A Phase I, Multi-center, Multi-part Study to Investigate Safety, Tolerability, PK, PD, and Immunogenicity of RO7669330 Intravitreal Injections in Participants With GA Secondary to AMD: Part 1A: Open-label, MAD; Part 1B: Randomized PD Pilot; Part 2: Masked, Randomized, Active-comparator-controlled

The main purpose of this study is to assess the ocular and systemic safety and tolerability of RO7669330 in GA secondary to AMD after multiple unilateral intravitreal (IVT) doses.

开始日期2025-05-21

申办/合作机构

Roche Holding AG

NCT06161584

/ RecruitingN/A

A Prospective, Multicenter, Open-Label, Observational Phase 4 Study to Evaluate Real-World Safety, Tolerability, and Treatment Patterns of Pegcetacoplan (Syfovre) in Patients With Geographic Atrophy Secondary to Age-Related Macular Degeneration

A Prospective, Multicenter, Open-Label, Observational Phase 4 Study to Evaluate Real-World Safety, Tolerability, and Treatment Patterns of Pegcetacoplan (Syfovre) in Patients with Geographic Atrophy Secondary to Age-Related Macular Degeneration

开始日期2023-09-28

申办/合作机构

Apellis Pharmaceuticals, Inc.

JPRN-jRCT2041230022

/ Recruiting临床3期IIT

A PHASE 3, RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE-BLINDED, MULTICENTER STUDY TO EVALUATE THE EFFICACY AND SAFETY OF PEGCETACOPLAN IN PATIENTS WITH C3 GLOMERULOPATHY OR IMMUNE-COMPLEX MEMBRANOPROLIFERATIVE GLOMERULONEPHRITIS - A PHASE 3 STUDY TO EVALUATE THE EFFICACY AND SAFETY OF PEGCETACOPLAN IN PATIENTS WITH C3 GLOMERULOPATHY OR IMMUNE-COMPLEX MEMBRANOPROLIFERATIVE GLOMERULONEPHRITIS

开始日期2023-08-18

申办/合作机构-

100 项与 Pegcetacoplan 相关的临床结果

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100 项与 Pegcetacoplan 相关的转化医学

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100 项与 Pegcetacoplan 相关的专利（医药）

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196

项与 Pegcetacoplan 相关的文献（医药）

2025-08-01·AMERICAN JOURNAL OF OPHTHALMOLOGY

Ocular Adverse Events Associated with Pegcetacoplan and Avacincaptad Pegol for Geographic Atrophy: A Population-Based Pharmacovigilance Study

Article

作者: Kertes, Peter J ; Muni, Rajeev H ; Popovic, Marko M ; Mihalache, Andrew ; Kailani, Zeena

OBJECTIVE:

To evaluate the postmarketing ocular adverse events (AEs) reported for avacincaptad pegol and pegcetacoplan, the only Food and Drug Administration (FDA)-approved treatments for geographic atrophy (GA).

DESIGN:

Retrospective pharmacovigilance analysis.

SUBJECTS:

Ocular AE reports in the FDA Adverse Event Reporting System (FAERS) in which pegcetacoplan or avacincaptad pegol was identified as the primary suspect drug were analyzed.

METHODS:

Using the OpenVigil 2.1 data mining software, we conducted a retrospective pharmacovigilance analysis of the FAERS database from inception to December 2024. We conducted disproportionality analyses to assess reporting odds ratios (RORs) for specific drug-AE combinations compared with all other drugs in the database.

MAIN OUTCOME MEASURES:

Ocular AEs were evaluated.

RESULTS:

A total of 752 and 80 patients with AEs secondary to pegcetacoplan and avacincaptad pegol, respectively, were identified. Ocular AEs disproportionately overreported for pegcetacoplan included anterior segment (iris) hemorrhage (ROR 1767, 95% CI 538-5803), iris neovascularization (ROR 1248, 95% CI 502-3099), choroidal neovascularization (ROR 1328, 95% CI 956-1845), intraocular injection complication (ROR 2552, 95% CI 1607-4053), hemorrhagic occlusive retinal vasculitis (ROR 4606, 95% CI 2000-10,611), retinal occlusive vasculitis (ROR 2352, 95% CI 1313-4212), and bacterial endophthalmitis (ROR 1260, 95% CI 613-2589). Ocular AEs disproportionately overreported for avacincaptad pegol included choroidal neovascularization (ROR 1169, 95% CI 426-3205), vitritis (ROR 782, 95% CI 316-1936), dry age-related macular degeneration (ROR 684, 95% CI 316-1936), and cystoid macular edema (ROR 445, 95% CI 140-1412).

