AN OPEN LABEL, SINGLE ARM, EXPLORATIVE TRIAL TO EVALUATE EFFICACY AND SAFETY OF BEROTRALSTAT IN PREVENTING ANGIOEDEMA ATTACKS IN SUBJECTS WITH ACQUIRED ANGIOEDEMA DUE TO C1 INHIBITOR DEFICIENCY (AAE)

开始日期2026-09-01

申办/合作机构

Policlinico San Donato SpA

NCT05453968

/ Active, not recruiting临床3期

A Phase 3 Study to Evaluate the Safety and Pharmacokinetics of Berotralstat Prophylaxis in Children With Hereditary Angioedema Who Are 2 to < 12 Years of Age

The purpose of this study is to evaluate the pharmacokinetics (PK), safety and effectiveness of berotralstat to determine the appropriate weight-based dose for pediatric participants 2 to < 12 years of age for prophylactic treatment to prevent attacks of hereditary angioedema (HAE).

开始日期2022-10-25

申办/合作机构

BioCryst Pharmaceuticals, Inc.

NCT04933721

/ Enrolling by invitation临床3期

An Open-label Study to Provide Berotralstat Access to Subjects With Type 1 and 2 Hereditary Angioedema Who Were Previously Enrolled in Berotralstat Studies

This is a phase 3b open-label study providing access to berotralstat for HAE patients who were previously enrolled in berotralstat studies.

开始日期2021-07-23

申办/合作机构

BioCryst Pharmaceuticals, Inc.

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100 项与贝罗司他相关的专利（医药）

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项与贝罗司他相关的文献（医药）

2026-09-02·EXPERT OPINION ON PHARMACOTHERAPY

Evaluating deucrictibant for the treatment of hereditary angioedema

Review

作者: Vu, Mai Thi ; Bhattarai, Bharosha ; Bui, Van ; Aydin, E. Pelinsu ; Luong, Long Hoang ; Craig, Timothy ; Stone, Shane

INTRODUCTION:

Hereditary Angioedema (HAE) is a rare disease affecting approximately 1 in 50,000 people. It results in angioedema of the skin, abdominal tract and upper airway that results in disfiguration, abdominal pain and potential for asphyxia. Early therapies for HAE have significant drug burden due to intravenous administration (C1-inhibitor) or adverse events (androgens); however, newer therapies have reduced injection requirements, and some are oral. Over the past 6 years two oral therapies have been developed for HAE to include berotralstat for long term prophylaxis (LTP) and sebetralstat for on-demand therapy (ODT). Presently, deucrictibant, another oral HAE therapy, is under investigation and appears effective for LTP and ODT.

AREAS COVERED:

This manuscript summarizes the development, pharmacokinetics, pharmacodynamics, and currently available clinical evidence regarding deucrictibant in HAE management.

EXPERT OPINION:

Deucrictibant is a promising therapeutic agent with a well-defined mechanism of action. Published and ongoing clinical studies demonstrate favorable safety, tolerability, and efficacy profiles for both LTP and ODT, offering a convenient oral alternative that may reduce treatment burden and improve patient quality of life.

2026-09-01·The journal of allergy and clinical immunology. Global

Sebetralstat for breakthrough attacks in patients with hereditary angioedema receiving long-term prophylaxis in KONFIDENT-S

Article

作者: Melamed, Isaac ; Farkas, Henriette ; Yea, Christopher M ; Bajcic, Paolo ; Honda, Daisuke ; Ohmura, Kazumasa ; Bouillet, Laurence ; Martinez-Saguer, Inmaculada ; Riedl, Marc A ; Kiani-Alikhan, Sorena ; Cancian, Mauro ; Zanichelli, Andrea ; Geng, Bob ; Bara, Noemi ; Cohn, Danny M ; Wedner, H James ; Kinaciyan, Tamar ; Rehman, Syed ; Ohsawa, Isao ; Craig, Timothy J ; Raasch, Jason P ; Busse, Paula J ; Aygören-Pürsün, Emel ; Kanarek, Henry J ; El-Shanawany, Tariq ; Manning, Michael E ; Lumry, William R ; Smith, Michael D ; Koleilat, Majed ; Li, H Henry ; Audhya, Paul K ; Fukunaga, Atsushi ; Iverson, Matthew ; Bernstein, Jonathan A ; Chuang, Ya-Hsiu ; Tachdjian, Raffi ; Soteres, Daniel F

Background:

Although long-term prophylaxis (LTP) reduces attack frequency in hereditary angioedema, patients may experience breakthrough attacks. Oral sebetralstat demonstrated favorable safety and efficacy compared with placebo in the randomized phase 3 KONFIDENT trial (NCT05259917), including in patients receiving LTP. The safety and effectiveness of sebetralstat in participants receiving LTP are being assessed in the KONFIDENT-S open-label extension study (NCT05505916).

Objective:

This interim analysis of the KONFIDENT-S study evaluated long-term safety and effectiveness of oral sebetralstat 600 mg for attacks of hereditary angioedema with C1-inhibitor deficiency in participants receiving LTP with lanadelumab, berotralstat, or C1 inhibitor.

Methods:

Efficacy end points included times to beginning of symptom relief, reduction in severity, and complete attack resolution.

Results:

As of September 14, 2024, 35 participants receiving LTP experienced a mean ± standard deviation of 1.7 ± 1.5 attacks per month and treated 382 attacks with sebetralstat (1.3 ± 1.1 sebetralstat-treated attacks per month). Median (interquartile range) time from attack recognition to sebetralstat administration was 6 (1-40) minutes; time to beginning of symptom relief was 1.3 (0.8 to 3.8) hours, time to reduction in severity was 4.2 (1.3 to >12) hours, and time to complete attack resolution was 14.8 (4.6 to >24) hours. Effectiveness was similar for participants receiving berotralstat, lanadelumab, or C1 inhibitor. No serious or severe treatment-related treatment-emergent adverse events were reported.

Conclusion:

Sebetralstat was well tolerated and enabled early on-demand treatment of attacks in patients with hereditary angioedema with C1-inhibitor deficiency receiving LTP. Treatment of breakthrough attacks with sebetralstat resulted in rapid symptom relief, reduction in attack severity, and complete attack resolution, regardless of LTP mechanism of action.

2026-08-01·ALLERGY

Switching Long‐Term Prophylaxis to Donidalorsen for Hereditary Angioedema: 1‐Year OASISplus Results

Article

作者: Cohn, Danny M. ; Treadwell, Sabrina ; Newman, Kenneth B. ; Craig, Timothy ; Perego, Francesca ; Yarlas, Aaron ; Bordone, Laura ; Riedl, Marc A. ; Wedner, H. James ; Bernstein, Jonathan A. ; Lumry, William R. ; Smith, Andrew ; Banerji, Aleena ; Lin, Tao ; Manning, Michael E. ; Gierer, Selina ; Jacobs, Joshua S.

ABSTRACT:

Background:

Donidalorsen is an antisense oligonucleotide designed to reduce plasma prekallikrein in patients with hereditary angioedema (HAE). The Switch cohort of the OASISplus study (NCT05392114) evaluated the long‐term safety and efficacy of donidalorsen in patients who switched from a long‐term prophylactic treatment (LTP) to donidalorsen with 1‐year outcomes.

