RegulationOrphan Drug (United States), Orphan Drug (European Union), Priority Review (China), Orphan Drug (South Korea), Orphan Drug (Australia), Orphan Drug (Japan)

Structure/Sequence

Sequence Code 66437L

Source: *****

Sequence Code 66453H

Source: *****

Clinical Trials associated with Eculizumab

NCT05646563

/ Not yet recruitingPhase 2

A Phase II, Open-Label Study of NM8074 in Soliris®-Treated Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)

This is a Phase II, open-label study designed to evaluate the safety, efficacy, and immunogenicity of NM8074 in PNH patients undergoing complement-inhibitor therapy with Soliris.

Start Date01 Jan 2026

Sponsor / Collaborator

NovelMed Therapeutics, Inc.

NCT06724809

/ RecruitingPhase 3

An Open-label, Single-arm, Multi-Center, Interventional Study to Evaluate the Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Eculizumab in Chinese Adult Participants With Anti-Aquaporin-4 Antibody Positive Neuromyelitis Optica Spectrum Disorders (NMOSD)

The primary objective of this study is to evaluate the efficacy, safety, pharmacokinetics, pharmacodynamics, and immunogenicity of Eculizumab in Chinese Adults with Neuromyelitis Optica Spectrum Disorders (NMOSD).

Start Date16 Jan 2025

Sponsor / Collaborator

Alexion Pharmaceuticals, Inc.

[+1]

CTR20244429

/ RecruitingPhase 3

一项在患有抗水通道蛋白4抗体阳性视神经脊髓炎谱系疾病（NMOSD）的中国成人受试者中评价依库珠单抗的有效性、安全性、药代动力学、药效学和免疫原性的开放性、单臂、多中心、干预性研究

[Translation] An open-label, single-arm, multicenter, interventional study to evaluate the efficacy, safety, pharmacokinetics, pharmacodynamics, and immunogenicity of eculizumab in Chinese adult subjects with anti-aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder (NMOSD)

试验目的是研究依库珠单抗在患有抗水通道蛋白4抗体阳性视神经脊髓炎谱系疾病（NMOSD）的中国成人受试者中的有效性、安全性、药代动力学、药效学和免疫原性。

[Translation]

The purpose of the trial is to study the efficacy, safety, pharmacokinetics, pharmacodynamics, and immunogenicity of eculizumab in Chinese adult subjects with anti-aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder (NMOSD).

Start Date16 Jan 2025

Sponsor / Collaborator

AstraZeneca Investment (China) Co. Ltd.

[+3]

100 Clinical Results associated with Eculizumab

100 Translational Medicine associated with Eculizumab

100 Patents (Medical) associated with Eculizumab

2,698

Literatures (Medical) associated with Eculizumab

31 Dec 2025·Hematology

Eculizumab treatment for Chinese patients with hemolytic paroxysmal nocturnal hemoglobinuria (PNH): efficacy and safety – a single-center study

Article

Author: Yang, Chen ; Chen, Miao ; Han, Bing ; Liu, Ziwei ; Wang, Leyu

OBJECTIVE:

To evaluate the short-term efficacy and safety of eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in China.

METHOD:

Data were retrospectively collected from patients with PNH who received at least 3 months of full-dose eculizumab. Changes in clinical and laboratory indicators after 1, 3, and 6 months of eculizumab therapy and at the end of follow-up were documented. The incidence rates of breakthrough hemolysis (BTH), extravascular hemolysis (EVH), and adverse events were recorded.

RESULT:

A total of 48 patients, including 27 males, with a median age of 46 (12-78) years were included. Twenty-four (50%) patients had classic PNH and 24 (50%) had bone marrow failure (BMF)/PNH. Eighteen (37.5%) patients required blood transfusion. The median duration of follow-up was 6 (3-15) months. During the follow-up period, Lactate Dehydrogenase (LDH) levels were lower than those at baseline (<0.05) at all observation points. The patients showed a significant reduction in creatinine levels from baseline (P = 0.022 and P = 0.039, respectively) at 1 and 3 months. At the end of the follow-up, fifteen (83.3%) became transfusion-independent. No new thrombotic events were observed. The FACIT-Fatigue score significantly improved (P < 0.05). No significant differences were observed in the changes in hemoglobin or LDH levels between patients with classic PNH and those with BMF/PNH. BTH was observed in 17.4% of patients and EVH in 10.4%. Mild adverse events occurred in 22.9% of patients. No deaths or clonal evolution was observed.