CONCLUSIONS:

Current prescribing patterns indicate that a broader spectrum of ocular AEs were reported for pegcetacoplan than avacincaptad pegol. These findings aim to enhance clinicians' understanding of the safety profiles of these agents, enabling informed patient care and heightened vigilance of these novel GA treatments.

2025-08-01·AMERICAN JOURNAL OF OPHTHALMOLOGY

Pegcetacoplan Treatment for Geographic Atrophy in Age-Related Macular Degeneration Over 36 Months: Data From OAKS, DERBY, and GALE

Article

作者: Boyer, David ; Abbey, Ashkan M ; Heier, Jeffrey ; Holz, Frank G ; Loewenstein, Anat ; Lin, Jason ; Mones, Jordi ; Li, Chao ; Wykoff, Charles C ; Vilupuru, Abhi ; Dhoot, Dilsher S ; Vajzovic, Lejla ; Singerman, Lawrence J ; Baumal, Caroline R ; Chiang, Allen

PURPOSE:

To report 12-month results from the GALE open-label extension study (NCT04770545), evaluating up to 36 months of intravitreal pegcetacoplan treatment for geographic atrophy (GA) in age-related macular degeneration (AMD).

DESIGN:

GALE is a prospective open-label extension study following the 24-month, sham-controlled, phase 3 OAKS (NCT03525613) and DERBY (NCT03525600) studies of pegcetacoplan.

PARTICIPANTS:

Patients with nonsubfoveal or subfoveal GA who completed OAKS, DERBY, or phase 1b APL2-103 (NCT03777332) studies.

METHODS:

Pegcetacoplan was administered monthly (PM) or every other month (PEOM) to all study eyes in GALE. Eyes receiving pegcetacoplan in OAKS and DERBY continued the same regimen (PM-PM and PEOM-PEOM), while eyes observed with sham in OAKS and DERBY crossed over to receive pegcetacoplan at the same dosing interval in GALE (SM-PM and SEOM-PEOM). Safety and efficacy through the first 12 months of GALE were assessed, reflecting up to 36 months of continuous pegcetacoplan treatment.

MAIN OUTCOME MEASURE:

Mean rate of change in GA area, total number of microperimetry scotomatous points, and adverse events.

RESULTS:

Through the first 12 months of GALE, 92.0% (727/790) patient retention was observed. Across all eyes, including eyes with nonsubfoveal and subfoveal GA, pegcetacoplan reduced the mean rate of change in GA area up to 32% versus projected sham. Year after year, the reductions in the mean rate of change in GA area increased, with up to a 42% reduction observed in eyes with nonsubfoveal GA in the PM-PM group compared with projected sham in the first year of GALE. An 18% reduction in new scotomatous points (P = .0156) was observed with PM-PM at 36 months, highlighting a significant impact in a prespecified microperimetry analysis. Adverse events included 33 (4.5%) eyes with exudative AMD, 15 (1.9%) intraocular inflammation (classified as mild or moderate in severity), 1 (0.1%) ischemic optic neuropathy, and 1 (0.1%) infectious endophthalmitis. No events of vasculitis were reported.

CONCLUSION:

Over 36 months, pegcetacoplan continued to reduce GA growth with increasing efficacy over time and reduced formation of new scotomatous points. The safety profile of pegcetacoplan in the first 12 months of GALE was consistent with the prior 24-month OAKS and DERBY studies.

2025-07-01·BLOOD CELLS MOLECULES AND DISEASES

Pegcetacoplan for the treatment of warm autoimmune hemolytic anemia: a case report

Letter

作者: Venou, Theodora Maria ; Vlantos, Vasileios Theodoros ; Gavriilaki, Eleni ; Mainou, Maria ; Evangelidis, Paschalis ; Vlachaki, Efthymia