Methods:

This study enrolled patients with HAE aged ≥ 12 years previously on stable doses of an LTP (lanadelumab, berotralstat, C1 inhibitor). Patients directly switched from an LTP to donidalorsen 80 mg administered subcutaneously once every 4 weeks. Endpoints included incidence and severity of treatment‐emergent adverse events (TEAEs; primary endpoint), monthly HAE attack rate, Angioedema Quality of Life (AE‐QoL), and Angioedema Control Test (AECT) measured at baseline and Week 52.

Results:

Sixty‐five patients enrolled, and 54 (83.1%) completed 1 year. Eleven (16.9%) patients discontinued treatment during the first year, and one discontinued treatment after 1 year. Donidalorsen reduced HAE attack rates from baseline on the prior LTP by 67.6% over 52 weeks. Fifty‐six of 64 (87.5%) dosed patients experienced a TEAE; most reported mild to moderate events. The most common treatment‐related TEAEs were injection‐site erythema (10.9%) and injection‐site pruritus (10.9%). One patient had a serious adverse event that was not treatment‐related. AE‐QoL total score improved by 12.2 points (clinically meaningful ≥ 6 points) from baseline to Week 52, and 90.4% of patients had well‐controlled disease (AECT ≥ 10) at Week 52.

Conclusion:

In patients who switched from another LTP, donidalorsen was well tolerated and improved long‐term HAE attack rates, quality of life, and disease control.

327

项与贝罗司他相关的新闻（医药）

2026-10-05

·动脉网

BioCryst宣布成立科学顾问委员会，以推动罕见病产品管线的创新与增长BioCryst Announces Formation of Scientific Advisory Board to Drive Rare Disease Pipeline Innovation and Growth