CONCLUSION:

Eculizumab can effectively control the hemolytic symptoms of PNH with good tolerance for Chinese patients.

01 Jun 2025·JOURNAL OF PHARMACEUTICAL AND BIOMEDICAL ANALYSIS

Development and application of UHPLC-MS/MS method to quantify eculizumab in PNH patients

Article

Author: Sheng, Ning ; Liu, Xin ; Zhang, Bo ; Zhang, Jinlan ; Han, Bing ; Zhang, Zhihui

To evaluate the optimal treatment scheme and minimize unnecessary use of the highly expensive orphan drug eculizumab, therapeutic drug monitoring should be performed. The aim of this study is to establish a validated ultra-high-performance liquid chromatography-tandem mass spectrometry (UHPLC-MS/MS) method to quantify the concentration of eculizumab in human plasma. Plasma samples were prepared using protein G beads, followed by trypsin digestion. A signature peptide for eculizumab (LLIYGATNLADGVPSR) was selected and quantified by UHPLC-MS/MS in the multiple reaction-monitoring mode. The stable isotope-labeled signature peptide *LLIYGATNLADGVPSR (¹³C₆, ¹⁵N₄-labeled arginine) was used as the internal standard (IS). The UHPLC-MS/MS exhibited good specificity and no matrix effects when employing IS. The calibration curves of eculizumab presented a strong linear relationship from 5 to 1000 µg·mL^-1, surpassing the scope of previously published methods and encompassing the concentration levels typically observed in clinical samples. A total of 15 Chinese PNH patients treated with eculizumab were enrolled. The successful application of the method to clinical samples and the data agreement with the ELISA data both demonstrated that established UHPLC-MS/MS is a reliable approach for quantifying eculizumab. Furthermore, this is the first published method used to determine the eculizumab concentration in Chinese PNH patients. The study also suggests that there are large interindividual variations in eculizumab pharmacokinetics among Chinese PNH patients.

01 May 2025·PEDIATRIC BLOOD & CANCER

Pegcetacoplan for Breakthrough Hemolysis in an Adolescent With Classical Paroxysmal Nocturnal Hemoglobinuria on Eculizumab

Letter

Author: Njeru, Catherine ; Baker, Danielle ; Amid, Ali ; Merkeley, Hayley

233

News (Medical) associated with Eculizumab

07 Apr 2025

·pipelinereview.com

Teva and Samsung Bioepis Announce Biosimilar EPYSQLI® (eculizumab-aagh) Injection Now Available in the United States