415

项与 Pegcetacoplan 相关的新闻（医药）

2025-06-12

·PRNewswire

Sobi share new clinical data across multiple hematologic diseases at EHA 2025

STOCKHOLM, June 12, 2025 /PRNewswire/ -- Sobi® (STO: SOBI) will present data at the 30th EHA (European Haematology Association) hybrid congress, in Milan, Italy (12-15 June). The congress will feature the latest advances in the treatment of diffuse large B-cell lymphoma (DLBCL), immune thrombocytopenia (ITP), myelofibrosis, paroxysmal nocturnal haemoglobinuria (PNH), and primary hemophagocytic lymphohistiocytosis (pHLH). An extensive programme of poster presentations will showcase Sobi's commitment to helping patients with rare diseases by advancing treatment options. In addition, Sobi will host several scientific symposiums at the congress including: Advancing Therapeutic Knowledge in Paroxysmal Nocturnal Haemoglobinuria: Reshaping disease management to unlock new norms, Thursday, 12 June, 10:00am - 11:30am CEST, at Amber Hall 3 & 4. Boosting Platelets: Expert Approaches to Adult Immune Thrombocytopenia (ITP), Saturday 14 June, 8.00 am - 9.30 am CEST, at Amber Hall 7 & 8. Dissecting Treatment Sequencing in relapsed/refractory DLBCL, from laboratory to real life. Saturday, 14 June, 8:00 am – 9:30 am, CEST at Coral Hall 1. "The breadth of data that we share at this year's EHA congress demonstrates Sobi's comprehensive approach to addressing rare conditions in haematology. We are proud to contribute to advancing the science in several indications from early clinical phases in diffuse large B-cell lymphoma to clinical and real-world evidence in myelofibrosis, primary hemophagocytic lymphohistiocytosis, immune thrombocytopenia and paroxysmal nocturnal haemoglobinuria," said Lydia Abad-Franch, MD, Head of R&D and Medical Affairs, and Chief Medical Officer at Sobi. Key data to be presented at EHA 2025 About pegcetacoplan in rare diseases Pegcetacoplan is a targeted C3 therapy designed to regulate excessive activation of the complement cascade, a part of the body's immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is under investigation for rare diseases across haematology and nephrology. Pegcetacoplan is approved for the treatment of paroxysmal nocturnal haemoglobinuria (PNH) as EMPAVELI®/Aspaveli® in the United States, European Union, and other countries globally. About Doptelet® (avatrombopag) Doptelet® (avatrombopag) is indicated for the treatment of primary chronic immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments, and a treatment of severe thrombocytopenia in adult patients with chronic liver disease (CLD) who are scheduled to undergo an invasive procedure. About Zynlonta® (loncastuximab tesirine) Zynlonta® (loncastuximab tesirine) is a CD19-directed antibody drug conjugate (ADC). Zynlonta as monotherapy is indicated for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) and high-grade B-cell lymphoma (HGBL), after two or more lines of systemic therapy. About Sobi® Sobi is a global biopharma company unlocking the potential of breakthrough innovations, transforming everyday life for people living with rare diseases. Sobi has approximately 1,900 employees across Europe, North America, the Middle East, Asia and Australia. In 2024, revenue amounted to SEK 26 billion. Sobi's share (STO: SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com and LinkedIn. Contacts For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here. This information was brought to you by Cision . The following files are available for download: WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

