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.

NCT07010302

/ Not yet recruitingPhase 4IIT

A Comparative Clinical Effectiveness Trial of Rituximab Versus Ravulizumab, Inebilizumab, Satralizumab and Eculizumab To Prevent Relapses in Neuromyelitis Optica Spectrum Disorder

Neuromyelitis Optica Spectrum Disorder (NMOSD) is a rare autoimmune condition that mainly affects the eyes and spinal cord, causing serious symptoms such as vision loss, paralysis, and severe pain. This trial compares the effectiveness and safety of five medications commonly used to prevent NMOSD relapses: rituximab, ravulizumab, inebilizumab, satralizumab, and eculizumab.
In this study, 160 adults with NMOSD who test positive for a specific antibody (AQP4-IgG) will participate. They will be randomly assigned to receive either rituximab or one of the four other FDA-approved medications. The main goal is to find out which treatment best prevents relapses and has fewer serious side effects. The trial will also measure disability, patient satisfaction, quality of life, and biomarkers that help track disease activity.
Participants will have regular assessments, including medical exams, surveys, and tests for vision, walking ability, and brain function. They will report any side effects or health issues experienced during the study. The trial will last from one to four years for each participant.
This research aims to help patients and doctors make better-informed treatment decisions by providing clear evidence about the best available therapies for NMOSD.

Start Date01 Nov 2025

Sponsor / Collaborator

The Massachusetts General Hospital

[+1]

NCT06673394

/ WithdrawnPhase 2IIT

Eculizumab For Acute Attack of Neuromyelitis Optica Spectrum Disorder (NMOSD): a Multi-Center, Phase 2, Open Label Trial (EASE-NMO)

Neuromyelitis optica spectrum disorder (NMOSD) is a relapsing, inflammatory autoimmune disorder of the central nervous system characterized by the pathogenic anti-aquaporin 4 antibody (AQP4-IgG). The objectives of this study are to assess the efficacy and safety of eculizumab for treatment of patients with neuromyelitis optica spectrum disorders during acute phase who are anti-aquaporin-4 (AQP4) antibody-positive. Eculizumab, a humanized monoclonal antibody, inhibits the terminal complement protein C5 and prevents its cleavage into C5a and the formation of C5b-9 (MAC), has approved for preventive treatment of NMOSD. Given the high efficacy of C5 inhibition, eculizumab is proposed to potentially provide rapid relief from astrocyte destruction by reducing MAC formation, which could contribute to the fast alleviation of neurological deficit during NMO acute attack. The potential of eculizumab warrants further investigation as a treatment for acute neuromyelitis optica spectrum disorders attacks.

Start Date03 Sep 2025

Sponsor / Collaborator

Tianjin Medical University General Hospital

100 Clinical Results associated with Eculizumab

100 Translational Medicine associated with Eculizumab

100 Patents (Medical) associated with Eculizumab

2,702

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 ; Liu, Ziwei ; Han, Bing ; 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 Nov 2025·JOURNAL OF HYPERTENSION

Malignant hypertension as a presentation of complement-mediated thrombotic microangiopathy: case series report

Article

Author: Ma, Chengjun ; Li, Kun ; Gao, Yanxia ; Zhang, Xiaohui ; Bi, Wenhua

Complement-mediated thrombotic microangiopathy (TMA) presenting as malignant hypertension (mHTN) is frequently misdiagnosed as mHTN-associated TMA, particularly when their clinical presentations overlap. The diagnostic challenge is further compounded when patients have normal serum levels of complement factor H (CFH) and test negative for anti-CFH. We presented two patients of complement-mediated TMA exhibiting clinical features consistent with mHTN-associated TMA, yet their renal function did not improve following the achievement of target blood pressure (BP) levels. Notably, both patients demonstrated elevated serum soluble C5b-9 levels. Based on these findings, we hypothesized the presence of complement-mediated TMA and initiated treatment with eculizumab. Consequently, one patient was successfully weaned off dialysis, and the other experienced substantial improvement in renal function, highlighting that in patients with mHTN-associated TMA where antihypertensive treatment fails to ameliorate renal function, consideration should be given to the possibility of complement-mediated TMA.