PARSIPPANY, NJ, USA and INCHEON, Korea I April 07, 2025 I Teva Pharmaceuticals, a U.S. affiliate of Teva Pharmaceutical Industries Ltd. (NYSE and TASE: TEVA), and Samsung Bioepis Co., Ltd. today announced the availability of EPYSQLI ® (eculizumab-aagh) in the U.S. EPYSQLI is a biosimilar to Soliris ® (eculizumab) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS) and generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AchR) antibody positive. EPYSQLI will be offered at a 30% discount of the Wholesale Acquisition Cost (WAC) of the reference product, Soliris ® , offering one of the greatest cost-saving biosimilars to Soliris ® in the U.S. “Individuals living with rare diseases, including paroxysmal nocturnal hemoglobinuria, atypical hemolytic uremic syndrome and generalized myasthenia gravis, often have limited access to life-enhancing medicines,” said Thomas Rainey, Senior Vice President, U.S. Biosimilars at Teva. “We are proud to launch EPYSQLI in the U.S. as a new, more affordable treatment option to help expand access to these underserved communities, further expanding our biosimilars medicine portfolio and efforts to deliver important medicines to patients through strategic partnerships that leverage our proven commercialization abilities.” “Life-threatening diseases such as PNH, aHUS and gMG, if left untreated, can lead to kidney disease, kidney failure or respiratory failure, and it is important for patients to have early access to treatment. The availability of EPYSQLI means that patients now have an additional treatment option available at a lower cost, with the proven quality, safety and efficacy comparable to the reference product. Based on our robust track record supplying biosimilars in the U.S., we are well-positioned to deliver this life-changing medicine to patients,” said Linda MacDonald, Executive Vice President and Head of Global Commercial Division of Samsung Bioepis. “Our mission is to positively impact and ensure the sustainability of health care systems by offering affordable, quality-assured, safe and effective biologic medicines. We will work closely with Teva to ensure access of this medicine for payers, healthcare professionals and patients in the U.S.,” she added. The monoclonal antibody and anti-C5 complement inhibitor eculizumab is a well-established standard of care to treat PNH and aHUS, rare diseases with an estimated U.S. prevalence of approximately 50,000 and 5,000 respectively. 1,2 Approximately 70% of eculizumab-treated PNH patients are not dosed according to the label, and two-thirds of patients discontinue eculizumab within an average of 1.5 years, which can be attributed to several factors including the high treatment cost. 3 Biosimilars, like EPYSQLI, are highly similar to their reference product with no clinically meaningful differences in safety, purity, or potency, and can increase the affordability and accessibility associated with these therapies. Biosimilars are approved according to the same standards of pharmaceutical quality that apply to all biological medicines, with comparable safety and efficacy to the reference product expected. 4 The introduction of biosimilars leads to higher utilization of the molecule as lower costs offer increased access to patients. 5 In July 2024, EPYSQLI was approved by the U.S. Food and Drug Administration (FDA) as a biosimilar to Soliris ® for the treatment of patients with PNH to reduce hemolysis, and aHUS to inhibit complement-mediated thrombotic microangiopathy. In November 2024, its indication was expanded to include the treatment of gMG in adult patients who are AchR antibody positive. The FDA has provisionally determined that EPYSQLI will be interchangeable with the reference biologic Soliris ® , following the expiration of exclusivity for the first interchangeable biosimilar. In January 2025, Teva and Samsung Bioepis entered into a strategic partnership for the commercialization of EPYSQLI in the U.S. market. Under the terms of the agreement, Samsung Bioepis handles development, manufacturing and supply, while Teva is responsible for the commercialization of the product in the U.S., leveraging its experience and extensive sales and marketing infrastructure. About EPYSQLI ® (eculizumab-aagh) injection, for intravenous use EPYSQLI is a complement inhibitor indicated for the treatment of patients with: Limitation of Use: EPYSQLI is not indicated for the treatment of patients with Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS). Please click here for full Prescribing Information for EPYSQLI, including BOXED WARNINGS. About Teva Teva Pharmaceutical Industries Ltd. (NYSE and TASE: TEVA) is a different kind of global biopharmaceutical leader, one that operates across the full spectrum of innovation to reliably deliver medicines to patients worldwide. For over 120 years, Teva’s commitment to bettering health has never wavered. Today, the company’s global network of capabilities enables its 37,000 employees across 57 markets to advance health by developing medicines for the future while championing the production of generics and biologics. We are dedicated to addressing patients’ needs, now and in the future. Moving forward together with science that treats, inspired by the people we serve. To learn more about how Teva is all in for better health, visit www.tevapharm.com . About Samsung Bioepis Established in 2012, Samsung Bioepis is a biopharmaceutical company committed to realizing healthcare that is accessible to everyone. Through innovations in product development and a firm commitment to quality, Samsung Bioepis aims to become the world’s leading biopharmaceutical company. Samsung Bioepis continues to advance a broad pipeline of biosimilar candidates that cover a spectrum of therapeutic areas, including immunology, oncology, ophthalmology, hematology, nephrology, and endocrinology. For more information, please visit: www.samsungbioepis.com and follow us on social media – X , LinkedIn . References SOURCE: Samsung Bioepis