免疫疗法上市批准

2025-06-12

·sobi.com

Sobi share new clinical data across multiple hematologic diseases at EHA 2025

Sobi® (STO: SOBI) will present data at the 30th EHA (European Haematology Association) hybrid congress, in Milan, Italy (12-15 June). The congress will feature the latest advances in the treatment of diffuse large B-cell lymphoma (DLBCL), immune thrombocytopenia (ITP), myelofibrosis, paroxysmal nocturnal haemoglobinuria (PNH), and primary hemophagocytic lymphohistiocytosis (pHLH). An extensive programme of poster presentations will showcase Sobi’s commitment to helping patients with rare diseases by advancing treatment options. In addition, Sobi will host several scientific symposiums at the congress including: 1.Advancing Therapeutic Knowledge in Paroxysmal Nocturnal Haemoglobinuria: Reshaping disease management to unlock new norms, Thursday, 12 June, 10:00am - 11:30am CEST, at Amber Hall 3 & 4. 2. Boosting Platelets: Expert Approaches to Adult Immune Thrombocytopenia (ITP), Saturday 14 June, 8.00 am - 9.30 am CEST, at Amber Hall 7 & 8. 3. Dissecting Treatment Sequencing in relapsed/refractory DLBCL, from laboratory to real life. Saturday, 14 June, 8:00 am – 9:30 am, CEST at Coral Hall 1. “The breadth of data that we share at this year’s EHA congress demonstrates Sobi’s comprehensive approach to addressing rare conditions in haematology. We are proud to contribute to advancing the science in several indications from early clinical phases in diffuse large B-cell lymphoma to clinical and real-world evidence in myelofibrosis, primary hemophagocytic lymphohistiocytosis, immune thrombocytopenia and paroxysmal nocturnal haemoglobinuria,” said Lydia Abad-Franch, MD, Head of R&D and Medical Affairs, and Chief Medical Officer at Sobi. Key data to be presented at EHA 2025 DLBCL PS1911: Initial Results From LOTIS-7: A Phase 1b Study of Loncastuximab Tesirine Plus Glofitamab in Patients With Relapsed/Refractory (R/R) Diffuse Large BCell Lymphoma (DLBCL) Presenting Author: Juan Pablo Alderuccio Poster presentation Session title: Poster Session 2Session date: Saturday, 14 June Session time: 18:30-19:30 CEST Location: Poster Hall PS1957: Updated Safety Run-in Results from LOTIS-5: A Phase 3, Randomized Trial of Loncastuximab Tesirine with Rituximab Versus Immunochemotherapy in Patients With R/R DLBCL Presenting Author: Carmelo Carlo-Stella Immune Thrombocytopenia (ITP) PF1236: Platelet Response to Avatrombopag Among Patients with Primary Immune Thrombocytopenia Who Switched from Eltrombopag or Romiplostim: the REAL-AVA 2.0 Real-World Study Presenting Author: Shruti Chaturvedi Poster presentation Session title: Poster Session 1Session date: Friday, 13 June Session time: 18:30 - 19:30 CEST Location: Poster Hall PF1239: Durability of Response to Avatrombopag Among Patients with Primary Immune Thrombocytopenia: The REAL-AVA 2.0 Real-World Study Presenting Author: Srikanth Nagalla PF1251: Clinically Meaningful Response to Avatrombopag: A Phase 3B Trial for Treatment of Children with ITP Presenting Author: Rachael F. Grace PS2231: Effectiveness and safety of avatrombopag for the treatment of adults with newly diagnosed, persistent, or chronic immune thrombocytopenia: Interim results from the phase 4 ADOPT study Presenting Author: Waleed Ghanima Poster presentation Session title: Poster Session 2Session date: Saturday, 14 June Session time: 18:30 - 19:30 CEST Location: Poster Hall PS2234: Efficacy and safety of avatrombopag for the treatment of pediatric immune thrombocytopenia in the open-label extension of a phase 3, randomized, double-blind, placebo-controlled trial Presenting Author: Rachael F. Grace PS2239: Real-World Treatment Patterns & Clinical Outcomes in Thrombopoietin Receptor Agonist Naive Patients with Immune Thrombocytopenia Treated with Avatrombopag: Interim Results from the REAL-AVA 3.0 Study Presenting Author: Sandhya Panch PS2242: Effectiveness and safety of avatrombopag for treatment of immune thrombocytopenia in older patients and those with comorbidities or prior TPO-RA exposure: Interim results from the phase 4 ADOPT study Presenting Author: María Eva Mingot-Castellano PS2244: Response to Avatrombopag Among Patients with Chronic and Persistent Primary Immune Thrombocytopenia: the REAL-AVA 2.0 Real-World Study Presenting Author: M Y Levy PS2250: Evaluation of Efficacy and Safety of Avatrombopag in Children with Immune Thrombocytopenia based on Disease Duration: Results from the Avatrombopag Phase 3-b Pediatric Trial Presenting Author: Rachael F. Grace PB3676: Baseline Correlates with Durability of Avatrombopag Response: A Phase 3B Trial for Treatment of Children with ITP Publication Only Published on May 14 at 15:30 CEST PB3684: Consistent Response to Avatrombopag across Various Baseline Characteristics: Results from the Phase 3-b Trial for the Treatment of Children with Immune Thrombocytopenia Myelofibrosis PF849: Hematologic improvement experienced by pacritinib-treated patients