14 Oct 2025·Blood Advances

Characterizing clinically significant extravascular hemolysis in adults with PNH on ravulizumab or eculizumab treatment

Article

Author: Peffault de Latour, Régis ; Brodsky, Robert A. ; Nishimura, Jun-ichi ; Patriquin, Christopher J. ; Barcellini, Wilma ; Kulasekararaj, Austin G. ; Yu, Ji ; Piatek, Caroline I. ; Lee, Jong Wook ; Mahdi, Mohammed ; Röth, Alexander

Abstract:

In patients with paroxysmal nocturnal hemoglobinuria (PNH), the complement component 5 (C5) inhibitors ravulizumab and eculizumab control terminal complement activity and intravascular hemolysis, which are drivers of morbidity and mortality. During C5 inhibitor treatment, ongoing C3 fragment deposition on surviving PNH red blood cells may cause clinically significant extravascular hemolysis (csEVH) in some patients. csEVH is not thought to be life threatening but may cause symptomatic anemia and need for transfusion. This post hoc analysis of studies 301 (NCT02946463) and 302 (NCT03056040) evaluated the prevalence of csEVH (symptomatic anemia [hemoglobin levels <9.5 g/dL] with absolute reticulocyte count ≥120 × 109/L) in adult patients with PNH treated with ravulizumab or eculizumab for 6 months (183 days). The association between csEVH and fatigue (measured using the Functional Assessment of Chronic Illness Therapy - Fatigue [FACIT-F] scale) was also evaluated in study 302 patients with stable disease. On day 183 of studies 301 and 302, csEVH prevalence was 23.2% (29/125) and 20.2% (19/94) with ravulizumab and 24.8% (30/121) and 21.3% (20/94) with eculizumab, respectively. All patients in study 302 with csEVH experienced fatigue, which was mostly mild (ravulizumab, 79%; eculizumab, 65%) or moderate (ravulizumab, 21%; eculizumab, 30%). FACIT-F scores remained stable from baseline (ravulizumab, 42.2; eculizumab, 38.3) to day 183 (ravulizumab, 44.3; eculizumab, 38.3), close to general population normative values. This analysis demonstrated that csEVH affected 20% to 25% of patients with PNH, most of whom experienced mild fatigue, with fatigue remaining stable during treatment. Ravulizumab and eculizumab continue to provide benefit to adults with PNH with csEVH.

264

News (Medical) associated with Eculizumab

19 Sep 2025

·www.astrazeneca.com

Alexion data presented at ECTRIMS 2025 reinforces Ultomiris leadership in transforming care for patients with NMOSD