Drug ApprovalLicense out/inAcquisition

03 Apr 2025

·endpts.com

FDA approves Amgen's Uplizna in IgG4-related disease

The FDA on Thursday approved Amgen’s rare disease drug Uplizna in an autoimmune disorder, setting the stage for the therapy to gain a foothold in the rare disease market. Uplizna is now approved to treat IgG4-related disease, a condition in which the immune system causes the formation of tumor-like masses that lead to organ damage. It’s also the first FDA-approved treatment for IgG4-related disease. Uplizna’s average wholesale acquisition cost is $140,248.50 per dose, an Amgen spokesperson said. Patients will take two initial loading doses followed by infusions every six months, leading to an annual cost of $280,497 after the first year. The new approval helps Amgen as it positions Uplizna as a drug that can potentially treat a variety of related rare autoimmune conditions. In addition to IgG4-related disease and the rare eye disorder, called neuromyelitis optica spectrum disorder, Amgen is also planning to submit the drug to the FDA for myasthenia gravis by July. AstraZeneca, UCB and argenx already have approved treatments for myasthenia gravis. “We’re covering a spectrum of cells that are responsible for generating antibodies,” Amgen SVP of global development Narimon Honarpour said. “It’s really about maximizing the opportunity once you have seen that your medicine has worked in one area, and knowing about the mechanism of action in a way that enables you to anticipate potential benefits in other conditions.” In the Phase 3 study for IgG4-related disease, Uplizna reduced the risk of a disease-caused flare by 87% after one year compared to placebo. It also achieved all secondary endpoints. Uplizna’s peak sales in IgG4-related disease could reach up to $3 billion, TD Cowen analyst Yaron Werber wrote in a note last year. He estimates the US market is between 7,000 to 14,000 patients. Though other companies haven’t focused much on IgG4-related disease, Uplizna may eventually have to carve out a slice of the myasthenia gravis market. AstraZeneca’s Soliris and Ultomiris drugs are both approved in this indication, as well as argenx’s Vyvgart. UCB’s Rystiggo and Zilbrysq are also approved for myasthenia gravis. Additionally, Johnson & Johnson, Immunovant and Biohaven are all developing drugs for myasthenia gravis that could reach the market in the next few years. The myasthenia gravis market was estimated to be about $1.5 billion last year, according to GlobalData, and forecasts for the next five to 10 years range from $3 billion to $7 billion. Uplizna succeeded in a Phase 3 trial in myasthenia gravis last September, and Amgen shared additional positive data last month. Amgen acquired Uplizna as part of its 2022 acquisition of Horizon Therapeutics, which got the drug in a buyout of Viela Bio. Editor’s note: This article was updated to include Uplizna’s pricing information.

Phase 3Drug ApprovalClinical ResultAcquisition

27 Mar 2025

·www.astrazeneca.com

Alexion data presented at the 2025 AAN Annual Meeting highlight advancements in improving care for rare neurological diseases