with myelofibrosis in real-world clinical settings Presenting Author: Michael Marrone Poster presentation Session title: Poster Session 1Session date: Friday, 13 June Session time: 18:30 - 19:30 CEST Location: Poster Hall PF1242: Efficacy of pacritinib vs momelotinib in patients with thrombocytopenic MF: a matched adjusted indicated treatment comparison Presenting Author: Koo Wilson PF1306: Transfusion-related cost and time burden offsets in patients with myelofibrosis treated with pacritinib compared to best available therapy based on PERSIST-2 trial Presenting Author: Abiola Oladapo PS1827: Real-world effectiveness of pacritinib in patients with myelofibrosis who have concurrent thrombocytopenia and anemia Presenting Author: Raajit Rampal Poster presentation Session title: Poster Session 2 Session date: Saturday, 14 June Session time: 18:30-19:30 CEST Location: Poster Hall PS1842: Real-World Treatment Patterns and Clinical Outcomes in Patients with Myelofibrosis Treated with Pacritinib (PAC) with platelets ≥50 x109/L at PAC initiation: Interim results from the MY-PAC Study Presenting Author: Doug Tremblay PS2295: Economic Burden of Cytopenia in Patients with Myelofibrosis: Analysis of a US National Administrative Claims Database Presenting Author: Lucia Marasova PB3079: Cytopenia is associated with real-world disease progression and diminished survival in patients with myelofibrosis: Analysis of a US national administrative claims database Publication Only Published on May 14 at 15:30 CEST Paroxysmal Nocturnal Hemoglobinuria PF672: Early response in complement inhibitor naïve patients with paroxysmal nocturnal hemoglobinuria treated with pegcetacoplan in the Phase 3 PRINCE trial Presenting Author: Austin Kulasekararaj Poster presentation Session title: Poster Session 1Session date: Friday, 13 June Session time: 18:30 - 19:30 CEST Location: Poster Hall PF676: Interim analysis of the ongoing COMPLETE study on the real-world effectiveness of pegcetacoplan in patients with paroxysmal nocturnal hemoglobinuria (PNH) Presenting Author: Regis Peffault de Latour PS1662: A benefit assessment of pegcetacoplan dose increase in the Phase 3 PEGASUS trial of PNH patients with difficult-to-control disease Presenting Author: Morag Griffin Poster presentation Session title: Poster Session 2Session date: Saturday, 14 June Session time: 18:30 - 19:30 CEST Location: Poster Hall PS1665: Benefit of pegcetacoplan in patients with paroxysmal nocturnal hemoglobinuria irrespective of baseline transfusion status Presenting Author: Britta Höchsmann Primary Hemophagocytic Lymphohistiocytosis (pHLH) PF1036: Emapalumab in patients with primary hemophagocytic lymphohistiocytosis: Efficacy and safety outcomes from a multinational, open-label, single-arm study Presenting Author: Franco Locatelli Poster presentation Session title: Poster Session 1Session date: Friday, 13 June Session time: 18:30 - 19:30 CEST Location: Poster Hall About pegcetacoplan in rare diseases Pegcetacoplan is a targeted C3 therapy designed to regulate excessive activation of the complement cascade, a part of the body’s immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is under investigation for rare diseases across haematology and nephrology. Pegcetacoplan is approved for the treatment of paroxysmal nocturnal haemoglobinuria (PNH) as EMPAVELI®/Aspaveli® in the United States, European Union, and other countries globally About Doptelet® (avatrombopag) Doptelet® (avatrombopag) is indicated for the treatment of primary chronic immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments, and a treatment of severe thrombocytopenia in adult patients with chronic liver disease (CLD) who are scheduled to undergo an invasive procedure. About Zynlonta® (loncastuximab tesirine)Zynlonta® (loncastuximab tesirine) is a CD19-directed antibody drug conjugate (ADC). Zynlonta as monotherapy is indicated for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) and high-grade B-cell lymphoma (HGBL), after two or more lines of systemic therapy. About Sobi® Sobi is a global biopharma company unlocking the potential of breakthrough innovations, transforming everyday life for people living with rare diseases. Sobi has approximately 1,900 employees across Europe, North America, the Middle East, Asia and Australia. In 2024, revenue amounted to SEK 26 billion. Sobi’s share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com and LinkedIn. Contacts For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here. We provide access to innovative treatments that transform life for people. Our therapies are concentrated within the areas of Haematology, Immunology and Specialty Care. We contribute to societies by improving access to treatment of rare diseases. Every day, we work actively to find better ways to understand and meet patient needs. Find out more about our business and financial performance. Here we present current and past media releases, images and videos.