Final results of CHAMPION-NMOSD Phase III trial long-term extension will show zero relapses in patients on Ultomiris through a median follow-up of 170.3 weeks Phase III data selected as one of ‘Best of ECTRIMS 2025’, reinforcing its significance in advancing therapeutic standards in NMOSD Alexion, AstraZeneca Rare Disease, will present four abstracts, including one oral presentation, from its leading rare neurology portfolio at the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) Annual Congress in Barcelona, Spain, 24 to 26 September 2025. Presentations will highlight the final long-term analysis of the CHAMPION-NMOSD Phase III trial as well as real-world data further supporting the safety profiles and efficacy of Ultomiris (ravulizumab) and Soliris (eculizumab) in adult patients with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder (NMOSD). Christophe Hotermans, Head of Global Medical Affairs, Alexion, said: “At ECTRIMS, new long-term clinical and real-world evidence will reinforce the potential of Ultomiris to eliminate relapses in most patients and elevate the standard of care for people living with AQP4-Ab+ NMOSD. From the final results of the CHAMPION-NMOSD Phase III trial to global, real-world Ultomiris and Soliris data, these presentations highlight Alexion’s continued commitment to reducing the burden of this debilitating disease and our aspiration to revolutionise what living with NMOSD can look like.” Sean Pittock, MD, Mayo Clinic Glenn W. and Katherine K. Hasse Chair Neurology, Applebaum Family Professor of Neuroscience, Professor of Neurology, Co-Director of the Neuroimmunology Laboratory and principal investigator in the CHAMPION-NMOSD Phase III trial, said: “These long-term data from the CHAMPION-NMOSD Phase III trial show that patients treated with Ultomiris remained relapse-free for more than three years, a significant achievement in a disease where every relapse can lead to permanent disability. It’s an honour to have these findings recognised as part of the ‘Best of ECTRIMS 2025’, underscoring the potential impact of these results in improving outcomes for patients with NMOSD.” Completion of CHAMPION-NMOSD Phase III trial long-term extension shows maintained relapse prevention with Ultomiris An oral presentation of the final safety and efficacy results from the long-term extension (LTE) of the global CHAMPION-NMOSD Phase III trial will demonstrate zero adjudicated on-trial relapses (OTR) in adults with AQP4-Ab+ NMOSD treated with Ultomiris through the median follow-up of 170.3 weeks (relapse risk reduction: 98.9%, hazard ratio (95% CI), 91.8-100; P < 0.0001). As one of the longest studies conducted in NMOSD, these data will reinforce the long-term clinical benefit of C5 inhibition with Ultomiris. Additionally, most patients treated with Ultomiris were clinically stable (81%) or improved (13.8%), as measured by the Hauser Ambulation Index (HAI) score, and had no clinically important worsening (91.4%) of disability measured with the Expanded Disability Status Scale (EDSS). Further, the data will show that 63% (17/27) of patients taking immunosuppressive therapy (IST) at baseline reduced their dose or discontinued at least one treatment, indicating a potential for reduced use IST while being treated with Ultomiris long-term. The safety profile was consistent with prior study analyses. Robust real-world evidence builds on the established benefits of C5 inhibition in NMOSD A virtual poster presentation will feature real-world clinical findings from the global NMO SPOTLIGHT Registry, showing that Ultomiris and Soliris led to a reduction in relapses for people with AQP4-Ab+ NMOSD. Among 56 adults, the annualised relapse rate (ARR) fell from 0.50 [95% CI: 0.33-0.72] during the year prior to starting treatment with Ultomiris or Soliris to 0.02 [95% CI: 0.00-0.05] while on treatment, with no one experiencing more than one relapse. Among patients who switched from rituximab (n=15), no relapses were reported while treated with Ultomiris or Soliris, compared to an ARR of 0.47 [95% CI: 0.19-0.96] in the year prior to switching. These findings highlight the strong real-world clinical benefit of Ultomiris and Soliris in relapse prevention. A separate poster presentation will share interim analysis results from a real-world study from Japan evaluating Ultomiris in patients with AQP4-Ab+ NMOSD who had a suboptimal response to satralizumab. Among 11 patients receiving Ultomiris for at least six months (10.9±1.4 months on average), 10 remained relapse-free. In addition, use of concomitant immunosuppressive drugs was reduced in all patients. The switch to Ultomiris also showed positive changes in inflammatory biomarkers, as measured by changes in CD19+CD27-IgD- double negative B cells (%DNB) and unswitched memory B cells (%USM) at two months follow-up after treatment initiation. Lastly, a virtual poster presentation will share findings from the ongoing AMAZE study, a 78-week observational trial to evaluate the biological effects of Ultomiris, on clinical, serological and radiological measures of disease, and better understand the impact of social determinants of health on access to care and treatment outcomes for living with AQP4-Ab+ NMOSD. Upon study completion, these data may help guide clinicians towards optimised and equitable healthcare practices for people living with AQP4-Ab+ NMOSD. Lead Author Abstract Title Presentation Details Lead Author Pittock, SJ Abstract Title Long-term efficacy and safety of ravulizumab in anti-aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder: final analysis of the phase 3 CHAMPION-NMOSD trial Presentation Details Oral Presentation Abstract ID: O109 26 September 2025 9:05 – 9:12 CEST Lead Author Sotirchos, ES Abstract Title Real-world clinical outcomes with eculizumab and ravulizumab in anti-aquaporin-4 antibody-positive (AQP4-Ab+) neuromyelitis optica spectrum disorder (NMOSD): results from the global NMO SPOTLIGHT Registry Presentation Details ePoster Presentation Abstract ID: P1749 Lead Author Sato, W Abstract Title A real-world study of the effectiveness and safety of ravulizumab in AQP4+ NMOSD patients with suboptimal response to satralizumab in Japan - interim analysis- Presentation Details Poster Presentation Abstract ID: P867 Lead Author Okuda, D Abstract Title Clinical and Radiological Outcomes in People with Aquaporin-4 Antibody Positive Neuromyelitis Optica Spectrum Disorder Following Ravulizumab Treatment (AMAZE) Presentation Details ePoster Presentation Abstract ID: P1058 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. tags Corporate and financial