Alexion, AstraZeneca Rare Disease, will present robust clinical and real-world data from its rare neurology portfolio at the American Academy of Neurology (AAN) Annual Meeting in San Diego, CA, 5 to 9 April 2025. The company will present 21 abstracts, including three oral presentations, across neuromyelitis optica spectrum disorder (NMOSD) and generalised myasthenia gravis (gMG). Presentations will offer analyses of the CHAMPION-NMOSD and PREVENT trials as well as real-world data evaluating the long-term safety profile and efficacy of Ultomiris (ravulizumab) and Soliris (eculizumab) in NMOSD and gMG. Christophe Hotermans, Senior Vice President, Head of Global Medical Affairs, Alexion, said: “At this year’s AAN, we continue to build on our legacy of innovation for people living with rare diseases by presenting new real-world and clinical evidence that highlight the transformational impact of Ultomiris and Soliris in the long-term treatment of NMOSD and gMG. This latest data will reinforce the sustained efficacy and safety profile of Ultomiris and Soliris, as well as the benefits of long-term treatment in these patient populations, offering meaningful insights to help improve patient care and deepen disease understanding.” Continued evidence reinforcing the safety profile and efficacy of Ultomiris and Soliris Two oral presentations will provide clinical and real-world evidence supporting the sustained long-term safety profile and efficacy of Ultomiris and Soliris in adults with the most common forms of NMOSD and gMG, respectively. An interim analysis of the ongoing Phase III CHAMPION-NMOSD clinical trial will reinforce the potential for Ultomiris to prevent relapses in adults with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) NMOSD. Data will demonstrate that zero patients treated with Ultomiris experienced an adjudicated relapse through a median follow-up of 170.3 weeks. In addition, most patients treated with Ultomiris were clinically stable or improved on disability measures through the longer-term follow-up highlighting the importance of preventing relapses to reduce disability decline in patients with NMOSD. Results from a presentation spotlighting the three-year analysis from postmarketing surveillance (PMS) in Japan will show that treatment with Soliris demonstrated sustained effectiveness in patients with anti-acetylcholine receptor (AChR) antibody-positive (Ab+) gMG, regardless of Myasthenia Gravis Foundation of America (MGFA) classification, which is used to assess the severity of disease. Additionally, in an encore poster presentation, results from the first interim analysis from the Ultomiris gMG PMS in Japan will show that clinical effectiveness benefits were maintained in adult patients with AChR-Ab+ gMG who switched to Ultomiris from prior Soliris treatment. Real-world data underscore benefit of C5 inhibitors in NMOSD and gMG clinical practice A poster featuring real-world evidence from a global NMOSD registry will underscore the strong clinical benefit of Ultomiris and Soliris in relapse prevention in adults with AQP4-Ab+ NMOSD. Global gMG registry findings will be shared in poster presentations, including real-world data demonstrating reduced oral corticosteroid burden in patients with AChR-Ab+ gMG receiving Soliris or Ultomiris and reduced hospitalisations, MG crises and exacerbations among patients with AChR-Ab+ gMG receiving Ultomiris. Advancing understanding and innovation for the NMOSD and gMG communities An oral presentation will share results of a study evaluating glial fibrillary acidic protein (GFAP) and Neurofilament Light Chain (NfL), biomarkers of astrocyte and neuronal injury, respectively, as potential indicators of disease activity in patients with AQP4-Ab+ NMOSD. In this analysis, high baseline GFAP and NfL levels in PREVENT and CHAMPION-NMOSD serum samples were associated with clinical characteristics related to age and ambulation. Data will show that no associations were observed between levels of these biomarkers and relapse risk or time to first adjudicated on-trial relapse. A poster presentation will showcase preliminary results from Ad Scientiam’s decentralised research study, funded by Alexion, highlighting the potential clinical value of using the app-based tool, ME&MGopenTM, to collect digital data to track the daily impact of gMG symptoms in adults with AChR-Ab+ gMG, supporting its use in patient care. Two poster presentations will feature ongoing clinical studies, including the study design for the Phase III, open-label, single-arm trial evaluating Ultomiris in paediatric patients with AChR-Ab+ gMG.Additionally, an encore poster will provide an overview of the ongoing Phase III PREVAIL trial assessing the safety profile and efficacy of gefurulimab, a novel, dual-binding nanobody, optimised for weekly subcutaneous administration, in adult patients with AChR-Ab+ gMG. Further, results from Project ASPIRE (eliminAte diSparities and Promote equIty in Rare diseasE) will highlight health disparities among persons of colour (POC) who are living with gMG, underscoring the need for nationwide action plans to reduce barriers to healthcare and support for those who are disproportionally affected. Alexion presentations during AAN 2025 Lead Author Abstract Title Presentation Details Lead Author NMOSD Lead Author Abbatemarco J, et al. Abstract Title Patient Preferences for Treatment Features in Neuromyelitis Optica Spectrum Disorder (NMOSD): Results From a Discrete Choice Experiment (DCE) Presentation Details Poster Presentation Abstract #1752 7 April 2025 5:00 – 6:00PM PST Lead Author Okuda D, et al. Abstract Title Clinical and Radiological Outcomes in People with Aquaporin-4 IgG Positive Neuromyelitis Optica Spectrum Disorder Following Ravulizumab Treatment (AMAZE) Presentation Details Poster Presentation Abstract #4569 7 April 2025 5:00 – 6:00PM PST Lead Author Osborne B, et al. Abstract Title The Total Economic Impact of Anti-Aquaporin-4 Antibody-Positive (AQP4-Ab+) Neuromyelitis Optica Spectrum Disorder (NMOSD) on Patients and Caregivers in the United States Presentation Details Poster Presentation Abstract #1746 7 April 2025 5:00 – 6:00PM PST Lead Author Bennett J, et al. Abstract Title Time to vaccination after rituximab (RTX) discontinuation in patients with anti–aquaporin–4 antibody–positive (AQP4-Ab+) neuromyelitis optica spectrum disorder (NMOSD): A post-hoc analysis of the CHAMPION-NMOSD trial