临床3期免疫疗法临床结果上市批准临床1期

2025-06-10

·药明康德

潜在数十年来首款！美国FDA受理小分子疗法上市申请；蛋白尿下降近70%，疗效持续一年！潜在重磅疗法3期结果公布……

潜在数十年来首款！美国FDA受理小分子疗法上市申请Innoviva Specialty Therapeutics今日宣布，美国FDA已受理旗下药品zoliflodacin的新药申请（NDA）。Zoliflodacin是一款潜在“first-in-class”的spiropyrimidinetrione类口服抗生素，设计用于治疗12岁及以上成人和青少年的单纯性淋病。根据新闻稿，如获批准，该药将成为数十年来首个用于治疗淋病的新型抗生素，为当前耐药性日益严峻的感染治疗提供全新选择。FDA此次受理该NDA是基于多项临床试验的完整数据支持，这些试验是在与全球抗菌药物研发伙伴关系（GARDP）合作框架下完成的。其中，关键的3期临床试验结果显示，单次口服zoliflodacin在治疗泌尿生殖道淋病感染方面，在微生物治愈率上不劣于当前标准治疗方案（单次肌肉注射500毫克头孢曲松加口服1克阿奇霉素）。此外，试验中zoliflodacin整体耐受性良好，未观察到严重不良事件或死亡报告。蛋白尿下降近70%，疗效持续一年！潜在重磅疗法3期结果公布Apellis Pharmaceuticals与Sobi近日公布其3期VALIANT研究开放标签期的新数据，该试验评估Empaveli（pegcetacoplan）在治疗C3肾小球病（C3G）及原发性免疫复合物性膜增生性肾小球肾炎（IC-MPGN）中的疗效与安全性。结果显示，与安慰剂相比，Empaveli在第26周实现了蛋白尿下降达68%，差异具有统计学意义。这一疗效在治疗持续一年后仍得以维持。同时，接受Empaveli治疗的患者在估算肾小球滤过率（eGFR）指标上显示出肾功能的持续稳定。此外，在开放标签期开始时从安慰剂转为Empaveli治疗的患者，也观察到与试验早期用药组相近程度的蛋白尿下降与肾功能稳定改善，进一步验证了该药在不同起始时间下的一致的疗效表现。Empaveli整体安全性良好，耐受性与既往研究结果一致，未发现新的安全性信号，支持其在该类补体介导性肾病中的持续开发潜力。美国FDA与欧洲药品管理局（EMA）正在对该药物用于肾病的监管申请进行审评。Empaveli是一款靶向C3补体蛋白的聚乙二醇化（PEGylated）双环肽疗法。双环肽分子集抗体、小分子药物及肽类的特性于一身，具有与抗体类似的亲和性和精确的靶向特异性；同时，由于它们分子量较小，使得其能够快速深入地渗透组织。2021年5月，Empaveli获FDA批准用于治疗阵发性睡眠性血红蛋白尿症（PNH）。2023年2月，它获得FDA批准用以治疗由年龄相关性黄斑变性（AMD）引起的地图样萎缩（GA）。值得一提的是，这款药物被行业媒体Evaluate评为2023年有望获批的十大潜在重磅疗法之一。首款！RNAi疗法在欧盟再获批准Alnylam Pharmaceuticals日前宣布，欧盟委员会（EC）已批准其RNAi疗法Amvuttra（vutrisiran）用于治疗伴有心肌病的转甲状腺素蛋白淀粉样变性（ATTR-CM）患者。根据新闻稿，这一批准使Amvuttra成为获得EC批准可用于治疗ATTR-CM及遗传性转甲状腺素蛋白介导（hATTR）淀粉样变性的多发性神经病（polyneuropathy）的首个RNAi疗法。此次批准主要基于关键3期HELIOS-B研究的积极结果。该研究为一项随机、双盲、安慰剂对照的多中心全球试验。分析显示，Amvuttra在总体和单药治疗人群中均达成所有10项预设的主要与次要终点，显著降低全因死亡与复发性心血管事件，同时改善6分钟步行距离、健康状态、生活质量（KCCQ评分）以及心力衰竭症状与严重程度（NYHA分级）。在总体人群中，与安慰剂相比，Amvuttra在主要复合终点（全因死亡和复发性心血管事件）方面实现了28%的风险降低。在一项预设的次要终点分析中，包括最长36个月的双盲期及6个月的开放标签延长期，该患者群体的死亡率显著下降了36%，进一步印证其临床益处。参考资料：[1] Innoviva Specialty Therapeutics Receives FDA New Drug Application Acceptance for Zoliflodacin, a First-in-Class Oral Antibiotic for Uncomplicated Gonorrhea in Adults. Retrieved June 10, 2025 from https://innovivaspecialtytherapeutics.com/innoviva-specialty-therapeutics-receives-fda-new-drug-application-acceptance-for-zoliflodacin-a-first-in-class-oral-antibiotic-for-uncomplicated-gonorrhea-in-adults/[2] Apellis and Sobi Announce EMPAVELI® (pegcetacoplan) Showed Sustained Efficacy at One Year in Phase 3 Study for C3G and Primary IC-MPGN. Retrieved June 10, 2025 from https://www.globenewswire.com/news-release/2025/06/06/3095128/0/en/apellis-and-sobi-announce-empaveli-pegcetacoplan-showed-sustained-efficacy-at-one-year-in-phase-3-study-for-c3g-and-primary-ic-mpgn.html[3] Alnylam Receives European Commission Approval for AMVUTTRA® (vutrisiran) for the Treatment of ATTR Amyloidosis with Cardiomyopathy. Retrieved June 10, 2025 from https://www.businesswire.com/news/home/20250609199979/en/Alnylam-Receives-European-Commission-Approval-for-AMVUTTRA-vutrisiran-for-the-Treatment-of-ATTR-Amyloidosis-with-Cardiomyopathy免责声明：本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

