [6][7] - The company reported a 22% revenue growth in China last year, indicating the market's significance in its global operations, with aspirations to rank among the top five markets globally in the next decade [8]

作为一款具备双重抑制作用机制的创新药物，泽卢克布仑钠能够有效阻断补体介导的神经肌肉接头损 伤。患者在家中仅需5-8秒即可完成皮下注射，不仅减少了往返医院的负担，也帮助他们在疾病管理中 获得更大的便利性与独立性。首都医科大学宣武医院神经内科主任医师笪宇威教授表示，"泽卢克布仑 钠的出现，为全身型重症肌无力治疗带来了重要突破。它不仅赋予患者灵活的用药方式，研究还显示， 该药不仅能在一周内快速改善症状，还能在长期治疗中保持疗效平稳和生活质量持续提升。" 新京报讯(记者张秀兰)10月11日，优时比宣布，国家药监局已批准泽卢克布仑钠(商品名为卓倍可)上市 许可，与常规治疗药物联合用于治疗抗乙酰胆碱受体(AChR)抗体阳性的成人全身型重症肌无力(gMG)患 者。该药也是全球首个且唯一经皮下注射、可自行给药并拥有双重抑制作用的新一代C5补体抑制剂。 全身型重症肌无力是一种罕见、慢性且异质性的自身免疫性疾病，主要表现为神经肌肉接头(NMJ)功能 障碍。据弗若斯特沙利文2025年报告，全球约有120万名全身型重症肌无力患者，其中约22万在中国， 患者可出现复视、眼睑下垂、吞咽/咀嚼/言语困难及危及生命的呼吸肌无力等症状。全身型 ...

Core Insights - UCB's drug, Zilucoplan (brand name: Zhuobeike), has received approval from China's National Medical Products Administration (NMPA) for the treatment of adult generalized myasthenia gravis (GMG) patients who are positive for acetylcholine receptor (AChR) antibodies [1] - Zilucoplan is the first and only subcutaneous self-administered C5 complement inhibitor, which can effectively block complement-mediated neuromuscular junction damage, allowing patients to self-administer the injection at home in just 5-8 seconds [1] - The prevalence of generalized myasthenia gravis in China is approximately 220,000 patients, with AChR antibodies detected in 80%-90% of cases [1] Drug Market Overview - Several drugs for generalized myasthenia gravis have entered the Chinese market recently, including UCB's other drug, Rozanolixizumab (brand name: Yudige), which was approved in March and covers both AChR-positive and MuSK-positive patients [1] - AstraZeneca's long-acting C5 complement inhibitor, Riluzole (brand name: WeiliRui), was approved in April for AChR-positive adult GMG patients [2] - Rongchang Biotech's Taitasip (brand name: Tai'ai) was also approved in May for AChR-positive adult GMG patients [2] - The number of subcutaneous injection options remains limited compared to intravenous formulations, with the approval of Efgartigimod (brand name: WeiliJia) in July last year, which requires a longer administration time of 30-90 seconds [2]

Group 1 - The stock of Beihai Kangcheng-B (01228) has surged over 9%, with a remarkable year-to-date increase of 16 times, currently trading at 2.4 HKD with a transaction volume of 16.13 million HKD [1] - In the first half of the year, Beihai Kangcheng reported revenues of 22.248 million HKD, a decrease of 50.33% year-on-year, while achieving a profit of 59.238 million HKD, compared to a loss of 247 million HKD in the same period last year [1] - The revenue decline is attributed to the company's strategic focus on rare diseases since 2021, with the termination of the distribution agreement for the product He Li An in Taiwan by the end of 2024, leading to a 170,000 HKD or 6.9% decrease in revenue excluding sales in Taiwan [1] Group 2 - In August, Beihai Kangcheng announced a share subscription agreement with Baiyang Pharmaceutical, where Baiyang will invest 100 million HKD to acquire shares in Beihai Kangcheng [2] - The agreement includes a commercial service contract that grants Baiyang's subsidiary promotional rights for specific products of Beihai Kangcheng in mainland China, Hong Kong, and Macau [2] - Beihai Kangcheng's three rare disease products have passed the preliminary review for the commercial insurance innovative drug directory announced by the National Healthcare Security Administration, including the recently approved long-term enzyme replacement product for Gaucher disease patients [2]

北海康成-B(01228)再涨近9%，截至发稿，涨3.47%，报2.09港元，成交额2027.73万港元。 此外，北海康成近日公布，向百洋医药（301015）(301015.SZ)发行7497万股新股，每股作价1.34港元， 较停牌前收报折让19.76%，认购事项所得款项净额约9866.18万港元，拟用于商业化产品的研发；营销 及推广活动；偿还贷款融资；及日常营运。完成后，百洋医药将持有公司股本14.99%，后者已表示其 无意于三年内收购公司任何控股股份。 消息面上，国家医保局8月12日公布商保创新药目录初审名单。北海康成三款罕见病产品均通过了商保 创新药目录的形式审查。包括今年5月获批上市的用于12岁及以上青少年和成人I型和Ⅲ型戈谢病患者的 长期酶替代产品戈芮宁(注射用维拉苷酶β)；用于治疗ALGS/PFIC胆汁淤积性瘙痒的迈芮倍(氯马昔巴特 口服液)；用于治疗黏多糖贮积症II型的海芮思(艾度硫酸酯酶β注射液)。 ...

Group 1 - On August 13, Beihai Kangcheng's stock price surged by 25.75% after announcing a strategic financing deal and an exclusive commercial service agreement with Baiyang Pharmaceutical [1][2] - Baiyang Pharmaceutical will act as the exclusive contract sales organization (CSO) to promote several of Beihai Kangcheng's products in mainland China, Hong Kong, and Macau [1][2] - Beihai Kangcheng's stock has increased over 1400% this year, highlighting strong market interest in rare disease treatments [1][4] Group 2 - The strategic investment amounts to 100 million HKD, with Baiyang Pharmaceutical acquiring approximately 17.65% of Beihai Kangcheng's existing shares [2] - The funds will be used to advance the commercialization of existing products and improve operational efficiency [2] - Beihai Kangcheng currently has three commercialized products targeting rare diseases, including Hai Ruisi, Mai Ruibei, and Wei La Gan En [2][3] Group 3 - The company faces challenges in market access for rare disease drugs, which are often priced high due to the limited patient population [3][6] - Despite being the leading drug for MPS II, only about 10% of diagnosed patients have used Hai Ruisi [6] - The company is exploring international markets and advocating for a specialized rare disease fund to support drug development [7]