Presentation Details Poster Presentation Abstract #1445 9 April 2025 8:00 – 9:00AM PST Lead Author Obeidat A, et al. Abstract Title NMO SPOTLIGHT Registry: Real-World Clinical Outcomes With Eculizumab and Ravulizumab in Anti-Aquaporin-4 Antibody-Positive (AQP4-Ab+) Neuromyelitis Optica Spectrum Disorder (NMOSD) Presentation Details Poster Presentation Abstract Number #1837 9 April 2025 8:00 – 9:00AM PST Lead Author Wingerchuk D, et al. Abstract Title Clinical Characteristics Associated With High Baseline Glial Fibrillary Acidic Protein (GFAP) and Neurofilament Light Chain (NfL) Levels in Patients With Anti-Aquaporin-4 Antibody-Positive (AQP4-Ab+) Neuromyelitis Optica Spectrum Disorder (NMOSD) From the PREVENT and CHAMPION-NMOSD Trials Presentation Details Oral Presentation Abstract #1840 9 April 2025 1:36PM PST Lead Author Pittock S, et al. Abstract Title Efficacy and Safety of Ravulizumab in Adults With Anti-Aquaporin-4 Antibody-Positive (AQP4-Ab+) Neuromyelitis Optica Spectrum Disorder (NMOSD): Interim Analysis From the Ongoing Phase 3 CHAMPION-NMOSD Trial Presentation Details Oral Presentation Abstract #1733 9 April 2025 1:24PM PST Lead Author gMG Lead Author Narayanaswami P, et al. Abstract Title Effectiveness and Safety of Ravulizumab in Generalized Myasthenia Gravis (gMG): Updated Analysis From a Global Registry Presentation Details Poster Presentation Abstract #1829 5 April 2025 11:45AM – 12:45PM PST Lead Author Tandan R, et al. Abstract Title Assessment of Hospitalizations in Patients With Generalized Myasthenia Gravis (gMG) Before and During Treatment With Ravulizumab: Results From A Global Registry Presentation Details Poster Presentation Abstract #1832 5 April 2025 11:45AM – 12:45PM PST Lead Author Scheiner C, et al. Abstract Title Outcomes for Patients With Generalized Myasthenia Gravis Prescribed Ravulizumab or Efgartigimod Treatment: A Retrospective Medical Record Analysis Presentation Details Poster Presentation Abstract #1820 5 April 2025 11:45AM – 12:45PM PST Lead Author Govindarajan R Abstract Title Real-World Treatment Outcomes in Patients with Acetylcholine Receptor Antibody-Positive Generalized Myasthenia Gravis Who Transitioned from Efgartigimod to Ravulizumab Presentation Details Poster Presentation Abstract #4766 6 April 2025 5:00 – 6:00PM PST Lead Author Bhattacharyya S, et al. Abstract Title Characteristics of Cohort with Generalized Myasthenia Gravis in PREDICT Study Presentation Details Poster Presentation Abstract #5026 7 April 2025 11:45AM – 12:45PM PST Lead Author Niebler G, et al. Abstract Title A Phase 3, Open-Label, Single-Arm, Multicenter Study to Evaluate Ravulizumab Administered Intravenously in Pediatric Patients With Acetylcholine Receptor Antibody-Positive (AChR-Ab+) Generalized Myasthenia Gravis (gMG) Presentation Details Poster Presentation Abstract #1814 7 April 2025 5:00 – 6:00PM PST Lead Author Watanabe M, et al. Abstract Title Ravulizumab in Patients With Generalized Myasthenia Gravis (gMG) From Japan: Real-World Outcomes (Encore) Presentation Details Poster Presentation Abstract #1808 7 April 2025 5:00 – 6:00PM PST Lead Author Nowak R, et al. Abstract Title Change in concomitant immunosuppressive therapies for generalized myasthenia gravis in patients receiving complement C5 inhibitor therapies: a retrospective analysis of registry data (Encore) Presentation Details Poster Presentation Abstract #1838 7 April 2025 5:00 – 6:00PM PST Lead Author Howard J, et al. Abstract Title The phase 3 PREVAIL study assessing the efficacy and safety of subcutaneous gefurulimab in adults with generalized myasthenia gravis: trial in progress (Encore) Presentation Details Poster Presentation Abstract #1816 7 April 2025 5:00 – 6:00PM PST Lead Author Barnett-Tapia C, et al. Abstract Title Tracking Daily Impact of Generalized Myasthenia Gravis Using Smartphone-Based Digital Biomarkers: Lessons Learned from a 1-Year Study with ME&MGopenTM* Presentation Details Poster Presentation Abstract #3616 8 April 2025 11:45AM – 12:45PM PST Lead Author Gwathmey K, et al. Abstract Title Evaluation of the indirect and nonmedical impacts of generalized myasthenia gravis on patients and caregivers Presentation Details Poster Presentation Abstract #1800 8 April 2025 11:45AM – 12:45PM PST Lead Author Murai H, et al. Abstract Title Long-term Effectiveness and Safety of Eculizumab in Patients With Generalized Myasthenia Gravis (gMG): Real-World Data from Japan Presentation Details Oral Presentation Abstract #1835 9 April 2025 2:00 PM PST Lead Author Jiang N, et al. Abstract Title The path to diagnosis and treatment among patients with generalized myasthenia gravis (gMG): health disparities among persons of color from project ASPIRE (eliminAte diSparities and Promote equIty in Rare diseasE) Presentation Details Poster Presentation Abstract #1828 9 April 2025 11:45AM – 12:45PM PST Lead Author Across TAs Lead Author Yu J, et al. Abstract Title The REthinking MeAsures of DivErsity (REMADE) Study: Developing New Diversity Measures to Ensure Fair Representation in Clinical Trials (Encore) Presentation Details Poster Presentation Abstract #99 5 April 2025 11:45AM – 12:45PM PST *Ad Scientiam research study supported by Alexion Notes Alexion Alexion, AstraZeneca Rare Disease is focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and delivery of life-changing medicines. A pioneering leader in rare disease for more than three decades, Alexion was the first to translate the complex biology of the complement system into transformative medicines, and today it continues to build a diversified pipeline across disease areas with significant unmet need, using an array of innovative modalities. As part of AstraZeneca, Alexion is continually expanding its global geographic footprint to serve more rare disease patients around the world. It is headquartered in Boston, US. AstraZeneca AstraZeneca (LSE/STO/Nasdaq: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development, and commercialisation of prescription medicines in Oncology, Rare Diseases, and BioPharmaceuticals, including Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca’s innovative medicines are sold in more than 125 countries and used by millions of patients worldwide. Please visit astrazeneca.com and follow the Company on Social Media @AstraZeneca. Contacts For details on how to contact the Investor Relations Team, please click here. For Media contacts, click here. Corporate and financial