—Supports BioCryst’s evolution toward a more disciplined R&D model centered on external innovation— ——支持BioCryst向以外部创新为核心的更严谨的研发模式转型——RESEARCH TRIANGLE PARK, N.C., Oct. 05, 2026 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced the formation of a Scientific Advisory Board (SAB) comprising seven accomplished scientific and industry leaders with deep expertise across rare disease research and drug development. 北卡罗来纳州研究三角园，2026年10月5日（环球社电）——BioCryst Pharmaceuticals, Inc.（纳斯达克股票代码：BCRX）今日宣布成立科学顾问委员会（SAB），该委员会由七位杰出的科学界和业界领袖组成，他们在罕见病研究和药物开发领域拥有深厚的专业知识。The SAB will provide independent scientific and strategic guidance as the Company advances its research and development strategy and builds a high value novel drug pipeline.. 随着公司推进其研发战略并构建高价值的创新药物管线，科学顾问委员会（SAB）将提供独立的科学与战略指导。“The formation of BioCryst’s Scientific Advisory Board marks an important step in the evolution of our new R&D model,” said Dr. Sandeep Menon, BioCryst’s Chief Research and Development Officer. “External innovation is increasingly central to how we build our pipeline, and guidance from leading scientists and physicians will sharpen our thinking and strengthen the diligence behind each program we evaluate and advance. “BioCryst科学顾问委员会的成立，标志着我们新研发模式演进中的重要一步。”BioCryst首席研发官Sandeep Menon博士表示，“外部创新在我们构建产品管线的过程中正日益占据核心地位，而来自顶尖科学家和临床医生的指导将帮助我们厘清思路，并加强对每个我们评估和推进项目的尽职调查力度。”The Scientific Advisory Board will work closely with me and our Chief Scientific Officer, Dr. David Jenkins, as we shape and execute BioCryst’s R&D strategy.”. “科学顾问委员会将与我及我们的首席科学官戴维·詹金斯博士紧密合作，共同制定并执行BioCryst的研发战略。”“Building on our success in developing and commercializing medicines for hereditary angioedema, we are bringing the same rigor to the next generation of BioCryst medicines,” said Charlie Gayer, Chief Executive Officer. “With the expertise of these highly experienced scientific advisors, BioCryst will be better positioned to evaluate emerging opportunities and translate their potential into meaningful benefits for patients.”. “基于我们在遗传性血管性水肿药物的开发和商业化方面取得的成功，我们将以同样的严谨态度推进BioCryst下一代药物的研发，”首席执行官Charlie Gayer表示。“凭借这些经验丰富的科学顾问的专业知识，BioCryst将能更好地评估新兴机遇，并将其潜力转化为为患者带来切实获益的成果。”The SAB will provide strategic guidance across BioCryst’s research and development activities, including pipeline expansion, external innovation opportunities, translational science and clinical development. The board will also offer insights on regulatory strategy and help strengthen connections with leading scientific, medical and patient communities worldwide.. 科学咨询委员会（SAB）将为BioCryst的研发活动提供战略指导，包括管线拓展、外部创新机会、转化科学以及临床开发。该委员会还将就监管策略提供见解，并帮助加强公司与全球领先的科学界、医学界及患者群体之间的联系。'Few companies have demonstrated BioCryst’s ability to translate rigorous science into rare disease medicines that reach patients, and that track record is what makes the Company’s evolution so compelling,' said Dr. Anthony Manning, Chair of the Scientific Advisory Board. 'The formation of this Board is an important step for BioCryst, and it is a privilege to chair a group with this depth of experience across rare disease drug discovery, translational science and clinical development. “很少有公司能像BioCryst那样，展现出将严谨的科学转化为惠及患者的罕见病药物的能力，而这一记录正是使该公司的发展如此引人注目的原因。”科学顾问委员会主席Anthony Manning博士表示。“该委员会的成立是BioCryst的重要一步，能够领导一个在罕见病药物发现、转化科学和临床开发方面拥有如此深厚经验的团队，我深感荣幸。”I am confident the Company’s decisions about which science to pursue and how to advance it will benefit from the collective judgment of its members.'. 我相信，公司在决定 pursue 哪些科学领域以及如何推进这些领域时，将从其成员的集体判断中受益。Scientific Advisory Board Members: 科学顾问委员会成员：Anthony (Tony) Manning, PhD, Chair, 安东尼（托尼）曼宁博士，主席，is Principal at Manning Bio Worldwide, LLC, advising biotechnology companies and academic institutions following a 30-year career in pharmaceutical drug discovery and development. As Chief Scientific Officer at Momenta Pharmaceuticals (now part of Johnson & Johnson), he built a pipeline of first-in-class therapeutics for rare autoantibody-driven diseases, including nipocalimab (IMAAVY), now FDA-approved for generalized myasthenia gravis and warm autoimmune hemolytic anemia, with additional indications in development. 现任 Manning Bio Worldwide, LLC 负责人，在拥有三十年药物发现与开发职业经历后，为生物技术公司和学术机构提供咨询。此前，他在 Momenta Pharmaceuticals（现为强生公司旗下子公司）担任首席科学官期间，构建了针对罕见自身抗体驱动疾病的首创疗法产品管线，其中包括已获美国食品药品监督管理局（FDA）批准用于治疗全身型重症肌无力和温性自身免疫性溶血性贫血的 nipocalimab（IMAAVY），另有多个适应症正在开发中。Earlier, he led drug discovery efforts at Roche Pharmaceuticals and Biogen Idec, contributing to the approvals of Actemra and Rituxan, and to the development of litifilimab, now in Phase 3 for lupus.. 此前，他在罗氏制药和Biogen Idec领导药物研发工作，为Actemra和Rituxan的获批以及litifilimab的研发做出了贡献，后者目前正处于治疗狼疮的三期临床试验阶段。Dr. Christine Bulawa, PhD, 克里斯汀·布拉瓦博士，哲学博士，received a PhD from the University of Wisconsin-Madison and completed postdoctoral studies at Massachusetts Institute of Technology (MIT). As part of her 20+ years in rare disease drug discovery, she was the first employee of FoldRx Pharmaceuticals and instrumental in the preclinical development of tafamidis, the first pharmacological treatment for transthyretin amyloidosis. 在威斯康星大学麦迪逊分校获得博士学位，并在麻省理工学院（MIT）完成博士后研究。在罕见病药物发现领域拥有二十余年经验，她是FoldRx Pharmaceuticals的首位员工，并在转甲状腺素蛋白淀粉样变性症的首个药物治疗药物——塔法米迪斯的临床前开发中发挥了关键作用。As a senior leader in Pfizer’s Rare Disease Research Unit, Dr. Bulawa built a pipeline of innovativeprograms based onrigorously validated targets,spanning portfolio entry to clinical development, witha focus on small molecule and genetic therapies to treat degenerative neurological, neuromuscular and protein aggregation diseases.. 作为辉瑞罕见病研究部门的高级领导，Bulawa博士构建了一条基于严格验证靶点的创新项目管线，涵盖从项目立项到临床开发的全过程，重点聚焦于治疗退行性神经疾病、神经肌肉疾病和蛋白质聚集性疾病的小分子药物和基因疗法。Dr. Eric A. Hughes, MD, PhD, 埃里克·A·休斯博士，医学博士，哲学博士，serves as Executive Vice President, Global Research & Development and Chief Medical Officer at Teva Pharmaceuticals. A physician-scientist who earned his MD and PhD from Yale University, Dr. Hughes brings nearly 20 years of biopharmaceutical industry experience and is helping advance Teva’s transformation into a leading innovative biopharmaceutical company, enabled by a world-class generics business. Prior to Teva, he served in leadership roles at Vertex, Novartis and Bristol Myers Squibb.. 在梯瓦制药公司（Teva Pharmaceuticals）担任执行副总裁，主管全球研发并兼任首席医疗官。休斯博士是一位医师科学家，拥有耶鲁大学医学博士和哲学博士学位，他在生物制药行业拥有近20年的经验，正助力梯瓦转型为一家由世界一流仿制药业务支撑的领先创新型生物制药公司。在加入梯瓦之前，他曾在福泰制药（Vertex）、诺华（Novartis）和百时美施贵宝（Bristol Myers Squibb）担任领导职务。Dr. Stephanie Noviello, MD, MPH, 斯蒂芬妮·诺维洛博士，医学博士，公共卫生硕士is Chief Medical Officer at ILiAD Biotechnologies, Inc. She is a clinical development leader with extensive experience across infectious diseases, virology, immunology, rare diseases and skeletal diseases at Novartis, Motif BioSciences, Bristol-Myers Squibb and Schering-Plough Research Institute. 担任ILiAD Biotechnologies, Inc.的首席医学官。她是一位临床开发领导者，在诺华、Motif BioSciences、百时美施贵宝和先灵葆雅研究所拥有传染病、病毒学、免疫学、罕见病和骨骼疾病领域的丰富经验。Dr. Shashi Ramaiah, DVM, PhD, DACVP, DABT, 沙希·拉马亚博士，兽医博士，哲学博士，美国兽医病理学家学会认证专家，美国毒理学委员会认证专家is a board-certified Veterinary Pathologist and Toxicologist who spent 18 years at Pfizer, most recently as Vice President and Global Head of Discovery, Investigative and Translational Sciences. He is Founder and CEO of SPECCRO, a drug development advisory firm that helps biotechnology companies and investors de-risk programs and portfolios, drawing on more than 40 senior industry experts and extensive engagement with national and international scientific consortia.. 是一位拥有董事会认证的兽医病理学家和毒理学家，在辉瑞公司工作了18年，最近担任副总裁兼全球发现、调查和转化科学负责人。他是SPECCRO的创始人和首席执行官，这是一家药物开发咨询公司，帮助生物技术公司和投资者降低项目和投资组合的风险，依托40多位资深行业专家以及与国内外科学联盟的广泛合作。Dr. Art Taveras, PhD, 阿特·塔韦拉斯博士，哲学博士，is a seasoned biopharmaceutical executive with over 35 years of industry experience, specializing in leading R&D divisions across major public pharmaceutical corporations and biotech firms includingBiogen Idec, Transform Therapeutics, X4 Pharmaceuticals, CoMET Therapeutics, ShangPharma ChemPartner, Alantos Pharmaceuticals and Schering-Plough. 是一位经验丰富的生物制药高管，拥有超过35年的行业经验，专长于领导多家大型上市制药公司和生物技术企业的研发部门，包括Biogen Idec、Transform Therapeutics、X4 Pharmaceuticals、CoMET Therapeutics、尚药化学伙伴（ShangPharma ChemPartner）、Alantos Pharmaceuticals以及先灵葆雅（Schering-Plough）。His strategic expertise as President, CEO, CSO and R&D Leader has advanced robust pipelines and key global R&D partnerships, culminating in over 175 patent application filings, 150 publications, 60 clinical trials, 6 regulatory agency approvals and 5 therapeutic platform technologies across commercial and clinical-stage assets for diseases in neurology, oncology, immunology, metabolism and rare disease.. 他在担任总裁、首席执行官、首席战略官及研发负责人期间，凭借卓越的战略专长，推动了强大的产品管线建设和关键全球研发合作伙伴关系的发展，最终成果包括提交175余项专利申请、发表150篇论文、开展60项临床试验、获得6项监管机构批准，并开发出5个治疗平台技术，涵盖针对神经学、肿瘤学、免疫学、代谢疾病和罕见病的商业化和临床阶段资产。Dr. Ajay Yekkirala, PhD, 阿贾伊·耶基拉拉博士，哲学博士，is an experienced serial entrepreneur who co-founded Superluminal Medicines at RA Capital and built a strong investor syndicate including NVIDIA, Eli Lilly, Insight Partners, Catalio, BVF and Perceptive Advisors. As Chief Scientific Officer, he directs the AI/ML platform build and all pipeline R&D.Prior to Superluminal,Dr. 是一位经验丰富的连续创业者，他在 RA Capital 共同创立了 Superluminal Medicines，并组建了一个强大的投资者联盟，包括英伟达（NVIDIA）、礼来公司（Eli Lilly）、Insight Partners、Catalio、BVF 和 Perceptive Advisors。作为首席科学官，他负责指导人工智能/机器学习平台的建设以及所有研发管线的研究工作。在加入 Superluminal 之前，博士Yekkiralafounded Blue Therapeutics to develop non-addictive painkillers. He received his PhD from the University of Minnesota and completed postdoctoral training at Harvard Medical School. He has authored several papers published in high-impact, peer-reviewed journals andadvised various committees at the NIH, including EPPIC-NET at NINDS and Medication Development Research Committee at NIDA.. 耶基拉拉创立了Blue Therapeutics，致力于开发非成瘾性止痛药。他于明尼苏达大学获得博士学位，并在哈佛医学院完成博士后培训。他在高影响力的同行评审期刊上发表了多篇论文，并担任美国国立卫生研究院（NIH）多个委员会的顾问，包括国家神经疾病与中风研究所（NINDS）的EPPIC-NET以及国家药物滥用研究所（NIDA）的药物开发研究委员会。About BioCryst Pharmaceuticals 关于 BioCryst PharmaceuticalsBioCryst is a global biotechnology company focused on developing and commercializing medicines for hereditary angioedema (HAE) and other rare diseases, driven by its deep commitment to improving the lives of people living with these conditions. BioCryst has commercialized ORLADEYO® (berotralstat), the first oral, once-daily plasma kallikrein inhibitor and is advancing a pipeline of potential first-in-class or best-in-class therapeutics for rare diseases. BioCryst 是一家全球性生物技术公司，专注于开发和商业化用于遗传性血管性水肿（HAE）及其他罕见病的药物，其动力源于改善这些患者生活的深切承诺。BioCryst 已实现 ORLADEYO®（berotralstat）的商业化，这是首款口服、每日一次的血浆激肽释放酶抑制剂，并正在推进一系列针对罕见病的潜在首创或同类最佳疗法的研发管线。For more information, please visit. 欲了解更多信息，请访问。www.biocryst.com www.biocryst.comor follow us on 或在以下平台关注我们LinkedIn 领英. 。Forward-Looking Statements 前瞻性陈述This press release contains forward-looking statements, including statements regarding future results, performance, or achievements, expectations regarding BioCryst’s strategic shift to prioritize external innovation, expectations regarding pipeline development, the anticipated role and potential benefits of BioCryst’s Scientific Advisory Board, and statements related to BioCryst’s future operations, prospects and pipeline programs. 本新闻稿包含前瞻性陈述，包括有关未来业绩、表现或成就的陈述；关于 BioCryst 战略转向以优先推动外部创新的预期；关于管线开发的预期；关于 BioCryst 科学顾问委员会的预期作用及潜在益处；以及有关 BioCryst 未来运营、前景和管线项目的陈述。These statements involve known and unknown risks, uncertainties and other factors which may cause BioCryst’s actual results, performance, or achievements to be materially different from any future results, performance, or achievements expressed or implied by the forward-looking statements. These statements reflect our current views with respect to future events and are based on assumptions and are subject to risks and uncertainties. 这些陈述涉及已知和未知的风险、不确定性和其他因素，可能导致 BioCryst 的实际结果、业绩或成就与前瞻性陈述中明示或暗示的任何未来结果、业绩或成就存在重大差异。这些陈述反映了我们对未来事件的当前看法，基于某些假设，并受风险和不确定性的影响。Given these uncertainties, you should not place undue reliance on these forward-looking statements. Some of the factors that could affect the forward-looking statements contained herein include: BioCryst’s ability to successfully progress its pipeline development plans; guidance and recommendations provided by the Scientific Advisory Board may not improve BioCryst’s evaluation or advancement of potential programs or result in the anticipated benefits; the commercial viability of BioCryst’s future rare disease therapies; ongoing and future development of product candidates may take longer than expected and may not have positive results; product candidates, if approved, may not achieve market acceptance; the results of BioCryst’s partnerships with third parties may not meet BioCryst’s current expectations; and BioCryst’s ability to realize the contemplated benefits of its new R&D strategy and model and successfully. 鉴于这些不确定性，您不应过度依赖这些前瞻性陈述。可能影响本文所载前瞻性陈述的因素包括：BioCryst 成功推进其管线开发计划的能力；科学顾问委员会提供的指导和建议可能无法改善 BioCryst 对潜在项目的评估或推进，也可能无法产生预期的效益；BioCryst 未来罕见病疗法的商业可行性；在研产品的持续及未来开发可能耗时超出预期，且可能无法取得积极成果；在研产品即使获得批准，也可能无法获得市场认可；BioCryst 与第三方合作的结果可能无法满足其当前预期；以及 BioCryst 实现其新研发战略和模式所预期的效益并取得成功的能力。Contact: 联系方式：Investors: 投资者：investorrelations@biocryst.com investorrelations@biocryst.comMedia: 媒体：media@biocryst.com media@biocryst.com

2026-10-05

·BioSpace

BioCryst Announces Formation of Scientific Advisory Board to Drive Rare Disease Pipeline Innovation and Growth

—Supports BioCryst’s evolution toward a more disciplined R&D model centered on external innovation— RESEARCH TRIANGLE PARK, N.C., Oct. 05, 2026 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced the formation of a Scientific Advisory Board (SAB) comprising seven accomplished scientific and industry leaders with deep expertise across rare disease research and drug development. The SAB will provide independent scientific and strategic guidance as the Company advances its research and development strategy and builds a high value novel drug pipeline. “The formation of BioCryst’s Scientific Advisory Board marks an important step in the evolution of our new R&D model,” said Dr. Sandeep Menon, BioCryst’s Chief Research and Development Officer. “External innovation is increasingly central to how we build our pipeline, and guidance from leading scientists and physicians will sharpen our thinking and strengthen the diligence behind each program we evaluate and advance. The Scientific Advisory Board will work closely with me and our Chief Scientific Officer, Dr. David Jenkins, as we shape and execute BioCryst’s R&D strategy.” “Building on our success in developing and commercializing medicines for hereditary angioedema, we are bringing the same rigor to the next generation of BioCryst medicines,” said Charlie Gayer, Chief Executive Officer. “With the expertise of these highly experienced scientific advisors, BioCryst will be better positioned to evaluate emerging opportunities and translate their potential into meaningful benefits for patients.” The SAB will provide strategic guidance across BioCryst’s research and development activities, including pipeline expansion, external innovation opportunities, translational science and clinical development. The board will also offer insights on regulatory strategy and help strengthen connections with leading scientific, medical and patient communities worldwide. "Few companies have demonstrated BioCryst’s ability to translate rigorous science into rare disease medicines that reach patients, and that track record is what makes the Company’s evolution so compelling," said Dr. Anthony Manning, Chair of the Scientific Advisory Board. "The formation of this Board is an important step for BioCryst, and it is a privilege to chair a group with this depth of experience across rare disease drug discovery, translational science and clinical development. I am confident the Company’s decisions about which science to pursue and how to advance it will benefit from the collective judgment of its members." Scientific Advisory Board Members: Anthony (Tony) Manning, PhD, Chair, is Principal at Manning Bio Worldwide, LLC, advising biotechnology companies and academic institutions following a 30-year career in pharmaceutical drug discovery and development. As Chief Scientific Officer at Momenta Pharmaceuticals (now part of Johnson & Johnson), he built a pipeline of first-in-class therapeutics for rare autoantibody-driven diseases, including nipocalimab (IMAAVY), now FDA-approved for generalized myasthenia gravis and warm autoimmune hemolytic anemia, with additional indications in development. Earlier, he led drug discovery efforts at Roche Pharmaceuticals and Biogen Idec, contributing to the approvals of Actemra and Rituxan, and to the development of litifilimab, now in Phase 3 for lupus. Dr. Christine Bulawa, PhD, received a PhD from the University of Wisconsin-Madison and completed postdoctoral studies at Massachusetts Institute of Technology (MIT). As part of her 20+ years in rare disease drug discovery, she was the first employee of FoldRx Pharmaceuticals and instrumental in the preclinical development of tafamidis, the first pharmacological treatment for transthyretin amyloidosis. As a senior leader in Pfizer’s Rare Disease Research Unit, Dr. Bulawa built a pipeline of innovative programs based on rigorously validated targets, spanning portfolio entry to clinical development, with a focus on small molecule and genetic therapies to treat degenerative neurological, neuromuscular and protein aggregation diseases. Dr. Eric A. Hughes, MD, PhD, serves as Executive Vice President, Global Research & Development and Chief Medical Officer at Teva Pharmaceuticals. A physician-scientist who earned his MD and PhD from Yale University, Dr. Hughes brings nearly 20 years of biopharmaceutical industry experience and is helping advance Teva’s transformation into a leading innovative biopharmaceutical company, enabled by a world-class generics business. Prior to Teva, he served in leadership roles at Vertex, Novartis and Bristol Myers Squibb. Dr. Stephanie Noviello, MD, MPH, is Chief Medical Officer at ILiAD Biotechnologies, Inc. She is a clinical development leader with extensive experience across infectious diseases, virology, immunology, rare diseases and skeletal diseases at Novartis, Motif BioSciences, Bristol-Myers Squibb and Schering-Plough Research Institute. Dr. Shashi Ramaiah, DVM, PhD, DACVP, DABT, is a board-certified Veterinary Pathologist and Toxicologist who spent 18 years at Pfizer, most recently as Vice President and Global Head of Discovery, Investigative and Translational Sciences. He is Founder and CEO of SPECCRO, a drug development advisory firm that helps biotechnology companies and investors de-risk programs and portfolios, drawing on more than 40 senior industry experts and extensive engagement with national and international scientific consortia. Dr. Art Taveras, PhD, is a seasoned biopharmaceutical executive with over 35 years of industry experience, specializing in leading R&D divisions across major public pharmaceutical corporations and biotech firms including Biogen Idec, Transform Therapeutics, X4 Pharmaceuticals, CoMET Therapeutics, ShangPharma ChemPartner, Alantos Pharmaceuticals and Schering-Plough. His strategic expertise as President, CEO, CSO and R&D Leader has advanced robust pipelines and key global R&D partnerships, culminating in over 175 patent application filings, 150 publications, 60 clinical trials, 6 regulatory agency approvals and 5 therapeutic platform technologies across commercial and clinical-stage assets for diseases in neurology, oncology, immunology, metabolism and rare disease. Dr. Ajay Yekkirala, PhD, is an experienced serial entrepreneur who co-founded Superluminal Medicines at RA Capital and built a strong investor syndicate including NVIDIA, Eli Lilly, Insight Partners, Catalio, BVF and Perceptive Advisors. As Chief Scientific Officer, he directs the AI/ML platform build and all pipeline R&D. Prior to Superluminal, Dr. Yekkirala founded Blue Therapeutics to develop non-addictive painkillers. He received his PhD from the University of Minnesota and completed postdoctoral training at Harvard Medical School. He has authored several papers published in high-impact, peer-reviewed journals and advised various committees at the NIH, including EPPIC-NET at NINDS and Medication Development Research Committee at NIDA. About BioCryst Pharmaceuticals BioCryst is a global biotechnology company focused on developing and commercializing medicines for hereditary angioedema (HAE) and other rare diseases, driven by its deep commitment to improving the lives of people living with these conditions. BioCryst has commercialized ORLADEYO® (berotralstat), the first oral, once-daily plasma kallikrein inhibitor and is advancing a pipeline of potential first-in-class or best-in-class therapeutics for rare diseases. For more information, please visit or follow us on LinkedIn . Forward-Looking Statements This press release contains forward-looking statements, including statements regarding future results, performance, or achievements, expectations regarding BioCryst’s strategic shift to prioritize external innovation, expectations regarding pipeline development, the anticipated role and potential benefits of BioCryst’s Scientific Advisory Board, and statements related to BioCryst’s future operations, prospects and pipeline programs. These statements involve known and unknown risks, uncertainties and other factors which may cause BioCryst’s actual results, performance, or achievements to be materially different from any future results, performance, or achievements expressed or implied by the forward-looking statements. These statements reflect our current views with respect to future events and are based on assumptions and are subject to risks and uncertainties. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Some of the factors that could affect the forward-looking statements contained herein include: BioCryst’s ability to successfully progress its pipeline development plans; guidance and recommendations provided by the Scientific Advisory Board may not improve BioCryst’s evaluation or advancement of potential programs or result in the anticipated benefits; the commercial viability of BioCryst’s future rare disease therapies; ongoing and future development of product candidates may take longer than expected and may not have positive results; product candidates, if approved, may not achieve market acceptance; the results of BioCryst’s partnerships with third parties may not meet BioCryst’s current expectations; and BioCryst’s ability to realize the contemplated benefits of its new R&D strategy and model and successfully evaluate and execute external opportunities. This list is not exclusive. To see a more comprehensive list of risks, please refer to the documents BioCryst files periodically with the Securities and Exchange Commission, specifically BioCryst’s most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K, which identify important factors that could cause actual results to differ materially from those contained in BioCryst’s projections and forward-looking statements. Contact: Investors: investorrelations@biocryst.com Media: media@biocryst.com

上市批准临床3期高管变更

2026-10-02

·Rainy药

Takeda-武田-消化道与炎症

本文介绍下武田制药（简称Takeda），主要为简述公司目前情况，历史沿革以及治疗领域；后续会陆续整理其他药企（国内和MNC或者biotech），仅为学习记录，欢迎关注以及分享；文末二维码放有可编辑的药企PPT模板。武田药品工业株式会社（Takeda Pharmaceutical Company Limited，东京证券交易所代码：4502；纽约证券交易所代码：TAK，每份ADS对应0.5股普通股）成立于1781年，是日本历史最悠久、规模最大的制药企业，总本社位于大阪，本社设于东京日本桥本町，在约80个国家和地区设有业务，主要研发中心分布于日本与美国。公司定位为研发驱动型全球生物制药企业，围绕消化道与炎症、神经科学、肿瘤三大核心治疗领域，并通过血浆衍生疗法与疫苗业务形成补充；罕见病则是其重要的商业化板块。 FY2025各业务板块收入为：消化道与炎症14075亿日元、血浆衍生疗法10575亿日元、罕见病7627亿日元、肿瘤5801亿日元、神经科学4143亿日元、疫苗596亿日元。截至2026年9月中旬，公司市值约9.2万亿日元（约580亿美元）。下面是各个领域的介绍：消化道与炎症消化道与炎症是武田收入占比最高的板块，FY2025收入14075亿日元，同比增长3.7%，核心资产为炎症性肠病生物制剂ENTYVIO，同时依托酸相关疾病、短肠综合征等产品线；下一阶段的增长重心是口服TYK2抑制剂zasocitinib。 ENTYVIO（维得利珠单抗，vedolizumab）是公司目前最大的单一产品，靶点为α4β7整合素，已获批用于溃疡性结肠炎和克罗恩病，并有静脉注射与皮下注射笔两种剂型。 FY2025全球销售额9580亿日元，同比增长4.8%，其中美国6237亿日元、欧洲与加拿大2567亿日元；FY2026 Q1销售额2683亿日元，同比增长15.4%。该药源自武田2008年以约88亿美元收购Millennium Pharmaceuticals所获得的资产，由公司主导开发上市。2026年6月至7月，公司在美国、欧洲和日本提交了溃疡性结肠炎与克罗恩病的儿科适应症申请。当前主要竞争品包括艾伯维的Skyrizi（risankizumab，IL-23）、Rinvoq（upadacitinib，JAK1），以及强生的Stelara（ustekinumab，IL-12/23）和Tremfya（guselkumab，IL-23），均用于溃疡性结肠炎和克罗恩病。 TAKECAB/VOCINTI（沃诺拉赞，vonoprazan）为钾离子竞争性酸阻滞剂（P-CAB），用于胃食管反流病、消化性溃疡及幽门螺杆菌根除等酸相关疾病。FY2025销售额1437亿日元，同比增长9.9%，增长主要来自中国和日本；FY2026 Q1未单独披露。该药由武田内部研发。主要竞争品为各类质子泵抑制剂，以及大冢制药的Jaqbo（tegoprazan，同为P-CAB机制，用于酸相关疾病）。 GATTEX/REVESTIVE（替度鲁肽，teduglutide）为GLP-2类似物，用于短肠综合征。FY2026 Q1销售额414亿日元，同比增长19.0%；FY2025全年未单独披露。该药来自2019年对Shire的收购。主要竞争品包括Zealand Pharma与勃林格殷格翰合作开发的glepaglutide等长效GLP-2类似物（用于短肠综合征）。 EOHILIA（布地奈德口服混悬液）用于嗜酸性粒细胞性食管炎，FY2025销售额88亿日元，同比增长61.0%。该药由公司内部开发，2024年2月获FDA批准。主要竞争品为赛诺菲与再生元的Dupixent（dupilumab，IL-4Rα），已获批用于嗜酸性粒细胞性食管炎。 zasocitinib（TAK-279）是武田在该领域最重要的在研资产，为高选择性口服TYK2变构抑制剂，目前处于III期，尚未获批。该项目来自公司2022年12月宣布、2023年2月完成的Nimbus Lakshmi收购，首付款40亿美元。当前主要同类项目包括百时美施贵宝的Sotyktu（deucravacitinib，TYK2）、Alumis的ESK-001（TYK2），以及强生与Protagonist的Icotyde（icotrokinra，口服IL-23受体拮抗肽），均以斑块状银屑病为主要适应症。 mezagitamab（TAK-079）为抗CD38单克隆抗体，目前处于III期，主要开发适应症为免疫性血小板减少症和IgA肾病，尚未获批。该药由公司内部研发。主要竞争品与同类项目包括argenx的Vyvgart（efgartigimod，FcRn拮抗剂，免疫性血小板减少症），以及诺华的Fabhalta（iptacopan）和Vanrafia（atrasentan），适应症为IgA肾病。 fazirsiran（TAK-999）为siRNA疗法，靶向异常Z型α1-抗胰蛋白酶的产生，目前处于III期，主要开发适应症为α1-抗胰蛋白酶缺乏症相关肝病，尚未获批。该项目为武田与Arrowhead Pharmaceuticals共同开发。目前该适应症尚无获批的病因治疗药物，同方向的在研项目包括Wave Life Sciences的WVE-006（RNA编辑）和贝殷思等企业的相关项目。血浆衍生疗法血浆衍生疗法板块FY2025收入10575亿日元，同比增长2.4%，业务基础来自Shire收购中取得的Baxalta血浆平台，主要包括免疫球蛋白与白蛋白两大产品群，以及血友病相关产品。免疫球蛋白产品群（GAMMAGARD LIQUID/KIOVIG、HYQVIA、CUVITRU）主要用于原发性免疫缺陷病、慢性炎性脱髓鞘性多发性神经根神经病和多灶性运动神经病。FY2025合计销售额7906亿日元，同比增长4.3%，增长主要由皮下注射制剂CUVITRU与HYQVIA带动；FY2026 Q1合计2139亿日元，同比增长10.2%。该产品群来自2019年对Shire的收购。主要竞争品包括杰特贝林的Hizentra与Privigen、基立福的Xembify与Gamunex-C，适应症同样覆盖原发性免疫缺陷病及慢性炎性脱髓鞘性多发性神经根神经病。白蛋白产品群（HUMAN ALBUMIN、FLEXBUMIN）用于低血容量、低白蛋白血症等。FY2025销售额1403亿日元；FY2026 Q1销售额346亿日元，同比增长7.5%，中国市场需求下降是主要拖累。该产品群同样来自Shire收购。主要竞争品为杰特贝林、基立福等企业的人血白蛋白产品。 ADVATE（重组人凝血因子VIII）用于A型血友病的出血控制与常规预防，FY2025销售额1055亿日元，同比下降5.6%；FY2026 Q1为258亿日元，同比下降7.7%。该药来自Shire收购（原Baxalta产品）。主要竞争品包括罗氏的Hemlibra（emicizumab，双特异性抗体）以及赛诺菲与Sobi的Altuviiio（efanesoctocog alfa），均用于A型血友病。罕见病领域罕见病板块FY2025收入7627亿日元，同比增长1.3%，主体资产来自Shire收购，覆盖遗传性血管性水肿、溶酶体贮积症、移植后感染及超罕见血液病等方向。 TAKHZYRO（拉那利尤单抗，lanadelumab）靶向血浆激肽释放酶，用于2岁及以上患者遗传性血管性水肿发作的长期预防，已在美国、欧洲、中国和日本获批。FY2025销售额2239亿日元，与上年基本持平；FY2026 Q1销售额599亿日元，同比增长8.7%，美国市场受竞争加剧影响下滑。该药来自2019年对Shire的收购（Shire于2016年通过收购Dyax取得该资产）。当前主要竞争品包括BioCryst的Orladeyo（berotralstat，口服血浆激肽释放酶抑制剂）、杰特贝林的Haegarda（C1酯酶抑制剂）以及Ionis与大冢的Dawnzera（donidalorsen，反义寡核苷酸），适应症均为遗传性血管性水肿的长期预防。 LIVTENCITY（马立巴韦，maribavir）靶向巨细胞病毒UL97激酶，用于移植后难治性巨细胞病毒感染。FY2025销售额469亿日元，同比增长42.2%；FY2026 Q1为141亿日元，同比增长33.7%，已在美国、日本上市并纳入中国医保。该药来自Shire收购（Shire于2014年收购ViroPharma取得）。主要竞争品为默沙东的Prevymis（letermovir），用于移植后巨细胞病毒感染的预防。 ADZYNMA（重组ADAMTS13）用于先天性血栓性血小板减少性紫癜，2023年12月获FDA批准。 FY2025销售额120亿日元，同比增长68.8%；FY2026 Q1为41亿日元，同比增长69.4%。该药由公司内部开发。该适应症目前尚无同机制的已上市竞争品，现有治疗以血浆输注为主。 ELAPRASE（艾度硫酸酯酶α，idursulfase）为酶替代疗法，用于亨特综合征（黏多糖贮积症II型），FY2025销售额1005亿日元。该药来自Shire收购。REPLAGAL（阿加糖酶α）用于法布雷病，同样来自Shire。肿瘤肿瘤板块FY2025收入5801亿日元，同比增长3.5%。上市产品以血液肿瘤与消化道肿瘤为主，在研端则通过信达生物与Keros的外部合作显著扩充了后期资产。 ADCETRIS（维布妥昔单抗，brentuximab vedotin）为靶向CD30的抗体偶联药物（ADC），已在70多个国家获批，用于霍奇金淋巴瘤、系统性间变性大细胞淋巴瘤等。FY2025销售额1402亿日元，同比增长8.7%；FY2026 Q1销售额485亿日元，同比增长30.3%，增长主要来自拉丁美洲。该药由武田与Seagen共同开发，武田持有美国和加拿大以外的全球权利（Seagen现为辉瑞子公司）。主要竞争品包括默沙东的Keytruda（帕博利珠单抗，PD-1）用于霍奇金淋巴瘤。 FRUZAQLA（呋喹替尼，fruquintinib）为高选择性VEGFR-1/2/3抑制剂，用于既往治疗失败的转移性结直肠癌，已在30多个国家获批或上市。FY2025销售额551亿日元，同比增长14.9%；FY2026 Q1销售额171亿日元，同比增长38.9%，增长主要来自欧洲。该药于2023年3月自和黄医药License-in，首付款4亿美元，另有最高7.3亿美元的研发、注册与销售里程碑及销售分成，武田取得中国大陆以外的全球权利，和黄医药保留中国大陆权益。主要竞争品包括拜耳的Stivarga（瑞戈非尼，多激酶抑制剂）和大鹏药品的Lonsurf（曲氟尿苷替匹嘧啶），均用于转移性结直肠癌后线治疗。 ICLUSIG（泊那替尼，ponatinib）为第三代BCR-ABL抑制剂，用于慢性髓性白血病和费城染色体阳性急性淋巴细胞白血病，FY2025销售额750亿日元，同比增长6.1%。该药来自2017年以约52亿美元收购ARIAD Pharmaceuticals。主要竞争品包括诺华的Scemblix（asciminib）和Tasigna（尼洛替尼），适应症同为慢性髓性白血病。 NINLARO（伊沙佐米，ixazomib）为口服蛋白酶体抑制剂，用于多发性骨髓瘤，FY2025销售额821亿日元，同比下降10.0%；FY2026 Q1为240亿日元。该药由公司内部研发。主要竞争品包括安进的Kyprolis（卡非佐米）与强生的Darzalex（达雷妥尤单抗），适应症同为多发性骨髓瘤。ALUNBRIG（布格替尼，brigatinib）用于ALK阳性非小细胞肺癌，来自ARIAD收购，FY2025因未来销售预期下调计提减值319亿日元。 elritercept是公司在血液肿瘤支持治疗方向的重点在研资产，为靶向activin A和B的融合蛋白，目前处于III期（RENEW研究、ELRiSE MDS研究），主要开发适应症为骨髓增生异常综合征相关输血依赖性贫血，并在骨髓纤维化相关贫血中推进，尚未获批。该项目于2024年12月自Keros Therapeutics引进，首付款2亿美元，研发、注册与销售里程碑合计可超过11亿美元，另有低双位数至高十几个百分点的分级销售分成，武田取得中国大陆、香港、澳门以外的全球权利。主要同类竞争品为百时美施贵宝的Reblozyl（luspatercept），同为activin受体配体捕获剂，已获批用于骨髓增生异常综合征相关贫血。 IBI363为PD-1/IL-2α-bias双特异性抗体融合蛋白，目前处于后期临床，主要开发适应症为非小细胞肺癌和结直肠癌，并在其他实体瘤中探索，尚未获批。 IBI343为靶向Claudin 18.2的ADC，已在日本和中国开展既往治疗胃癌的III期研究，并完成既往治疗胰腺癌的全球I/II期研究，武田计划将其推进至胃癌和胰腺癌一线治疗，尚未获批。两个项目均来自2025年10月与信达生物达成、2025年12月完成的授权与合作协议：首付款12亿美元（含1亿美元股权投资），里程碑最高约102亿美元，交易总额最高114亿美元； IBI363的主要同类竞争项目包括康方生物与Summit Therapeutics的ivonescimab（PD-1/VEGF双抗，非小细胞肺癌）；IBI343的主要竞争项目包括阿斯利康的AZD0901（Claudin 18.2 ADC，胃癌）以及安斯泰来已上市的Vyloy（zolbetuximab，Claudin 18.2单抗，胃癌与胃食管结合部腺癌）。 MIMRYLO（rusfertide）为首创hepcidin模拟肽，每周一次皮下注射，2026年8月28日获FDA批准，用于成人真性红细胞增多症的红细胞增多症治疗。获批主要基于III期VERIFY研究（293例患者），第20—32周临床应答率为76.9%，对照组为32.9%，常见不良反应为注射部位反应和贫血。该药于2024年1月自Protagonist Therapeutics License-in，首付款3亿美元，原协议约定美国市场50:50损益分成；2026年4月Protagonist行使美国opt-out权利，改为由武田承担美国商业化，武田在行权时支付2亿美元、并在FDA批准后支付2.75亿美元，Protagonist另可获得最高约9.75亿美元里程碑及全球分级销售分成。主要竞争品包括Incyte的Jakafi（芦可替尼，JAK1/2）和PharmaEssentia的Besremi（培罗干扰素α-2b），适应症同为真性红细胞增多症。神经科学神经科学板块FY2025收入4143亿日元，同比下降26.8%，主要源于VYVANSE/ELVANSE（赖氨酸右苯丙胺）在美国受仿制药冲击，其FY2025销售额2032亿日元，同比下降42.0%，FY2026 Q1为541亿日元，同比下降6.5%。 TRINTELLIX（沃替西汀，vortioxetine）用于重度抑郁症，FY2025销售额1218亿日元，FY2026 Q1为370亿日元。公司在该领域的新增长点为orexin产品线。 ORZEYFUL（oveporexton，TAK-861）为口服orexin受体2（OX2R）选择性激动剂，是首个针对1型发作性睡病病因机制的药物。该药由公司内部研发，为武田orexin产品线的先导资产，同系列的TAK-360正在1型发作性睡病、2型发作性睡病和特发性嗜睡症中研究，TAK-495处于早期阶段。当前主要同类竞争项目包括Alkermes的alixorexton和Centessa Pharmaceuticals的ORX750，均为口服OX2R激动剂并以发作性睡病为主要开发适应症；疫苗疫苗板块FY2025收入596亿日元，同比增长7.6%，核心产品为登革热疫苗。 QDENGA（TAK-003）为登革热减毒活疫苗，用于预防登革热，已在约30个国家和地区上市，涵盖多个登革热流行国家。FY2025销售额408亿日元，同比增长14.6%；FY2026 Q1销售额115亿日元，同比增长30.5%，增长主要来自拉丁美洲。该疫苗由公司内部开发（技术来源于2013年收购Inviragen）。主要竞争品为赛诺菲的Dengvaxia（登革热疫苗）。微信扫码，领取武田可编辑PPT 扫下方二维码，关注公众号后续会分享更多药企资料

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外链

KEGG	Wiki	ATC	Drug Bank
D11673 D11674	-	B06AC	DB15982

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
遗传性血管性水肿	美国	BioCryst Pharmaceuticals, Inc.	2020-12-03

未上市

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
I型和II型遗传性血管性水肿	临床3期	美国	BioCryst Pharmaceuticals, Inc.	2018-02-16
I型和II型遗传性血管性水肿	临床3期	澳大利亚	BioCryst Pharmaceuticals, Inc.	2018-02-16
I型和II型遗传性血管性水肿	临床3期	丹麦	BioCryst Pharmaceuticals, Inc.	2018-02-16
I型和II型遗传性血管性水肿	临床3期	以色列	BioCryst Pharmaceuticals, Inc.	2018-02-16
I型和II型遗传性血管性水肿	临床3期	意大利	BioCryst Pharmaceuticals, Inc.	2018-02-16
I型和II型遗传性血管性水肿	临床3期	瑞士	BioCryst Pharmaceuticals, Inc.	2018-02-16

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临床结果

适应症

分期

评价

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


AAAAI2026	N/A	遗传性血管性水肿	207	Berotralstat	醖製範網願繭鹽窪窪淵(遞醖獵範簾膚廠襯衊選) = 製遞窪範鬱糧鏇糧積築鹹觸製憲餘鬱膚餘顧繭 (鏇醖淵鹹壓網製選繭築 ) 更多	积极	2026-02-27
APeX-P (AAAAI2026)	临床3期	遗传性血管性水肿	29	Berotralstat	齋艱襯鏇鏇淵襯壓顧網(鑰獵鏇膚鹽網廠衊鹹艱) = Adverse events (AE) were consistent with those in prior berotralstat studies. No patients discontinued due to AEs 膚遞襯鏇鏇鹹構鹹鏇製 (獵齋製鑰顧夢憲淵齋鏇 )	积极	2026-02-27
APeX-P (AAAAI2026)	临床3期	遗传性血管性水肿	29	Standard of Care (SOC)		积极	2026-02-27
AAAAI2026	N/A	遗传性血管性水肿	466	Berotralstat (LTP-naïve)	餘築積獵製製糧壓鑰積(遞鏇窪簾鏇願繭簾觸淵) = 窪憲製鑰壓遞願蓋鏇鹽壓齋餘艱繭遞齋膚廠繭 (鬱糧顧齋願網鹽鬱襯繭 ) 更多	积极	2026-02-27
AAAAI2026	N/A	遗传性血管性水肿	466	Berotralstat (LTP-experienced)	餘築積獵製製糧壓鑰積(遞鏇窪簾鏇願繭簾觸淵) = 積餘網積襯鏇網窪範簾壓齋餘艱繭遞齋膚廠繭 (鬱糧顧齋願網鹽鬱襯繭 ) 更多	积极	2026-02-27
AAAAI2026	N/A	遗传性血管性水肿	499	Takhzyro	齋觸淵襯餘觸齋齋鑰鏇(窪夢鹽網膚鹽糧顧衊鹹) = 顧齋壓遞選鑰鑰顧選積遞繭膚簾夢廠蓋獵鬱顧 (鹹鏇襯鬱積範顧糧衊齋 ) 更多	不佳	2026-02-27
NCT03472040 (APeX-S, Pubmed \| Ann Allergy Asthma Immunol)	临床2期	遗传性血管性水肿 C1 inhibitor deficiency	387	Berotralstat 150 mg	製齋糧鹹憲鑰遞淵餘夢(遞繭鑰簾獵艱糧廠繭顧) = 11.1% 鹹衊齋構繭憲構艱醖願 (鏇鏇膚顧簾夢觸獵壓築 ) 更多	积极	2025-09-01
GlobeNewswire 人工标引	N/A	遗传性血管性水肿	56	ORLADEYO	鏇鏇衊鏇鹹顧糧願築願(淵齋觸簾鏇觸觸衊衊選) = 願遞網艱衊艱鏇糧蓋憲淵鑰範齋蓋鹹廠選範襯 (糧獵衊願構廠夢選遞觸 ) 更多	积极	2025-05-16
GlobeNewswire 人工标引	N/A	遗传性血管性水肿	99	ORLADEYO (aged 12-17)	積餘鏇鬱膚簾積繭淵築(艱觸製鬱選醖網淵齋繭) = 淵蓋鑰糧憲鹽鏇窪壓選製鹽顧遞膚窪鏇鏇築製 (積顧膚鬱鏇膚餘遞艱簾 ) 更多	积极	2025-05-16
APeX-P (AAAAI2025)	临床3期	遗传性血管性水肿	29	Berotralstat	淵淵願壓鏇顧襯鬱壓鹽(餘願觸鏇壓選製淵廠憲) = There were no drug-related Grade 3/4 AEs 簾夢壓鏇積齋壓蓋膚鏇 (鏇獵鹽製糧網鹹獵淵蓋 ) 更多	积极	2025-02-28
AAAAI2025	N/A	遗传性血管性水肿	242	Lanadelumab	築憲齋鑰鹹齋鬱襯夢築(鹽獵廠構膚遞築鏇遞構) = 鬱築鹹網艱積選範選構選壓蓋衊淵選壓積壓衊 (製淵鏇築選餘糧憲鬱衊 ) 更多	积极	2025-02-28
AAAAI2025	N/A	遗传性血管性水肿	242	Berotralstat	築憲齋鑰鹹齋鬱襯夢築(鹽獵廠構膚遞築鏇遞構) = 衊選築構醖積獵積構觸選壓蓋衊淵選壓積壓衊 (製淵鏇築選餘糧憲鬱衊 ) 更多	积极	2025-02-28
ACAAI2024 人工标引	N/A	遗传性血管性水肿	257	Berotralstat	憲鏇選願築衊鹹夢構淵(鑰構餘廠願獵憲鬱簾鑰) = 襯鹹衊遞遞夢獵網餘積網膚壓鏇膚廠鹹顧構築 (顧遞顧淵鹽鏇繭糧壓顧 ) 更多	积极	2024-10-24
ACAAI2024 人工标引	N/A	遗传性血管性水肿	257	Lanadelumab	憲鏇選願築衊鹹夢構淵(鑰構餘廠願獵憲鬱簾鑰) = 襯餘願窪鑰製壓淵觸鹹網膚壓鏇膚廠鹹顧構築 (顧遞顧淵鹽鏇繭糧壓顧 ) 更多	积极	2024-10-24