北海康成创始人、董事长兼首席执行官薛群日前在接受中国证券报记者专访时表示，罕见病创新药市场 是"蓝海中的蓝海"，国内存在大量未被满足的临床需求。凭借二十余年深耕积累，公司已构建起多元产 品矩阵，未来将以"双核驱动"战略深化全球创新布局，借力政策红利与产业协同，让更多罕见病患者享 受到创新药带来的希望。 逾二十载深耕筑根基 薛群介绍，公司的三款上市产品已精准填补多个临床空白：海芮思和迈芮倍两款产品是公司通过专利买 断引进后在国内上市，填补了黏多糖贮积症Ⅱ型（MPS Ⅱ）、阿拉杰里（ALGS）综合征等领域的临床 空白，还刷新了相关适应症的国家诊疗共识与指南；戈芮宁于2025年5月获批上市，是我国首个本土自 主研发生产的针对戈谢病的酶替代1类创新药。 政策赋能破困局 在罕见病创新药研发的高成本赛道上，政策支持成为破解产业困境的关键力量。薛群直言，公司核心产 品戈芮宁的成功上市，既离不开企业直面研发挑战的坚守，更得益于国家生物制品分段生产试点政策的 精准赋能。 戈芮宁的研发历程曾深陷高成本困境。作为国内首个本土自主研发的戈谢病酶替代治疗药物，其采 用"上海外高桥（600648）基地生产原液+江苏无锡GMP厂房制剂罐装 ...

Core Insights - The company, Beihai Kangcheng, has established itself as a global biopharmaceutical firm focused on rare diseases, with three approved products and seven in development [1][2] - The founder, Xue Qun, emphasizes the unmet clinical needs in the rare disease sector as a significant opportunity for growth and innovation [4] Company Development - Beihai Kangcheng was founded in 2012 after Xue Qun's decade-long experience in the rare disease field, initially focusing on oncology due to funding challenges [1] - A pivotal moment for the company was in 2018 when the Chinese government released a rare disease directory, leading to strategic partnerships and significant funding [1][3] Product Portfolio - The company has three marketed products that address clinical gaps in conditions such as MPS II and ALGS, with the first domestically developed enzyme replacement therapy for Gaucher disease, Gorenin, expected to be approved by May 2025 [2][4] Policy Support - Government policies have played a crucial role in alleviating challenges in rare disease drug development, particularly through the segmented production pilot policy that supports innovative biopharmaceuticals [3][5] - Gorenin's successful development benefited from this policy, allowing for a segmented production model that meets clinical needs while navigating regulatory hurdles [3][5] Market Strategy - The company is entering a 3.0 development phase, implementing a "dual-core drive" strategy to solidify its leadership in the domestic rare disease market while expanding internationally [7] - A strategic partnership with Baiyang Pharmaceutical aims to enhance commercialization efforts and leverage each other's strengths in the rare disease sector [7][8] Future Outlook - The company anticipates a significant market expansion for rare disease drugs, particularly with the inclusion of Gorenin in the first commercial insurance innovation drug directory set to take effect in January 2026 [5][6] - Xue Qun envisions a collaborative ecosystem that integrates diagnostics and patient education to improve awareness and treatment of rare diseases [8]

北海康成创始人、董事长兼首席执行官薛群日前在接受中国证券报记者专访时表示，罕见病创新药市场 是"蓝海中的蓝海"，国内存在大量未被满足的临床需求。凭借二十余年深耕积累，公司已构建起多元产 品矩阵，未来将以"双核驱动"战略深化全球创新布局，借力政策红利与产业协同，让更多罕见病患者享 受到创新药带来的希望。 逾二十载深耕筑根基 作为专注罕见疾病领域的全球化生物制药公司，北海康成如今已拥有3个已获批上市产品和7个在研药 物。 在2012年创建北海康成之前，薛群已在罕见病领域深耕十年，其中有四五年时间以拓荒者的身份在国内 推广，彼时国内罕见病领域尚处启蒙阶段。2011年，他任职的健赞公司被收购，薛群表示，出于对更具 挑战性、更高节奏创业环境的追求，他当时主动选择离开。"在我看来，国内存在大量未被满足的临床 需求，是更有作为的创业沃土。"基于这一判断，北海康成应运而生。 创业初期的道路并不平坦。"受融资难题限制，无法直接布局罕见病赛道。"薛群坦言，当时公司的首要 目标是活下来，因此早期管线均围绕肿瘤领域展开。2018年成为公司发展的关键转折点，行业风口迎来 集中爆发：国家五部门联合发布首批罕见病目录，将121种疾病纳入其 ...