Phase 3Clinical Result

15 Sep 2025

·www.biospace.com

Rush of Targeted Myasthenia Gravis Therapies Hit Once Sparse Market

iStock, quantic69 After decades without much movement, a handful of new treatments for this rare autoimmune disease are now approved, and several companies, including argenx and Regeneron, have recently released promising late-stage trial results. A new generation of drugs is revolutionizing the treatment of myasthenia gravis, a chronic, debilitating autoimmune condition in which communication breaks down along the neuromuscular junctions that bridge the signals between the brain and muscles. In less than a decade, the number of myasthenia gravis (MG)-specific therapies available to patients has bloomed, sparking double-digit growth in the global market and prompting analysts to project revenues exceeding $10 billion by 2033. Now, argenx, UCB, Amgen , Regeneron and more are vying for a share of this niche market. “Until recently, we hadn’t seen a newly approved MG therapy in many decades,” Tom Ragole, a neuromuscular neurologist at the UCHealth Neurosciences Center in Colorado, told BioSpace . But in the past eight years, starting with AstraZeneca’s Soliris , five new MG-specific therapies have hit the market, and today, several groups are launching late-stage clinical trials—so many that it has sometimes been hard to recruit enough patients for them all. “Part of it is that being on a therapy often excludes you from other trials, but it’s also just that you need a certain disease burden, and we have so many options now for treating people that they no longer qualify.” While myasthenia gravis is among the most commonly diagnosed conditions affecting neuromuscular junctions, it remains a rare disease, afflicting roughly 37 out of every 100,000 people in the U.S. Early symptoms often manifest in the head—including drooping eyelids, double vision and facial weakness—but can eventually expand to other parts of the body in a form known as generalized myasthenia gravis (gMG). The underlying causes of MG remain murky, and there is no cure, but researchers are beginning to understand more about the disease’s drivers. Broadly, MG is mediated by a class of abnormal IgG antibodies that target receptors or proteins in the neuromuscular junction, most commonly the acetylcholine receptor (AChR), muscle-specific kinase and lipoprotein receptor-related protein 4. Patients can be positive for some, but not all, of these antibodies, or negative for all three, suggesting additional targets that researchers have yet to identify. Ragole said treatments have historically pulled from the toolkit leveraged for many autoimmune disorders: corticosteroids, immunosuppressants and plasma exchanges. But while such treatments can be effective, they remain the equivalent of a Band-Aid, according to Ragole, and it’s unclear whether steroid-based therapies have long-term side effects. Other fields, notably rheumatology, have already moved away from high-dose steroids and toward more targeted therapies, Ragole said, and he’s been pleased to see the surge in interest for myasthenia gravis drugs that do the same. “There’s definitely been a lot of movement on the front of these more targeted therapies,” he said. “MG is a disease that varies so much from patient to patient. The more options we have, the better I see things going moving forward.” The New Guard This new generation of drugs draws on scientists’ growing understanding of myasthenia gravis, targeting one of two late-stage pathways in the disease’s progression: the complement system—part of the innate immune system—and neonatal Fc receptor (FcRn) signaling. In 2017, the FDA approved Soliris as the first MG-specific therapy. This so-called C5 complement inhibitor stops destruction of muscle receptors in the neuromuscular junction. A few years later, in 2021, immunology-focused argenx followed with a FcRn-targeted therapy called Vyvgart that degrades disease-causing antibodies. Three more approvals have since followed, including UCB’s C5 complement inhibitor Zilbrysq and FcRn therapy Rystiggo in 2023 and J&J’s Imaavy earlier this year. Despite the surge in MG approvals, research is not slowing down. In August, argenx reported positive results from a Phase III trial testing Vygart’s effectiveness in patients lacking the AChR antibody. According to the company, this population accounts for roughly 20% of patients with gMG, and they often experience a more severe disease burden. Gaining approval to treat this group would open the drug up to a further 11,000 patients, analysts at William Blair said in an Aug. 25 note. Also in August, Regeneron released Phase III results for its RNA-based candidate cemdisiran in patients with AChR-positive gMG. The study met its primary and secondary endpoints while only inhibiting the complement system by 74%, suggesting that patients could improve their quality of life without completely shutting down complement, “which otherwise is very important in defending us from infections,” Andres Sirulnik, head of the Hematology Clinical Development unit at Regeneron, told BioSpace. Several features of cemdisiran “represent improvements over existing MG therapies” that Regeneron hopes will push the drug candidate past its competitors, Sirulnik said. For example, existing treatments are dosed monthly or even daily, while cemdisiran is dosed via an injection that lasts for 12 weeks. That is something that patients will “absolutely” value, Ragole said. The Future: Near and Long-Term Despite the surge of activity in the MG space, experts agree that there’s still plenty of room for discovery and innovation. “There is still a high unmet need for patients in this disease,” Sirulnik said, adding that “a multipronged approach” is required. Long term, Ragole said it’s worth looking to other fields, such as cancer, that are increasingly leaning into cell-based therapies and precision medicine to further broaden options for patients. Treatments that leverage CD19+ B cells and CAR T cells have already been approved, as have synthetic, bispecific antibodies that bind to multiple targets simultaneously. “There’s increasing data that the types of breakthroughs we’ve seen in oncology are likely to be effective in some of these autoimmune disorders as well,” Ragole said. Near term, Argenx plans to supplemental application to expand Vyvgart into patients with triple-negative MG by the end of 2025. If successful, Vyvgart would enjoy the broadest label of all FcRn antagonists approved so far. Regeneron, meanwhile, will be submitting for FDA approval of cemdisiran in the first quarter of 2026. Omar Sinno , U.S. medical strategy lead for rare disease at UCB, said his company is also gearing up to share new updates. UCB continues to publish long-term safety and efficacy data on Zilbrysq and Rystiggo, including 10 abstracts to be presented at the upcoming American Association of Neuromuscular & Electrodiagnostic Medicine meeting , Oct. 29 to Nov. 1 in San Francisco. According to Sinno, UCB remains the only company offering two therapies that target different pathways, including the only complement inhibitor that can be used in conjunction with an FcRN therapy, allowing flexible treatment plans. Given the increasingly crowded treatment landscape, UCB is also partnering with the Myasthenia Gravis Foundation of America on a set of resources to help patients better understand and communicate their needs. “The thing about MG is that every case is actually different, and so having this breadth of treatments is good, but we need patients to understand their options,” Sinno told BioSpace . “Fortunately, patients are asking a lot more questions and advocating for themselves.”

