肝脏是"沉默的器官"，而原发性胆汁性胆管炎（PBC）更是一个"缓慢的杀手"。由于早期缺乏特异性症 状，这个罕见病正悄然困扰着越来越多的中国患者。 今年1月，治疗原发性胆汁性胆管炎的创新药Seladelpar在首都医科大学附属北京友谊医院开出中国内地 首张处方。Seladelpar是全球首个获批用于治疗原发性胆汁性胆管炎的强效选择性过氧化物酶体增殖物 激活受体δ（PPARδ）激动剂，适用于与熊去氧胆酸（UDCA）联合治疗UDCA应答不佳的PBC成人患 者，或作为单药用于治疗无法耐受UDCA的PBC成人患者。 作为此次开出Seladelpar国内首张处方的专家，首都医科大学附属北京友谊医院肝病中心首席专家贾继 东教授在接受智通财经（www.thepaper.cn）采访时表示，创新药物既能显著改善PBC患者的生化指标， 也能缓解瘙痒症状，真正实现"一石二鸟"的治疗效果。 授现场开具Seladelpar首张处方。受访者供图 原发性胆汁性胆管炎是"罕见病中的常见病" 原发性胆汁性胆管炎是一种罕见的、易被忽视的慢性自身免疫性肝病，已被纳入我国《第二批罕见病目 录》。这一疾病使人体免疫失调，破坏胆管上皮细胞，从而导致胆汁淤 ...

创新药国内首方落地，为我国原发性胆汁性胆管炎患者带来治疗新选择 中新网北京1月23日电 日前，治疗原发性胆汁性胆管炎(PBC)的罕见病创新药Seladelpar在首都医科大学 附属北京友谊医院肝病中心门诊开出中国内地首张处方，标志着PBC创新疗法惠及中国患者，也体现了 我国罕见病用药可及性的进一步提升，为更多创新药物的引进提供了可供借鉴的范式。 Seladelpar是全球首个获批用于治疗原发性胆汁性胆管炎的强效选择性过氧化物酶体增殖物激活受体 δ(PPARδ)激动剂，适用于与熊去氧胆酸(UDCA)联合治疗UDCA应答不佳的原发性胆汁性胆管炎成人患 者，或作为单药用于治疗无法耐受UDCA的原发性胆汁性胆管炎成人患者。 首都医科大学附属北京友谊医院肝病中心首席专家贾继东教授表示，肝脏是"沉默的器官"，PBC则是一 个"缓慢的杀手"，因为其早期没有明显临床症状。PBC患者不仅亟需控制疾病进展，也迫切希望改善生 活质量。"此次Seladelpar在友谊医院开出首张处方，得益于北京天竺罕见病药物临床急需临时进口政 策，意味着我国PBC患者也可以同步享有全球前沿的治疗方案，有了全新的治疗选择。" 原发性胆汁性胆管炎是一种 ...

北京商报讯(记者王寅浩宋雨盈)1月6日，据首都医科大学附属北京友谊医院消息，该院肝病中心门诊贾 继东主任医师为一位对一线药物应答不佳的原发性胆汁性胆管炎患者开出了有望阻止、延缓疾病进展的 药物Seladelpar。这是中国内地开出的首张处方，也是因北京天竺罕见病药物临床急需临时进口政策获 益的第一位患者。 据了解，Seladelpar是一种强效、高选择性的PPARδ激动剂，能够有效改善胆汁淤积、减轻肝脏炎症和 纤维化，并改善原发性胆汁性胆管炎(PBC)患者的症状和预后，分别于2024年8月、2025年2月于美国及 欧盟上市。借助天竺罕见病保障先行区的政策优势，Seladelpar得以快速获批进入北京友谊医院，为更 多亟需用药的PBC患者提供与国际接轨的前沿治疗。 ...

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]

新京报讯（记者张秀兰）12月22日，原发性胆汁性胆管炎（PBC）药物Seladelpar在海南博鳌乐城国际 医疗旅游先行区获批，适用于与熊去氧胆酸（UDCA）联合治疗对UDCA单药应答不佳的PBC成人患 者，或作为单药用于治疗无法耐受UDCA的PBC成人患者。该药也是全球首个获批用于治疗原发性胆汁 性胆管炎的强效选择性过氧化物酶体增殖物激活受体δ（PPARδ）激动剂。 原发性胆汁性胆管炎已被纳入我国第二批罕见病目录。数据显示，中国PBC患病率为20.5/10万，该病好 发于中老年女性，男女比例约为1:9，近年来男性患者比例也有所增加。谢青指出，随着诊断水平提升 以及临床医生关注度提高，越来越多的PBC患者在肝功能生化指标的常规体检中被发现，但目前我国尚 缺乏基于全国人群的大规模流行病学调查数据，实际发病率可能仍被低估。 长期以来，以熊去氧胆酸（UDCA）为主的一线治疗方案难以满足患者的治疗需求，近40%的患者应答 不佳，这部分患者疾病进展风险高，可以进展为失代偿期肝硬化、肝衰竭，甚至需要肝移植治疗。与此 同时，传统治疗对瘙痒症状改善有限。谢青表示，Seladelpar以其独特的作用机制为原发性胆汁性胆管 炎的 ...

Core Insights - Gilead showcased its commitment to health innovation at the China International Import Expo, presenting two significant products: the long-acting HIV pre-exposure prophylaxis drug Lenacapavir and the new drug for primary biliary cholangitis (PBC), Seladelpar [1][2][3] Product Highlights - Lenacapavir is a long-acting HIV capsid inhibitor that requires administration only twice a year, demonstrating over 99.9% efficacy in preventing HIV in global Phase III clinical trials [2] - Seladelpar is an innovative drug for PBC, a chronic autoimmune liver disease, which was approved in August 2023 and is the first therapy to improve biochemical markers and alleviate itching symptoms in PBC patients [3] Market Impact - Gilead has launched 13 innovative drugs in China, benefiting approximately 1.8 million patients, and aims to deepen its local strategy while enhancing drug accessibility and health equity [4] Collaborative Efforts - Gilead is actively pursuing collaborations to support the "Healthy China 2030" initiative, focusing on improving public health through academic exchanges, enhancing treatment capabilities in rural areas, and expanding innovative partnerships in oncology [6]

Core Insights - Gilead showcased two significant products at the China International Import Expo: the long-acting HIV pre-exposure prophylaxis drug Lenacapavir and the new drug for primary biliary cholangitis (PBC) Seladelpar, emphasizing its commitment to creating a healthier world [1][3] Product Highlights - Lenacapavir is a groundbreaking long-acting HIV capsid inhibitor that requires administration only twice a year, demonstrating over 99.9% efficacy in preventing HIV in global Phase III clinical trials [3][4] - Seladelpar is the first innovative therapy that improves biochemical markers and alleviates itching symptoms for PBC patients, which was included in the National Health Commission's list of rare diseases in 2023 [4] Market Entry and Approval - Lenacapavir received approval for HIV pre-exposure prophylaxis in Hainan Boao Lecheng International Medical Tourism Pilot Zone, almost simultaneously with FDA approval, marking a significant milestone for domestic drug approval processes [4] - Seladelpar was approved in Beijing under the urgent clinical need policy, providing new treatment hope for PBC patients in China [4] R&D Initiatives - Gilead is actively advancing local R&D with 16 ongoing projects in China, including 10 in oncology and 6 in non-oncology, aiming for synchronized global development to benefit Chinese patients [5] Access to Innovative Medicines - Over the past eight years, Gilead has launched 13 global innovative drugs in China, benefiting approximately 1.8 million patients, including notable products for HIV treatment and hepatitis [8] - The company emphasizes collaboration with various sectors to enhance access to innovative medicines and promote health equity [9]

Core Insights - Gilead Sciences' stock price dropped by 3.70% on November 7, attracting significant investor attention, despite the company's strong focus on innovation and development in the healthcare sector [1][2] - At the 8th China International Import Expo, Gilead showcased several innovative drugs, including the long-acting HIV pre-exposure prophylaxis drug Lenacapavir and the new drug Seladelpar for primary biliary cholangitis, highlighting its robust R&D capabilities [1] - Lenacapavir, as a first-in-class drug, requires only two doses per year and has demonstrated over 99.9% efficacy in global Phase III clinical trials, earning multiple international accolades [1] - Seladelpar is the first global innovative therapy that can simultaneously improve biochemical markers and alleviate pruritus symptoms in patients with primary biliary cholangitis, entering the Chinese market rapidly due to expedited drug review processes and favorable policies [1] Market Strategy - Gilead Sciences has made significant breakthroughs in introducing innovative drugs and actively integrates into the Chinese market through localized R&D strategies, conducting 16 clinical studies in China, many of which align with global timelines, particularly in oncology [2] - The company's commitment to the Chinese market is reflected in its plans to sign nearly ten collaborative projects to enhance the accessibility of innovative drugs and promote standardized treatment practices [2] - This proactive strategy not only boosts the company's market competitiveness but also contributes to the achievement of the "Healthy China 2030" goal [2] Long-term Outlook - Despite short-term stock price fluctuations causing market pressure, Gilead Sciences' ongoing innovation and strategic collaborations demonstrate long-term growth potential, instilling confidence in investors [2] - Investors are encouraged to monitor the company's movements in the Chinese market and its deep collaborations with partners to seize future investment opportunities [2] - Diversifying risks and maintaining a long-term perspective may be effective strategies for investing in such innovative companies [2]

Core Insights - Gilead Sciences showcased multiple products at the 8th China International Import Expo, including two Asia debut products: long-acting HIV pre-exposure prophylaxis drug Lenacapavir and a new drug for primary biliary cholangitis (PBC) named Seladelpar [1][2] Group 1: Lenacapavir - Lenacapavir is a globally first-of-its-kind long-acting HIV capsid inhibitor that requires administration only twice a year, demonstrating over 99.9% effectiveness in preventing HIV based on global Phase III clinical studies [1] - Lenacapavir has received several prestigious recognitions, including being ranked first in Science magazine's "Top 10 Scientific Breakthroughs of 2024" and being included in Time magazine's "Best Inventions of 2025" [1] - The drug was approved for HIV pre-exposure prophylaxis in Hainan Boao Lecheng International Medical Tourism Pilot Zone in late June, almost simultaneously with the approval from the U.S. FDA [1] Group 2: Seladelpar - Seladelpar is an innovative drug targeting patients with primary biliary cholangitis, a chronic autoimmune liver disease recognized as a "silent killer" and included in the National Health Commission's list of rare diseases in 2023 [2] - The drug is the first global therapy that can improve biochemical indicators in PBC patients while alleviating itching symptoms, which significantly impact the quality of life [2] - Seladelpar was approved in August under Beijing's clinical urgent import policy, marking a significant step in addressing the needs of PBC patients [2] Group 3: Gilead's Market Presence - Gilead Sciences has launched 13 global innovative drugs in China over the past eight years, benefiting approximately 1.8 million patients [2] - At the expo, Gilead plans to deepen collaborations with various parties to enhance the accessibility of innovative drugs and promote standardized diagnosis and treatment [2]

Core Insights - The China International Import Expo (CIIE) serves as a significant platform for global companies to showcase their innovations and engage with key decision-makers in the Chinese pharmaceutical market [1][2][3] - Gilead Sciences has experienced the benefits of the CIIE, particularly in accelerating the approval and market entry of innovative therapies in China [1][2][3] Policy and Regulatory Environment - China's continuous policy reforms in drug approval processes have created a favorable environment for innovative drugs, enhancing the attractiveness of the Chinese market for multinational pharmaceutical companies [1][2] - The establishment of expedited approval channels for breakthrough therapies and conditional approvals has significantly reduced the time for innovative drugs to enter the Chinese market [2][3] Market Dynamics and Opportunities - The CIIE has become a "policy windfall" and "information hub" for new drugs entering China, allowing companies to effectively communicate with regulatory bodies and enhance product recognition among healthcare professionals and patients [2][3] - Gilead's innovative drugs, such as Lenacapavir and Seladelpar, have demonstrated rapid approval and market entry following their showcases at the CIIE, highlighting the event's role in expediting access to new treatments [3][4][7] Collaboration and Local Partnerships - Gilead is actively pursuing collaborations with local pharmaceutical companies and healthcare platforms to strengthen its supply chain and enhance product accessibility in China [9][10] - The company is also focusing on educational initiatives to reduce stigma around diseases like HIV, thereby fostering a more informed patient population and improving market conditions [9][10] Future Directions - Gilead plans to continue its focus on both treatment and prevention in the HIV space, exploring innovative dosing regimens to improve patient adherence [8][9] - The company is engaged in multiple clinical studies globally, with a significant emphasis on advancing HIV treatment and prevention strategies [8][11] - Gilead's ongoing partnerships and collaborations with local innovators reflect a broader trend of multinational companies seeking to capitalize on China's growing pharmaceutical market [12]