临床3期临床结果突破性疗法加速审批申请上市

100 项与 Pegcetacoplan 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D11613	Pegcetacoplan	B03XA	-

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
地图样萎缩	美国	Apellis Pharmaceuticals, Inc.	2023-02-17
阵发性睡眠性血红蛋白尿症	美国	Apellis Pharmaceuticals, Inc.	2021-05-14

未上市

10 条进展最快的记录,

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
C3肾小球病	申请上市	欧盟	Apellis Pharmaceuticals, Inc.	2025-02-20
C3肾小球病	申请上市	欧盟	Swedish Orphan Biovitrum AB	2025-02-20
膜增生性肾小球肾炎	申请上市	欧盟	Apellis Pharmaceuticals, Inc.	2025-02-20
膜增生性肾小球肾炎	申请上市	欧盟	Swedish Orphan Biovitrum AB	2025-02-20
移植物功能延迟恢复	临床3期	-	Apellis Pharmaceuticals, Inc.	2025-09-01
终末期肾脏病	临床3期	-	Apellis Pharmaceuticals, Inc.	2025-09-01
冷凝集素病	临床3期	美国	Swedish Orphan Biovitrum AB	2022-10-20
冷凝集素病	临床3期	日本	Swedish Orphan Biovitrum AB	2022-10-20
冷凝集素病	临床3期	奥地利	Swedish Orphan Biovitrum AB	2022-10-20
冷凝集素病	临床3期	比利时	Swedish Orphan Biovitrum AB	2022-10-20

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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


NCT04770545 (OAKS, DERBY, and GALE, Pubmed \| Am J Ophthalmol)	临床3期	年龄相关性黄斑变性	790	Pegcetacoplan monthly	膚襯鏇齋淵齋蓋範積膚(壓鬱製顧積鹹遞襯繭窪) = 33 (4.5%) eyes with exudative AMD 醖願簾鹹選願鹹糧壓鏇 (憲觸壓鹽構糧糧積窪網 ) 更多	积极	2025-08-01
NCT03500549 (Pubmed \| Eur J Haematol)	临床3期	阵发性睡眠性血红蛋白尿症 complement 5 inhibitors (C5i; eculizumab, ravulizumab)	-	Pegcetacoplan	鏇鏇網選鑰醖糧襯積餘(壓壓衊夢廠糧範衊鑰積) = 鑰選艱窪餘壓築築遞衊廠顧獵糧遞餘觸糧淵遞 (壓鏇鑰鑰構獵鬱壓鑰觸, -90.02 ~ 21.21)	积极	2025-04-25
NCT04579666 (CTgov)	临床2期	肌萎缩侧索硬化	249	RTP+Pegcetacoplan (RTP: Pegcetacoplan)	簾餘簾醖衊網膚鬱範鑰(窪蓋構憲糧製艱鬱膚範) = 築觸網壓窪糧鹹鏇積艱鬱願獵築製鏇糧選簾醖 (醖膚窪蓋獵鏇廠簾壓遞, 4.71) 更多	-	2025-04-24
				placebo+pegcetacoplan (RTP: Placebo)	簾餘簾醖衊網膚鬱範鑰(窪蓋構憲糧製艱鬱膚範) = 糧繭獵遞醖鏇願衊願遞鬱願獵築製鏇糧選簾醖 (醖膚窪蓋獵鏇廠簾壓遞, 6.89) 更多
NCT03453619 (CTgov)	临床2期	特发性膜性肾病 \| 膜增生性肾小球肾炎 \| C3肾小球病 ... 更多	21	pegcetacoplan (Part A: IgAN Subjects)	遞蓋繭網積鏇憲鑰製艱(壓齋鏇鹹壓構遞淵獵壓) = 壓齋齋膚壓淵選夢淵積艱齋顧遞蓋夢獵選鏇顧 (鏇鑰鑰廠淵網鏇鹹醖膚, 齋醖夢觸鏇鑰網齋糧顧 ~ 蓋網遞觸窪憲遞衊餘積) 更多	-	2025-02-13
				pegcetacoplan (Part A: LN Subjects)	遞蓋繭網積鏇憲鑰製艱(壓齋鏇鹹壓構遞淵獵壓) = 廠廠網膚製壓衊窪鹹繭艱齋顧遞蓋夢獵選鏇顧 (鏇鑰鑰廠淵網鏇鹹醖膚, 襯艱鹹膚齋觸鹽構淵簾 ~ 選遞願繭憲願膚餘觸鬱) 更多
NCT04572854 (NOBLE, CTgov)	临床2期	膜增生性肾小球肾炎 \| C3肾小球病 \| 肾小球肾炎慢性补体成分 3 肾小球病	13	pegcetacoplan (Part A: Controlled Portion: Group 1)	醖夢艱鏇獵築願選襯鏇 = 糧艱製廠膚築選鑰齋鬱襯築製鏇遞製顧淵範構 (鑰選鹽餘構壓網獵構蓋, 窪餘選鏇築獵觸繭夢顧 ~ 窪醖積窪鑰選積廠衊願) 更多	-	2025-02-07
				pegcetacoplan (Part A: Controlled Portion: Group 2)	醖夢艱鏇獵築願選襯鏇 = 積願簾齋觸糧餘構鬱網襯築製鏇遞製顧淵範構 (鑰選鹽餘構壓網獵構蓋, 糧鹹觸膚夢襯製鹽築膚 ~ 遞襯觸範鏇壓積窪餘鬱) 更多
NCT03226678 (CTgov)	临床2期	冷凝集素病 \| 温热型自身免疫性溶血性贫血	24	wAIHA (Part A: Cohort 1 wAIHA - 270 mg)	鹽蓋襯鏇積衊觸壓憲願 = 製衊餘醖醖鹽遞顧獵範選積顧夢艱衊襯鏇獵積 (積醖齋選簾膚淵觸製網, 獵艱鑰膚遞鑰蓋壓夢艱 ~ 遞膚壓鹽構醖餘壓獵鑰) 更多	-	2024-12-12
				wAIHA (Part A: Cohort 1 wAIHA - 360 mg)	鹽蓋襯鏇積衊觸壓憲願 = 廠衊窪獵鹹衊醖築蓋窪選積顧夢艱衊襯鏇獵積 (積醖齋選簾膚淵觸製網, 願鬱鏇糧窪遞蓋醖鏇襯 ~ 網繭願製廠窪壓積積觸) 更多
ASH2024	N/A	阵发性睡眠性血红蛋白尿症	-	Complement Inhibitor Experienced	簾觸獵廠糧願網壓選顧(觸夢淵獵餘衊壓觸鹹獵) = 鹹鏇鹹遞網鏇鬱膚鬱製鹹範鹹憲繭築夢艱蓋壓 (製範網齋鏇範衊鹹壓簾, 1.7) 更多	-	2024-12-09
				Complement Inhibitor Naïve	簾觸獵廠糧願網壓選顧(觸夢淵獵餘衊壓觸鹹獵) = 鹽築顧簾廠積積網鬱憲鹹範鹹憲繭築夢艱蓋壓 (製範網齋鏇範衊鹹壓簾, 1.3) 更多
ASH2024	N/A	阵发性睡眠性血红蛋白尿症	-	Pegcetacoplan	鏇範夢顧範淵艱齋壓夢(糧憲範築獵觸齋襯膚遞) = 13 patients experienced 1 to 3 acute hemolytic events (n=20, median 1 per patient, IQR [1; 2]) with hospitalization required to manage 6 events 壓齋襯積鑰鬱醖網醖蓋 (選鹽選憲鹽願網醖構鹹 ) 更多	-	2024-12-07
EURETINA2024	N/A	地图样萎缩	-	Pegcetacoplan 15 mg monthly	鹽積遞構餘獵艱膚鑰醖(製蓋鑰觸鑰簾廠糧築蓋) = 鹽廠鹹淵簾範繭窪餘觸鹹醖衊鹽構鹹窪範顧糧 (醖鹹蓋製壓顧艱觸觸範, -1.385 ~ -0.046)	-	2024-09-19
EURETINA2024	N/A	地图样萎缩	-	Intravitreal pegcetacoplan	糧廠鹽鹽壓鏇壓齋鑰繭(鑰構襯膚顧齋簾鹹築憲) = 齋願蓋鬱築顧觸獵膚艱築願廠願餘鹽蓋餘遞衊 (獵淵製餘窪製艱夢鬱襯, 0.13) 更多	-	2024-09-19
				Sham	糧廠鹽鹽壓鏇壓齋鑰繭(鑰構襯膚顧齋簾鹹築憲) = 觸顧餘網餘積淵製鹹襯築願廠願餘鹽蓋餘遞衊 (獵淵製餘窪製艱夢鬱襯, 0.18) 更多