Clinical ResultPhase 3Drug Approval

09 Sep 2025

·pharma.economictimes.indiatimes.com

AstraZeneca launches rare blood, kidney disease drug Eculizumab in India

New Delhi: UK Pharma major AstraZeneca India arm has announced the launch of its C-5 inhibitor, Eculizumab in India The drug is approved by the CDSCO for the treatment of rare, life-threatening disease Paroxysmal Nocturnal Hemoglobinuria (PNH) and atypical Hemolytic Uremic Syndrome (aHUS) in both adults and children. In India the pharma giant India arm has secured approval for the import, sale, and distribution of the product. Eculizumab, a novel monoclonal antibody developed by Alexion Pharmaceuticals, Inc, later became part of the UK major portfolio following its acquisition of Alexion for around $39 billion in 2021. The drug is designed to block the C5 protein which damages blood cells and kidney tissues in conditions like PNH, aHUS. “The launch of Eculizumab as India’s first approved anti-complement therapy demonstrates our dedication to delivering innovative medicines at the earliest opportunity, especially where there is significant unmet medical need,” said Praveen Akkinepally, Country President and Managing Director, AstraZeneca India. In the US the drug is marketed under the brand name ‘ Soliris ’ and has been accused of evergreening the patent with minor increments in the country. The therapy compositions are set to lose their patent protection by 2027. According to AstraZeneca 2024 annual sales, Soliris (Eculizumab) is the company’s second leading rare disease asset with net world wide sales of $2.58 billion (~around Rs 22,000 crore). By Online Bureau ,