Phase 3Clinical Result

100 Deals associated with Eculizumab

External Link

KEGG	Wiki	ATC	Drug Bank
D03940	Eculizumab	L04AA25	DB01257

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
AQP4-IgG positive Neuromyelitis optica spectrum disorder	Australia	Alexion Pharmaceuticals, Inc.	20 Mar 2009
Atypical Hemolytic Uremic Syndrome	European Union	Alexion Europe SAS	20 Jun 2007
Atypical Hemolytic Uremic Syndrome	Iceland	Alexion Europe SAS	20 Jun 2007
Atypical Hemolytic Uremic Syndrome	Liechtenstein	Alexion Europe SAS	20 Jun 2007
Atypical Hemolytic Uremic Syndrome	Norway	Alexion Europe SAS	20 Jun 2007
Myasthenia Gravis	European Union	Alexion Europe SAS	20 Jun 2007
Myasthenia Gravis	Iceland	Alexion Europe SAS	20 Jun 2007
Myasthenia Gravis	Liechtenstein	Alexion Europe SAS	20 Jun 2007
Myasthenia Gravis	Norway	Alexion Europe SAS	20 Jun 2007
Neuromyelitis Optica	European Union	Alexion Europe SAS	20 Jun 2007
Neuromyelitis Optica	Iceland	Alexion Europe SAS	20 Jun 2007
Neuromyelitis Optica	Liechtenstein	Alexion Europe SAS	20 Jun 2007
Neuromyelitis Optica	Norway	Alexion Europe SAS	20 Jun 2007
Hemoglobinuria, Paroxysmal	United States	Alexion Pharmaceuticals, Inc.	16 Mar 2007

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Delayed Graft Function	Phase 3	United States	Alexion Pharmaceuticals, Inc.	21 Aug 2014
Delayed Graft Function	Phase 3	Australia	Alexion Pharmaceuticals, Inc.	21 Aug 2014
Delayed Graft Function	Phase 3	Brazil	Alexion Pharmaceuticals, Inc.	21 Aug 2014
Delayed Graft Function	Phase 3	Canada	Alexion Pharmaceuticals, Inc.	21 Aug 2014
Delayed Graft Function	Phase 3	Czechia	Alexion Pharmaceuticals, Inc.	21 Aug 2014
Delayed Graft Function	Phase 3	France	Alexion Pharmaceuticals, Inc.	21 Aug 2014
Delayed Graft Function	Phase 3	Germany	Alexion Pharmaceuticals, Inc.	21 Aug 2014
Delayed Graft Function	Phase 3	Italy	Alexion Pharmaceuticals, Inc.	21 Aug 2014
Delayed Graft Function	Phase 3	Spain	Alexion Pharmaceuticals, Inc.	21 Aug 2014
AQP4-IgG positive Optic Neuritis	Phase 3	United States	Alexion Pharmaceuticals, Inc.	11 Apr 2014