薛群介绍，公司的三款上市产品已精准填补多个临床空白：海芮思和迈芮倍两款产品是公司通过专利买 断引进后在国内上市，填补了黏多糖贮积症Ⅱ型（MPS Ⅱ）、阿拉杰里（ALGS）综合征等领域的临床 空白，还刷新了相关适应症的国家诊疗共识与指南；戈芮宁于2025年5月获批上市，是我国首个本土自 主研发生产的针对戈谢病的酶替代1类创新药。 政策赋能破困局 在罕见病创新药研发的高成本赛道上，政策支持成为破解产业困境的关键力量。薛群直言，公司核心产 品戈芮宁的成功上市，既离不开企业直面研发挑战的坚守，更得益于国家生物制品分段生产试点政策的 精准赋能。 戈芮宁的研发历程曾深陷高成本困境。作为国内首个本土自主研发的戈谢病酶替代治疗药物，其采 用"上海外高桥基地生产原液+江苏无锡GMP厂房制剂罐装"的分段生产模式推进临床试验，该模式虽适 配临床需求，但在原有政策框架下，药品生产全流程需集中于同一厂房才能获得GMP认证，给商业化 推进带来巨大阻碍。更关键的是，一期临床患者接受治疗后，经过六个月剂量爬坡后，患者无法中途停 药的治疗连贯性，促使企业必须推动上海外高桥车间完成GMP认证改造。 ● 本报记者 傅苏颖 北海康成创始人、董事长兼 ...

Core Viewpoint - Gilead's drug Seladelpar for primary biliary cholangitis (PBC) has been officially approved in the Boao Lecheng International Medical Tourism Pilot Zone, providing a new treatment option for PBC patients in China [1] Group 1: Drug Approval and Indications - Seladelpar is a potent selective peroxisome proliferator-activated receptor delta (PPARδ) agonist approved for the treatment of PBC [1] - The drug is indicated for adult patients with PBC who have an inadequate response to monotherapy with ursodeoxycholic acid (UDCA) or as a monotherapy for those who cannot tolerate UDCA [1] Group 2: Impact on Rare Disease Treatment - The approval of Seladelpar reflects China's ongoing efforts to enhance the accessibility of innovative drugs for rare diseases and to meet unmet clinical needs [1] - PBC has been included in China's "Second Batch of Rare Disease Catalog," highlighting the importance of addressing rare diseases in the healthcare system [1]

Core Viewpoint - The probability of rare disease patients receiving compensation from commercial insurance is extremely low, akin to the likelihood of an average person contracting a rare disease. The demand for better coverage from commercial insurance is highlighted, especially as basic medical insurance has started to cover some high-cost medications for rare diseases [2][3]. Summary by Sections Rare Disease Patient Insurance Challenges - Rare disease patients face significant challenges in obtaining commercial insurance coverage due to strict underwriting criteria and the exclusion of pre-existing conditions. Even government-backed health insurance options have seen drastic cuts in coverage for pre-existing conditions [2][3][4]. - The National Healthcare Security Administration reported that there are approximately 100 rare disease medications included in the national insurance directory, covering 42 types of rare diseases, with a projected payment of 8.6 billion yuan for these medications in 2024, accounting for 7.7% of total payments during the agreement period [2]. Commercial Insurance Market Dynamics - Approximately half of the 40 innovative drugs submitted for commercial insurance in the second half of the year are for rare diseases. However, insurance companies remain cautious about covering rare disease patients due to the inherent risks associated with genetic and hereditary conditions [3][4]. - The commercial insurance market traditionally excludes congenital diseases and genetic disorders from coverage, which poses a significant barrier for rare disease patients. The risk associated with known hereditary conditions makes it difficult for insurers to price policies effectively [4][5]. Risk Assessment and Pricing Issues - The lack of precise research on the mechanisms of specific rare diseases complicates risk assessment for insurers, leading to higher premiums and conservative coverage terms. Insurers are concerned about the potential for adverse selection if they lower underwriting standards for rare disease patients [6][7]. - The commercial health insurance market is likened to a reservoir that must balance inflow (new premiums) and outflow (claims). The small patient base for rare diseases makes it less attractive for insurers to develop tailored products, as the risk and treatment costs vary significantly across different rare diseases [5][6]. Coverage Gaps and Patient Experiences - While some commercial insurance products do cover specific hereditary diseases, the majority still exclude them, leaving patients with limited options. The coverage for rare diseases is often inadequate, with high deductibles and low reimbursement rates for outpatient treatments [7][10]. - The integration of rare disease medications into commercial insurance products is becoming a selling point, but the actual coverage and reimbursement rates remain unclear. Many patients report significant out-of-pocket expenses despite having insurance [10][11]. Regulatory and Market Developments - The National Healthcare Security Administration's dual-directory negotiations for innovative drugs have led to a higher proportion of rare disease medications being submitted for commercial insurance. However, insurers may lack the motivation to include high-cost rare disease treatments due to the associated risks [10][11]. - The lack of a clear directory for rare disease medications complicates the process for hospitals and insurers, leading to delays and rejections in coverage for necessary treatments [12].