Drug ApprovalAcquisitionPatent Expiration

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


NCT05966467 (NMO SPOTLIGHT Registry, ECTRIMS2025 \| Company_Website) Manual	Not Applicable	AQP4-IgG positive Neuromyelitis optica spectrum disorder AQP4-IgG	56	ALXN-C5IT (eculizumab or ravulizumab)	mjppjysyue(vbqhexostf) = kamzmtyeee wncngrjrcl (jctqqxcqqr, 0.00 - 0.05) View more	Positive	09 Sep 2025
				ALXN-C5IT (eculizumab or ravulizumab) (switched from rituximab)	mjppjysyue(vbqhexostf) = gcgbajimth wncngrjrcl (jctqqxcqqr ) View more
PB2827 (EHA2025)	Not Applicable	Hemoglobinuria, Paroxysmal	52	Eculizumab	erwszzdvdt(bvqakouakm) = occurred in 30.8% cnaurrumpq (wodfhykyfl ) View more	-	14 May 2025
S34.006 (AAN2025)	Not Applicable	Myasthenia Gravis anti-acetylcholine receptor antibody-positive (AChR-Ab+)	254	Eculizumab	sulxgrspcz(xtouoygynq) = 40.5% experienced adverse drug reactions (ADRs) nlvickusyr (ovooubchze ) View more	Positive	07 Apr 2025
NCT05966467 (NMO SPOTLIGHT Registry, AAN2025)	Not Applicable	AQP4-IgG positive Neuromyelitis optica spectrum disorder	30	Eculizumab	uhuijuboks(rdlzsxestb) = prhpnllzfj ybwdgfjywc (qoctbfmbgl, 24.0 - 49.7)	Positive	07 Apr 2025
				Ravulizumab	uhuijuboks(rdlzsxestb) = desxnzchuo ybwdgfjywc (qoctbfmbgl, 0.7 - 1.3)
ASH2024	Not Applicable	Atypical Hemolytic Uremic Syndrome	-	Eculizumab	nylcatrhwi(cixvwitwmo) = tlysiuuuzg kewbvzzeee (vuewzlznyt, 230.0 - 362.0) View more	-	08 Dec 2024
				Ravulizumab	toilwxsvqm(kzikvfrxdo) = bvlugrghgp qwdavhugoh (irmrobpuhe, 11.5% - 36.4%)
SOHO2024	Not Applicable	Hemoglobinuria, Paroxysmal	-	Eculizumab	snitsajvov(qazulwctem) = sleznrtrly tkxutyvtpp (rknybfvbvb ) View more	-	04 Sep 2024
				Ravulizumab	snitsajvov(qazulwctem) = feqwffcoml tkxutyvtpp (rknybfvbvb ) View more
NCT04320602 (CTgov)	Phase 4	Hemoglobinuria, Paroxysmal	18	Eculizumab+Ravulizumab	rloqfdjftb = zbejhgqlpo bzxlkcepzt (yxjraybdom, cgibevlnyv - dqbmxlcswe) View more	-	12 Aug 2024
NCT01770951 (C09-001r, FDA_CDER) Manual	Not Applicable	Atypical Hemolytic Uremic Syndrome	19	Eculizumab (<2 yrs)	vhlokcljig(oxlqmspnny) = ljxduqcqsy hbrjrmrtgd (udphsbtucz ) View more	Positive	19 Jul 2024
				Eculizumab (2 to <12 yrs)	vhlokcljig(oxlqmspnny) = hagdcyiwoj hbrjrmrtgd (udphsbtucz ) View more
NCT00844844 \| NCT00844545 (C08-002B, FDA_CDER) Manual	Phase 2	Atypical Hemolytic Uremic Syndrome	17	Eculizumab (at 26 wks)	ygabgpvtnh(fgpzkcchxg) = ahhptmoald nyzqgpovgo (oxykhpqbci ) View more	Positive	19 Jul 2024
				Eculizumab (at 2 yrs)	ygabgpvtnh(fgpzkcchxg) = hwcurzjgjn nyzqgpovgo (oxykhpqbci ) View more
NCT01193348 (C10-003, FDA_CDER) Manual	Phase 2	Atypical Hemolytic Uremic Syndrome	22	Eculizumab (Patients 1 month to <12 years)	tzhoftvwag(flowusijqe) = yjxapkczrr yoftzcztci (iszsqoynts, 36 - 83) View more	Positive	19 Jul 2024
				Eculizumab (All Patients)	tzhoftvwag(flowusijqe) = dzvbknjoxv yoftzcztci (iszsqoynts, 41 - 83) View more

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