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04320602 (CTgov)	Phase 4	Hemoglobinuria, Paroxysmal	18	Eculizumab+Ravulizumab	mcjkwzgkwk = vxyltowdhu efdbswiatm (uvzhnbihmc, ejzbafgnjs - ezejvabhai) View more	-	12 Aug 2024
NCT01194973 (C10-004, FDA_CDER) Manual	Phase 2	Atypical Hemolytic Uremic Syndrome	41	Eculizumab	ckaeggxeqx(wnmkhhzpvi) = zktdqyvbak sqjkzrixqf (vsnjvmvztc, 40 - 72) View more	Positive	19 Jul 2024
NCT00844844 \| NCT00844545 (C08-002B, FDA_CDER) Manual	Phase 2	Atypical Hemolytic Uremic Syndrome	17	Eculizumab (at 26 wks)	tqdsbmeqxd(vcdwagqgru) = tbgfgcyliy kvevcosmms (dgyzfsctqw ) View more	Positive	19 Jul 2024
				Eculizumab (at 2 yrs)	tqdsbmeqxd(vcdwagqgru) = mrzlzhqynh kvevcosmms (dgyzfsctqw ) View more
NCT00844428 \| NCT00838513 (C08-003B, FDA_CDER) Manual	Phase 2	Atypical Hemolytic Uremic Syndrome	20	Eculizumab (at 26 wks)	jaqcerrssr(pzikocjnzd) = gaztmhcajp juqzvrzvju (jysrrtyaql ) View more	Positive	19 Jul 2024
				Eculizumab (at 2 yrs)	jaqcerrssr(pzikocjnzd) = vsijmmmwhu juqzvrzvju (jysrrtyaql ) View more
NCT01193348 (C10-003, FDA_CDER) Manual	Phase 2	Atypical Hemolytic Uremic Syndrome	22	Eculizumab (Patients 1 month to <12 years)	gtsvqenwkz(kzthhbxqfd) = urovituklg hgyisgfjom (smguwbuvee, 36 - 83) View more	Positive	19 Jul 2024
				Eculizumab (All Patients)	gtsvqenwkz(kzthhbxqfd) = yhsntodhax hgyisgfjom (smguwbuvee, 41 - 83) View more
NCT01770951 (C09-001r, FDA_CDER) Manual	Not Applicable	Atypical Hemolytic Uremic Syndrome	19	Eculizumab (<2 yrs)	fqwnxpevcs(hmjhtcofpk) = wfntjjzsod gyuufvhdan (nfaozokyri ) View more	Positive	19 Jul 2024
				Eculizumab (2 to <12 yrs)	fqwnxpevcs(hmjhtcofpk) = awnwtkhfvb gyuufvhdan (nfaozokyri ) View more
NCT00122330 (FDA_CDER) Manual	Phase 3	Hemoglobinuria, Paroxysmal	87	Placebo	lbqibvmrto(rikcokxijq) = tqqgdcxgom reyfzftjof (kfvwnhmbtz ) View more	Positive	19 Jul 2024
				Eculizumab	lbqibvmrto(rikcokxijq) = yamcvjglxe reyfzftjof (kfvwnhmbtz ) View more
NCT03500549 (PEGASUS, EHA2024) Manual	Phase 3	Hemoglobinuria, Paroxysmal	68	pegcetacoplaneculizumab	-	Positive	14 May 2024
				pegcetacoplan +eculizumab (patients with baseline IO)	umdtsshaoi(durhpjcsjr) = ehdkqhkxvn woertjfilw (nadmtpbpvx, 8.0) View more
EHA2024	Not Applicable	Hemoglobinuria, Paroxysmal	200	Eculizumab	ylnwedlrvw(zzjwyntdsb) = mhgkcmkpjd suiiqwdhqf (mihgvgrynt ) View more	Positive	14 May 2024
				Ravulizumab	ylnwedlrvw(zzjwyntdsb) = wcijvcrokg suiiqwdhqf (mihgvgrynt ) View more
ISN WCN2024	Not Applicable	Atypical Hemolytic Uremic Syndrome genetic mutations in the alternate complement pathway	6	Eculizumab sparing protocols	uwycwhkfts(cgjnjseuhv) = None had BK virus infection jxnccdcyfb (mhlcbdvrab ) View more	Positive	01 Apr 2024

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Biosimilar

Competitive landscape of biosimilars in different countries/locations. Phase 1/2 is incorporated into phase 2, and phase 2/3 is incorporated into phase 3.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

Chat with Hiro

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis