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.

开始日期2026-01-01

申办/合作机构

NovelMed Therapeutics, Inc.

NCT06724809

/ Recruiting临床3期

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).

开始日期2025-01-16

申办/合作机构

Alexion Pharmaceuticals, Inc.

[+1]

NCT06764160

/ Recruiting临床3期

An Open-label, Single-arm, Multi-center Study to Evaluate the Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Eculizumab in Chinese Participants With Refractory Generalized Myasthenia Gravis (gMG)

This is an open-label, single-arm, multi-center study to evaluate the efficacy, safety, PK, PD, and immunogenicity of eculizumab in Chinese participants with refractory gMG. Approximately 15 participants will be enrolled in the study.

开始日期2025-01-06

申办/合作机构

Alexion Pharmaceuticals, Inc.

100 项与依库珠单抗相关的临床结果

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100 项与依库珠单抗相关的转化医学

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100 项与依库珠单抗相关的专利（医药）

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2,711

项与依库珠单抗相关的文献（医药）

2025-12-31·Hematology

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

Article

作者: 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.

2025-06-01·JOURNAL OF PHARMACEUTICAL AND BIOMEDICAL ANALYSIS

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

Article

作者: 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.

2025-06-01·JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY

Real-world multicentre cohort study on choices and effectiveness of immunotherapies in NMOSD and MOGAD

Article

作者: Pompsch, Mosche ; Dawin, Eva ; Senel, Makbule ; Klotz, Luisa ; Kleiter, Ingo ; Hümmert, Martin W ; Wickel, Jonathan ; Herfurth, Mariella ; Jarius, Sven ; Korporal-Kuhnke, Mirjam ; Tumani, Hayrettin ; Fischer, Katinka ; Schiffmann, Insa ; Schindler, Patrick ; Aktas, Orhan ; Schwake, Carolin ; Berthele, Achim ; Giglhuber, Katrin ; Rommer, Paulus Stefan ; Lindenblatt, Gero ; Trebst, Corinna ; Grothe, Matthias ; Stellmann, Jan-Patrick ; Kümpfel, Tania ; Sieb, Jörn Peter ; Friede, Tim ; Ayzenberg, Ilya ; Angstwurm, Klemens ; Duchow, Ankelien ; Pakeerathan, Thivya ; Then Bergh, Florian ; Kaste, Matthias ; Havla, Joachim ; Walter, Annette ; Krämer, Markus ; Ringelstein, Marius ; Bellmann-Strobl, Judith ; Wildemann, Brigitte ; Häußler, Vivien ; Bütow, Franziska ; Flaskamp, Martina ; Paul, Friedemann ; Pellkofer, Hanna ; Tkachenko, Daria ; Revie, Lisa ; Engels, Daniel

Background:

Recurrent attacks in neuromyelitis optica spectrum disorders (NMOSDs) or myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD) can lead to severe disability. We aimed to analyse the real-world use of immunotherapies in patients with NMOSD and MOGAD, focusing on changes in treatment strategies, effects on attack rates (ARR) and risk factors for attacks.

Methods:

This longitudinal registry-based cohort study included 493 patients (320 with aquaporin-4 immunoglobulin G (AQP4-IgG) seropositive NMOSD (65%), 44 with AQP4-IgG seronegative NMOSD (9%) and 129 MOGAD (26%)) with 1247 treatments from 19 German and one Austrian centre from the registry of the neuromyelitis optica study group (NEMOS). We analysed unadjusted ARR and implemented survival analyses and Cox proportional hazard regression to assess efficiency and risk factors for subsequent attacks over time.

Results:

Rituximab and azathioprine are the most widely used immunotherapies in NMOSD as well as in MOGAD, with changes in distribution over the last decade. Immunotherapy demonstrated significant therapeutic effects in NMOSD but less pronounced effects in MOGAD. Risk factors for attacks included younger age and prior attacks under the same therapy. Efficacy varied among the different immunotherapies, with azathioprine, rituximab and eculizumab showing significant risk reductions in AQP4-IgG seropositive NMOSD.

Conclusions:

This study provides insights into the evolving treatment landscape and effectiveness of immunotherapies in NMOSD and MOGAD. Established off-label therapies continue to play an important role, especially for patients with stable disease, with emerging evidence supporting newly approved therapies. Future studies are needed to refine treatment algorithms and address the ongoing uncertainties in MOGAD management.

467

项与依库珠单抗相关的新闻（医药）

2025-05-22

·PRNewswire

Johnson & Johnson's IMAAVY Gets FDA Approval, Taking on Top Players in Myasthenia Gravis Market | DelveInsight

The FDA has approved Johnson & Johnson's anti-FcRn antibody, nipocalimab, for the treatment of generalized myasthenia gravis. The drug will be sold under the brand name IMAAVY. This approval positions J&J to challenge leading competitors in the myasthenia gravis market, such as AstraZeneca, which markets the monoclonal antibodies SOLIRIS and ULTOMIRIS. J&J will also be competing with Argenx's VYVGART and UCB's RYSTIGGO and ZILBRYSQ. LAS VEGAS, May 22, 2025 /PRNewswire/ -- Myasthenia gravis is an autoimmune disorder in which antibodies disrupt the communication between nerves and muscles, resulting in skeletal muscle weakness. This weakness primarily affects voluntary muscles that control eye movement, facial expression, swallowing, and limb function. The condition is predominantly driven by autoantibodies targeting either the acetylcholine receptor (AChR-Ab) or a receptor-associated protein known as muscle-specific tyrosine kinase (MuSK-Ab). According to DelveInsight's analysis, there were approximately 300,000 diagnosed prevalent cases of myasthenia gravis across the 7MM in 2024. This number is expected to grow steadily over the forecast period (2025–2034), with a projected CAGR. The current myasthenia gravis therapeutics market mainly consists of symptomatic treatments such as acetylcholinesterase inhibitors like Mestinon. However, achieving full clinical remission with symptomatic therapy alone is challenging. As a result, immunosuppressive drugs are frequently introduced. First-line immunotherapy typically involves high-dose oral corticosteroids such as prednisone or prednisolone, which can induce a rapid response within 2 to 4 weeks. To reduce reliance on steroids and improve long-term disease control, nonsteroidal immunosuppressants (NSISTs) are often used. These include antimetabolites (e.g., azathioprine, mycophenolate mofetil, methotrexate, and cyclophosphamide) and calcineurin inhibitors (e.g., tacrolimus and cyclosporine). While NSISTs may be used as standalone treatments, they usually take 6 to 9 months to produce noticeable effects. Immunomodulatory therapies, including intravenous immunoglobulin (IVIg) and plasmapheresis (PLEX), are typically reserved for acute exacerbations or myasthenic crises. They are also employed as chronic treatments in patients who cannot tolerate or do not respond to standard immunosuppressants. Although traditional immunosuppressive strategies can help manage symptoms, achieving long-term, stable remission remains difficult. Additionally, about 15% of patients are considered treatment-refractory, meaning they either fail to respond to or cannot tolerate multiple immunosuppressive options. These patients often face the dual challenge of persistent symptoms and the adverse effects of long-term immunosuppression. Learn more about the myasthenia gravis disease market @ Myasthenia Gravis Treatment Medications The myasthenia gravis treatment market is well-established, with multiple approved drugs for myasthenia gravis, including SOLIRIS, ULTOMIRIS, RYSTIGGO, ZILBRYSQ, and VYVGART, among others. Alexion Pharmaceuticals' SOLIRIS is a monoclonal antibody that targets and inhibits complement protein C5, thereby blocking the formation of the terminal complement complex. This action helps reduce complement-driven hemolysis in PNH and thrombotic microangiopathy in aHUS. Although its exact mechanism in gMG and NMOSD is not fully known, it is believed to decrease complement complex accumulation. Between 2017 and 2023, SOLIRIS gained regulatory approvals in Japan, the US, and the EU for treating gMG, including pediatric indications in Japan. ULTOMIRIS (ravulizumab), also from Alexion Pharmaceuticals, is the first long-acting C5 inhibitor, offering rapid, complete, and sustained suppression of complement activity. It binds to C5, preventing its cleavage and subsequent formation of the membrane attack complex (C5b-9). ULTOMIRIS is effective in managing terminal complement-mediated hemolysis in PNH and thrombotic microangiopathy in aHUS. Although the precise mechanism in gMG and NMOSD remains unclear, it is thought to reduce complement deposition at the neuromuscular junction and block aquaporin-4 antibody-mediated complement activation. It received approvals from the US FDA, EMA, and Japan's PMDA in 2022. Get an in-depth assessment on generalized myasthenia gravis medication for adults @ ULTOMIRIS vs SOLIRIS for Myasthenia Gravis UCB Biopharma's RYSTIGGO is a subcutaneously administered, humanized monoclonal antibody that binds with high affinity to the human neonatal Fc receptor (FcRn). It received regulatory approval from the US FDA and Japan's PMDA in 2023, followed by the EC in January 2024. ZILBRYSQ, also developed by UCB Biopharma, is the first once-daily subcutaneous peptide-based inhibitor specifically targeting complement protein C5. It works by blocking C5 cleavage and the formation of the terminal complement complex C5b-9. Although its precise action in gMG remains uncertain, it is thought to limit C5b-9 accumulation at the neuromuscular junction. ZILBRYSQ gained approval from the US FDA, EMA, and PMDA in 2023. VYVGART, from Argenx, is a human IgG1 antibody fragment that targets FcRn, leading to a decrease in circulating IgG autoantibodies. It is the first FcRn blocker to receive regulatory approval. Argenx's VYVGART HYTRULO (also marketed as VYVDURA) combines efgartigimod alfa, an FcRn inhibitor, with hyaluronidase, an endoglycosidase, for treating adults with generalized myasthenia gravis. This formulation was approved by the US FDA and EMA in 2023 and by the PMDA in January 2024. Uncover the mechanism of FcRn inhibitor myasthenia gravis medication @ Myasthenia Gravis MoA On April 30, 2025, the FDA approved Johnson & Johnson's nipocalimab, branded as IMAAVY, as a new therapy for gMG. This approval puts IMAAVY among the list of approved drugs for myasthenia gravis treatment. The approval, which J&J says encompasses the widest eligible patient population with gMG, includes individuals aged 12 and above who test positive for AChR or anti-muscle-specific kinase (MuSK) antibodies. IMAAVY functions by inhibiting the FcRn protein, which plays a role in maintaining circulating IgG antibodies, key drivers in many autoimmune disorders. By blocking FcRn, IMAAVY significantly reduces harmful IgG autoantibodies while preserving the body's overall immune response, according to clinical data. J&J sees broader potential for IMAAVY beyond gMG, believing its mechanism of action could apply across multiple autoimmune conditions due to IgG's involvement in various diseases. The FDA's decision followed results from the ongoing Phase III Vivacity-MG3 trial, which evaluates IMAAVY in combination with standard care versus placebo and standard care in 199 adult gMG patients, including 153 antibody-positive individuals. Additionally, J&J is conducting a Phase II/III pediatric trial called Vibrance, where IMAAVY, combined with standard care, achieved its primary goal by reducing total serum IgG levels by 69% over 24 weeks. J&J acquired IMAAVY through its USD 6.5 billion acquisition of Momenta Pharmaceuticals in 2020, with the FcRn inhibitor myasthenia gravis serving as the centerpiece of that deal. The company is aiming for blockbuster status, forecasting peak annual sales of over USD 5 billion for the myasthenia gravis medication. Dive deep into the myasthenia gravis treatment cost @ Generalized Myasthenia Gravis Drug with Copay Myasthenia gravis pipeline possesses some drugs in mid- and late-stage development to be approved in the near future. The expected launch of myasthenia gravis treatment medications, such as Batoclimab (Immunovant Sciences GmbH), Descartes-08 (Cartesian Therapeutics), and others, are expected to further create a positive impact on the myasthenia gravis disease market. Discover which therapies are expected to grab major myasthenia gravis therapeutics market share @ Market Analysis on Myasthenia Gravis Batoclimab is a fully humanized monoclonal antibody designed to inhibit neonatal Fc receptors by blocking the interaction between FcRn and IgG, thereby promoting the breakdown of autoantibodies. It has received Orphan Drug Designation (ODD) from both the US FDA and the EMA. In March 2025, Immunovant announced topline results from its Phase III trial evaluating batoclimab in patients with myasthenia gravis. Descartes-08 represents the first RNA-engineered CAR T-cell (rCAR-T) therapy developed for autoimmune conditions. This therapy uses cytotoxic T-cells programmed to target and eliminate pathogenic plasma cells that produce autoantibodies. Created from a patient's own blood, Descartes-08 has also received ODD from the US FDA for treating myasthenia gravis. In December 2024, Cartesian Therapeutics shared encouraging updated findings from its Phase IIb study of Descartes-08 in myasthenia gravis. Furthermore, in January 2025, the FDA agreed to the company's Phase III AURORA trial design under a Special Protocol Assessment, with trial initiation planned for the first half of 2025. Curious about what the new myasthenia gravis treatments are in development? Visit Myasthenia Gravis Clinical Trials The anticipated launch of these emerging myasthenia gravis treatments are poised to transform the market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the myasthenia gravis market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. DelveInsight estimates that the myasthenia gravis market size in the 7MM is expected to grow from USD 5.2 billion in 2024 at a significant CAGR by 2034. According to DelveInsight's analysis, the myasthenia gravis market growth is anticipated with the introduction of emerging therapies, leading to an expansion in the myasthenia gravis market size throughout the forecast period (2025–2034). This anticipated myasthenia gravis market growth is driven by advancements in treatment options, greater healthcare access, and a rising myasthenia gravis prevalence, which together foster higher demand for innovative and effective therapies. DelveInsight's latest published market report titled Myasthenia Gravis Market Insight, Epidemiology, and Market Forecast – 2034 will help you to discover which myasthenia gravis treatment market leader is going to capture the largest market share. The report provides comprehensive insights into the country-specific myasthenia gravis treatment guidelines, patient pool analysis, and epidemiology forecast to help understand the key opportunities and assess the market's underlying potential. The myasthenia gravis market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM, segmented into: Total Diagnosed Prevalent Cases of Myasthenia Gravis Gender-specific Diagnosed Prevalent Cases of Myasthenia Gravis Age-specific Diagnosed Prevalent Cases of Myasthenia Gravis Diagnosed Prevalent Cases of Myasthenia Gravis by MGFA Classification Diagnosed Prevalent Cases of Generalized Myasthenia Gravis by Antibody Serology The report provides an edge while developing business strategies by understanding trends shaping and driving the 7MM myasthenia gravis market. Highlights include: 10-year Forecast 7MM Analysis Epidemiology-based Market Forecasting Historical and Forecasted Market Analysis upto 2034 Emerging Drug Market Uptake Peak Sales Analysis Key Cross Competition Analysis Industry Expert's Opinion Access and Reimbursement Download this myasthenia gravis market report to assess the epidemiology forecasts, understand the patient journeys, know KOLs' opinions about the upcoming treatment paradigms, and determine the factors contributing to the shift in the myasthenia gravis market. Also, stay abreast of the mitigating factors to improve your market position in the myasthenia gravis therapeutic space. Related Reports Myasthenia Gravis Epidemiology Forecast Myasthenia Gravis Epidemiology Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted myasthenia gravis epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Myasthenia Gravis Pipeline Myasthenia Gravis Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key myasthenia gravis companies, including Harbour BioMed (Guangzhou) Co. Ltd., Kyverna Therapeutics, Cabaletta Bio, Takeda, Hoffmann-La Roche, Immunovant Sciences GmbH, Regeneron Pharmaceuticals, Novartis Pharmaceuticals, Janssen Research & Development, LLC, Momenta Pharmaceuticals, Inc., Amgen, Dianthus Therapeutics, Cartesian Therapeutics, COUR Pharmaceutical Development Company, Inc., Alexion Pharmaceuticals, Inc., among others. Generalized Myasthenia Gravis Market Generalized Myasthenia Gravis Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key gMG companies including Alexion Pharmaceuticals, Argenx, UCB Biopharma, Argenx-Halozyme Therapeutics, Horizon Therapeutics, Hoffmann-La Roche, Janssen Research & Development, LLC, Immunovant Sciences GmbH, Bristol Myers Squibb, Biogen Inc., Pfizer Inc., Novartis AG, Sanofi S.A., Roche Holding AG, Johnson & Johnson, Takeda Pharmaceutical Company Limited, AbbVie Inc., Eli Lilly and Company, Merck & Co., Inc., GlaxoSmithKline plc, AstraZeneca plc, Amgen Inc., Boehringer Ingelheim International GmbH, Genentech, Inc., Gilead Sciences, Inc., Celgene Corporation, Vertex Pharmaceuticals Incorporated , among others. Generalized Myasthenia Gravis Pipeline Generalized Myasthenia Gravis Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key gMG companies, including Biocon, Cartesian Therapeutics, UCB, Momenta Pharmaceuticals, HanAll Biopharma, Roche, Alexion, Novartis, Takeda, BioMarin, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

免疫疗法临床3期上市批准孤儿药临床结果

2025-05-21

·GlobeNewswire-Health Care

Phase II Data in Treatment-Naïve Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients: Ruxoprubart Demonstrates Best-in-Class Efficacy as Monotherapy

--- Ruxoprubart (NM8074) met all clinical endpoints, offering a safe, differentiated treatment for Paroxysmal Nocturnal Hemoglobinuria (PNH). Paroxysmal Nocturnal Hemoglobinuria (PNH) is a rare hematological (Blood) disorder. Regulatory approval for the Phase II trial in PNH subjects was granted based on the safety profile of healthy subjects in the Phase I trial. Interim results from this 12-subject, three-month, multi-dose Phase II efficacy trial are compelling. The drug was safe and well-tolerated in all treated PNH subjects with expected safety & efficacy, meeting all clinical endpoints with no reported side effects. In this efficacy trial, Ruxoprubart: Protected PNH Red Blood cells against lysis and enabled complete transfusion avoidance, meeting the primary endpoint. Increased hemoglobin levels above baseline by 1.4 to 2.0 g/dL in most subjects, with some showing intra-subject hemoglobin increase of 1.5 to 4.3 g/dL, exceeding the threshold set for the primary endpoint. An average hemoglobin increase of >1.6 g/dL was observed across the cohort from baseline to the end of the treatment period. Reduced LDH levels to baseline, meeting the primary efficacy endpoint. Significantly increased PNH Type III clone size by protecting PNH Red Blood Cells from complement-mediated lysis. Allowed normal pathogenic clearance in all treated PNH subjects, as infection cleared as expected. The regulatory agency has recently approved a once-weekly subcutaneous protocol for Ruxoprubart — allowing for self-administration and offering a distinct advantage over Apellis, which requires twice-weekly dosing. Ruxoprubart effectively inhibited the Alternative Pathway (AP) without affecting the Classical Pathway (CP) in the trial. More importantly, the FDA has granted Orphan Drug Designation (ODD) to Ruxoprubart for treating PNH. A Breakthrough Therapy Designation (BTD) application for Ruxoprubart in PNH will soon be filed for FDA review. Ruxoprubart is set to be developed as a novel treatment for several complement-mediated disorders, including but not limited to renal, hematological, dermatological, and ocular diseases. May 19, 2025 -- NovelMed is pleased to announce positive 12-week interim results from the ongoing multi-dose Phase II trial of Ruxoprubart, a novel complement-targeting immunotherapy, in adult patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). These interim data demonstrate that Ruxoprubart, administered as monotherapy, was safe, well-tolerated, and met all primary efficacy endpoints. Ruxoprubart achieved transfusion avoidance, increased hemoglobin levels, reduced LDH, and increased PNH clone size — key outcomes that contributed to improved disease control and enhanced quality of life for PNH patients. “The promising interim results from our Phase II PNH study solidify Ruxoprubart as a novel treatment for patients with advanced hematological conditions, offering hope where existing therapies fall short. This achievement not only underscores our commitment to advancing Ruxoprubart, an immunotherapy for PNH, but also marks a significant step toward addressing critical unmet needs within this patient population," said Dr. Rekha Bansal, CEO. "I extend my sincere gratitude to the PNH subjects and investigators whose dedication has been instrumental in this success. We are eager to present our findings to regulatory authorities and proceed with the Phase III trial." Ruxoprubart works by binding to Bb and selectively inhibiting the protease activity of the Alternative Pathway C3 convertase, while preserving the Classical Pathway. By acting proximally in the Alternative Pathway, Ruxoprubart provides complete control over both intravascular and extravascular hemolysis, resulting in marked increases in hemoglobin levels observed in this trial. These findings support the drug’s advancement as a novel, next-generation immunotherapy for the treatment of PNH and a broad range of hematological, renal, inflammatory, and ocular diseases. “We are excited to announce that our Alternative Pathway strategy for treating PNH is delivering exceptional results,” said Mr. Alex Kumar, Chief Strategy Advisor at NovelMed. “These data reinforce our confidence as we look forward to working with the regulatory agency to bring Ruxoprubart to PNH patients without delay. Our mission is to transform the lives of chronically ill patients for whom an ideal treatment has yet to be discovered.” This Phase II trial was initiated following the successful completion of a Phase I study involving 40 (forty) healthy volunteers. Ruxoprubart was well tolerated across all dose levels, ranging from 0.3 to 20 mg/kg, with no safety concerns. The drug demonstrated selective inhibition of the Alternative Pathway through Bb binding, without affecting the Classical Pathway. These promising safety findings paved the way for regulatory approval to initiate a Phase II study in patients with PNH. This ongoing 12-week clinical trial of Ruxoprubart is an open-label, multi-dose study in treatment-naïve adult subjects with PNH. The primary objectives are to evaluate the safety, tolerability, and efficacy of Ruxoprubart as a monotherapy in the Alternative Pathway-mediated lysis of Red Blood Cells. To date, 10 out of 12 PNH subjects have completed the study. Subjects 11 & 12 have been enrolled to complete the two-cohort trial. Interim results confirm that Ruxoprubart achieved all primary efficacy endpoints, delivering sustained clinical benefit in PNH. The primary efficacy endpoints include: Transfusion Avoidance: PNH patients typically require multiple blood transfusions to maintain normal hemoglobin levels. Ruxoprubart effectively protected PNH Type III cells from Alternative Pathway-mediated destruction, resulting in complete transfusion avoidance and successfully meeting the primary efficacy endpoint. Hemoglobin Increase: PNH patients experience reduced hemoglobin levels, leading to severe anemia. Ruxoprubart increased hemoglobin levels above baseline by 1.4 to 2.0 g/dL in most subjects, with some showing intra-subject increases ranging from 1.5 to 4.3 g/dL, surpassing the efficacy threshold defined for the primary endpoint. An average increase of >1.6 g/dL in hemoglobin was observed across the cohort from the beginning to the end of the treatment period. LDH Reduction: PNH patients experience severe lysis of Red Blood Cells (RBCs), leading to elevated lactate dehydrogenase (LDH) levels. By controlling complement-mediated hemolysis, Ruxoprubart reduced LDH levels to baseline, achieving the primary efficacy endpoint. Preservation of PNH Cells: Continuous lysis of Red Blood Cells in PNH leads to a dramatic reduction in total RBC count, often exceeding 80 to 90%. By effectively preventing this lysis, Ruxoprubart significantly preserved PNH cells, resulting in a substantial increase in the total PNH clone size and hemoglobin, successfully meeting the primary efficacy endpoint. Pathogenic Clearance: As expected, Ruxoprubart facilitated effective pathogenic clearance across all treated PNH subjects. In summary, the observed changes in efficacy parameters support the drug's mechanism of action and validate Ruxoprubart’s potential as a highly effective monotherapy for PNH. “We are thrilled to share these data, which demonstrate the potential of Ruxoprubart in advanced PNH, and look forward to working with regulatory authorities to bring this new immunotherapy to this patient community,” said Mr. Robert Bard, Vice President of Regulatory Affairs at NovelMed. “It’s very clear that we are on a promising path with exciting Phase II data in this serious hematological disorder.” PNH is a rare, acquired hematologic disorder in which Red Blood Cells lack protective surface proteins due to a mutation in the phosphatidylinositol glycan class A (PIGA) gene, leaving them vulnerable to complement-mediated destruction. In healthy individuals, Type I RBCs express complement regulatory proteins and are fully protected from complement-mediated destruction. In contrast, PNH patients have a significant percentage of Type III RBCs, which completely lack these protective proteins and are therefore highly susceptible to Alternative Pathway-mediated lysis. PNH is primarily an Alternative Pathway-mediated disease, with no involvement of the Classical or Lectin pathways in its pathology. Key laboratory markers indicative of disease progression include reduced Type III Red Blood Cells, low hemoglobin, elevated lactate dehydrogenase (LDH), and an increased need for blood transfusions. These markers reflect the underlying hemolytic anemia, which in turn leads to debilitating symptoms such as chronic fatigue, pain, jaundice, and organ damage. As the disease progresses, it significantly affects the patient’s quality of life and can ultimately result in life-threatening complications and death if not properly managed. While Soliris® (eculizumab) is the current FDA-approved standard of care for PNH – administered via intravenous infusion to provide C5 blockade – clinical limitations persist. Up to 88% of Soliris-treated patients continue to experience persistent anemia, with more than one-third of them requiring blood transfusions at least once every year. Despite its effectiveness in some patients, Soliris does not fully address the needs of many patients, as a significant proportion remain anemic, fatigued, and require blood transfusions, highlighting the need for specific, effective, and comprehensive therapies. Nearly all FDA-approved therapies for PNH and other complement-mediated disorders, oral or intravenous, suffer from the well-documented Black Box warning label because of the broad immunosuppression of the Classical Pathway, elevating the risk of life-threatening infections. Ruxoprubart offers a selective, upstream, and targeted approach to Alternative Pathway inhibition, without compromising the Classical Pathway, offering the best-in-class novel therapy. These attributes position Ruxoprubart as a safer and more effective treatment for patients with PNH. Ruxoprubart, an engineered human immunoglobulin, exhibits dual specificity: 1) it blocks only the Alternative Pathway, dysregulated by the disease, and 2) it binds Bb which has no binding to Factor B. Ruxoprubart met all primary endpoints in the interim evaluation of the ongoing Phase II clinical trial in PNH, positioning it as the first immunotherapy in this indication with the potential for approval without a Black Box warning. This immunotherapy has demonstrated a safe and well-tolerated profile across all five cohorts in the Phase I trial, including the highest dose of 20 mg/kg. Our findings confirm that the drug selectively blocks the Alternative Pathway. This differentiated, novel mechanism sets Ruxoprubart apart from other complement blockers (Soliris®, Ultomiris®, Empaveli®, Voydeya®, and Fabhalta®) currently approved or in development worldwide. Voydeya®, which received FDA approval in 2024 as a combination therapy, was developed by Achillion and acquired by AstraZeneca for $930 million. Despite its limited clinical efficacy, Voydeya®'s reliance on co-administration with a C5 inhibitor restricts its potential as a standalone therapy. Unlike Fabhalta®, which targets Factor B, Ruxoprubart (NM8074) binds to Bb, which is generated only upon activation of the Alternative Pathway, offering a unique mechanism of action as monotherapy. As a novel monotherapy with a unique, upstream, and pathway-specific mechanism, Ruxoprubart is emerging as a best-in-class candidate for the treatment of PNH and numerous complement-mediated diseases. NovelMed is a clinical-stage biopharmaceutical company dedicated to developing innovative novel biologics (immunotherapies) to treat a wide range of complement-mediated diseases. NovelMed proudly leads the development of Alternative Pathway-specific antibodies (NM8074, NM5072, and NM8070) to address a wide range of chronic complement-mediated disorders. The company is actively conducting a Phase II clinical trial in treatment-naïve PNH subjects to expedite the availability of this promising treatment. Additionally, the FDA has approved Phase II studies for several rare diseases, including but not limited to Atypical Hemolytic Uremic Syndrome (aHUS), C3 Glomerulopathy (C3G), IgA Nephropathy (IgAN), ANCA-Associated Vasculitis (AAV), and Dermatomyositis (DM). The content above comes from the network. if any infringement, please contact us to modify.

临床结果免疫疗法孤儿药上市批准

2025-05-19

·EINPresswire

NovelMed’s Phase II Data in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients: Ruxoprubart Shows Best-in-Class Efficacy as Monotherapy

--- Ruxoprubart (NM8074) met all clinical endpoints, offering a safe, differentiated treatment for Paroxysmal Nocturnal Hemoglobinuria (PNH). Paroxysmal Nocturnal Hemoglobinuria (PNH) is a rare hematological (Blood) disorder. Regulatory approval for the Phase II trial in PNH subjects was granted based on the safety profile of healthy subjects in the Phase I trial. Interim results from this 12-subject, three-month, multi-dose Phase II efficacy trial are compelling. The drug was safe and well-tolerated in all treated PNH subjects with expected safety & efficacy, meeting all clinical endpoints with no reported side effects. In this efficacy trial, Ruxoprubart: Protected PNH Red Blood cells against lysis and enabled complete transfusion avoidance, meeting the primary endpoint. Increased hemoglobin levels above baseline by 1.4 to 2.0 g/dL in most subjects, with some showing intra-subject hemoglobin increase of 1.5 to 4.3 g/dL, exceeding the threshold set for the primary endpoint. An average hemoglobin increase of >1.6 g/dL was observed across the cohort from baseline to the end of the treatment period. Reduced LDH levels to baseline, meeting the primary efficacy endpoint. Significantly increased PNH Type III clone size by protecting PNH Red Blood Cells from complement-mediated lysis. Allowed normal pathogenic clearance in all treated PNH subjects, as infection cleared as expected. The regulatory agency has recently approved a once-weekly subcutaneous protocol for Ruxoprubart — allowing for self-administration and offering a distinct advantage over Apellis, which requires twice-weekly dosing. Ruxoprubart effectively inhibited the Alternative Pathway (AP) without affecting the Classical Pathway (CP) in the trial. More importantly, the FDA has granted Orphan Drug Designation (ODD) to Ruxoprubart for treating PNH. A Breakthrough Therapy Designation (BTD) application for Ruxoprubart in PNH will soon be filed for FDA review. Ruxoprubart is set to be developed as a novel treatment for several complement-mediated disorders, including but not limited to renal, hematological, dermatological, and ocular diseases. CLEVELAND, May 19, 2025 (GLOBE NEWSWIRE) -- NovelMed is pleased to announce positive 12-week interim results from the ongoing multi-dose Phase II trial of Ruxoprubart, a novel complement-targeting immunotherapy, in adult patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). These interim data demonstrate that Ruxoprubart, administered as monotherapy, was safe, well-tolerated, and met all primary efficacy endpoints. Ruxoprubart achieved transfusion avoidance, increased hemoglobin levels, reduced LDH, and increased PNH clone size — key outcomes that contributed to improved disease control and enhanced quality of life for PNH patients. “The promising interim results from our Phase II PNH study solidify Ruxoprubart as a novel treatment for patients with advanced hematological conditions, offering hope where existing therapies fall short. This achievement not only underscores our commitment to advancing Ruxoprubart, an immunotherapy for PNH, but also marks a significant step toward addressing critical unmet needs within this patient population," said Dr. Rekha Bansal, CEO. "I extend my sincere gratitude to the PNH subjects and investigators whose dedication has been instrumental in this success. We are eager to present our findings to regulatory authorities and proceed with the Phase III trial." Figure. NovelMed Scientist Prepares Complement Test Assays to Support Clinical Development of Ruxoprubart (NM8074) for Paroxysmal Nocturnal Hemoglobinuria (PNH). Ruxoprubart works by binding to Bb and selectively inhibiting the protease activity of the Alternative Pathway C3 convertase, while preserving the Classical Pathway. By acting proximally in the Alternative Pathway, Ruxoprubart provides complete control over both intravascular and extravascular hemolysis, resulting in marked increases in hemoglobin levels observed in this trial. These findings support the drug’s advancement as a novel, next-generation immunotherapy for the treatment of PNH and a broad range of hematological, renal, inflammatory, and ocular diseases. “We are excited to announce that our Alternative Pathway strategy for treating PNH is delivering exceptional results,” said Mr. Alex Kumar, Chief Strategy Advisor at NovelMed. “These data reinforce our confidence as we look forward to working with the regulatory agency to bring Ruxoprubart to PNH patients without delay. Our mission is to transform the lives of chronically ill patients for whom an ideal treatment has yet to be discovered.” Clinical Study Overview and Efficacy Summary in PNH This Phase II trial was initiated following the successful completion of a Phase I study involving 40 (forty) healthy volunteers. Ruxoprubart was well tolerated across all dose levels, ranging from 0.3 to 20 mg/kg, with no safety concerns. The drug demonstrated selective inhibition of the Alternative Pathway through Bb binding, without affecting the Classical Pathway. These promising safety findings paved the way for regulatory approval to initiate a Phase II study in patients with PNH. This ongoing 12-week clinical trial of Ruxoprubart is an open-label, multi-dose study in treatment-naïve adult subjects with PNH. The primary objectives are to evaluate the safety, tolerability, and efficacy of Ruxoprubart as a monotherapy in the Alternative Pathway-mediated lysis of Red Blood Cells. To date, 10 out of 12 PNH subjects have completed the study. Subjects 11 & 12 have been enrolled to complete the two-cohort trial. Interim results confirm that Ruxoprubart achieved all primary efficacy endpoints, delivering sustained clinical benefit in PNH. The primary efficacy endpoints include: Transfusion Avoidance : PNH patients typically require multiple blood transfusions to maintain normal hemoglobin levels. Ruxoprubart effectively protected PNH Type III cells from Alternative Pathway-mediated destruction, resulting in complete transfusion avoidance and successfully meeting the primary efficacy endpoint. Hemoglobin Increase : PNH patients experience reduced hemoglobin levels, leading to severe anemia. Ruxoprubart increased hemoglobin levels above baseline by 1.4 to 2.0 g/dL in most subjects, with some showing intra-subject increases ranging from 1.5 to 4.3 g/dL, surpassing the efficacy threshold defined for the primary endpoint. An average increase of >1.6 g/dL in hemoglobin was observed across the cohort from the beginning to the end of the treatment period. LDH Reduction : PNH patients experience severe lysis of Red Blood Cells (RBCs), leading to elevated lactate dehydrogenase (LDH) levels. By controlling complement-mediated hemolysis, Ruxoprubart reduced LDH levels to baseline, achieving the primary efficacy endpoint. Preservation of PNH Cells : Continuous lysis of Red Blood Cells in PNH leads to a dramatic reduction in total RBC count, often exceeding 80 to 90%. By effectively preventing this lysis, Ruxoprubart significantly preserved PNH cells, resulting in a substantial increase in the total PNH clone size and hemoglobin, successfully meeting the primary efficacy endpoint. Pathogenic Clearance : As expected, Ruxoprubart facilitated effective pathogenic clearance across all treated PNH subjects. In summary, the observed changes in efficacy parameters support the drug's mechanism of action and validate Ruxoprubart’s potential as a highly effective monotherapy for PNH. “We are thrilled to share these data, which demonstrate the potential of Ruxoprubart in advanced PNH, and look forward to working with regulatory authorities to bring this new immunotherapy to this patient community,” said Mr. Robert Bard, Vice President of Regulatory Affairs at NovelMed. “It’s very clear that we are on a promising path with exciting Phase II data in this serious hematological disorder.” Paroxysmal Nocturnal Hemoglobinuria (PNH) PNH is a rare, acquired hematologic disorder in which Red Blood Cells lack protective surface proteins due to a mutation in the phosphatidylinositol glycan class A (PIGA) gene, leaving them vulnerable to complement-mediated destruction. In healthy individuals, Type I RBCs express complement regulatory proteins and are fully protected from complement-mediated destruction. In contrast, PNH patients have a significant percentage of Type III RBCs, which completely lack these protective proteins and are therefore highly susceptible to Alternative Pathway-mediated lysis. PNH is primarily an Alternative Pathway-mediated disease, with no involvement of the Classical or Lectin pathways in its pathology. Key laboratory markers indicative of disease progression include reduced Type III Red Blood Cells, low hemoglobin, elevated lactate dehydrogenase (LDH), and an increased need for blood transfusions. These markers reflect the underlying hemolytic anemia, which in turn leads to debilitating symptoms such as chronic fatigue, pain, jaundice, and organ damage. As the disease progresses, it significantly affects the patient’s quality of life and can ultimately result in life-threatening complications and death if not properly managed. While Soliris® (eculizumab) is the current FDA-approved standard of care for PNH – administered via intravenous infusion to provide C5 blockade – clinical limitations persist. Up to 88% of Soliris-treated patients continue to experience persistent anemia, with more than one-third of them requiring blood transfusions at least once every year. Despite its effectiveness in some patients, Soliris does not fully address the needs of many patients, as a significant proportion remain anemic, fatigued, and require blood transfusions, highlighting the need for specific, effective, and comprehensive therapies. Nearly all FDA-approved therapies for PNH and other complement-mediated disorders, oral or intravenous, suffer from the well-documented Black Box warning label because of the broad immunosuppression of the Classical Pathway, elevating the risk of life-threatening infections. Ruxoprubart offers a selective, upstream, and targeted approach to Alternative Pathway inhibition, without compromising the Classical Pathway, offering the best-in-class novel therapy. These attributes position Ruxoprubart as a safer and more effective treatment for patients with PNH. Ruxoprubart (NM8074) Ruxoprubart, an engineered human immunoglobulin, exhibits dual specificity: 1) it blocks only the Alternative Pathway, dysregulated by the disease, and 2) it binds Bb which has no binding to Factor B. Ruxoprubart met all primary endpoints in the interim evaluation of the ongoing Phase II clinical trial in PNH, positioning it as the first immunotherapy in this indication with the potential for approval without a Black Box warning. This immunotherapy has demonstrated a safe and well-tolerated profile across all five cohorts in the Phase I trial, including the highest dose of 20 mg/kg. Our findings confirm that the drug selectively blocks the Alternative Pathway. This differentiated, novel mechanism sets Ruxoprubart apart from other complement blockers (Soliris®, Ultomiris®, Empaveli®, Voydeya®, and Fabhalta®) currently approved or in development worldwide. Voydeya®, which received FDA approval in 2024 as a combination therapy, was developed by Achillion and acquired by AstraZeneca for $930 million. Despite its limited clinical efficacy, Voydeya®'s reliance on co-administration with a C5 inhibitor restricts its potential as a standalone therapy. Unlike Fabhalta®, which targets Factor B, Ruxoprubart (NM8074) binds to Bb, which is generated only upon activation of the Alternative Pathway, offering a unique mechanism of action as monotherapy. As a novel monotherapy with a unique, upstream, and pathway-specific mechanism, Ruxoprubart is emerging as a best-in-class candidate for the treatment of PNH and numerous complement-mediated diseases. NovelMed Therapeutics ( www.novelmed.com ) NovelMed is a clinical-stage biopharmaceutical company dedicated to developing innovative novel biologics (immunotherapies) to treat a wide range of complement-mediated diseases. NovelMed proudly leads the development of Alternative Pathway-specific antibodies (NM8074, NM5072, and NM8070) to address a wide range of chronic complement-mediated disorders. The company is actively conducting a Phase II clinical trial in treatment-naïve PNH subjects to expedite the availability of this promising treatment. Additionally, the FDA has approved Phase II studies for several rare diseases, including but not limited to Atypical Hemolytic Uremic Syndrome (aHUS), C3 Glomerulopathy (C3G), IgA Nephropathy (IgAN), ANCA-Associated Vasculitis (AAV), and Dermatomyositis (DM). We are committed to delivering life-changing therapies to patients across hematology, ophthalmology, nephrology, dermatology, and neurology. NovelMed is currently seeking partnerships to advance the development of Ruxoprubart to approval in several chronic diseases. We are open to discussions regarding investment, out-licensing, or acquisition. For more details regarding our progress, please visit www.novelmed.com/news . Watch our introductory Video to learn more about NovelMed. Contact: Ya Gao, MS NovelMed Communication Team BD@novelmed.com (216) 440 2696 Figure. NovelMed Scientist Prepares Clinical Trial Samples for Analysis https://www.globenewswire.com/NewsRoom/AttachmentNg/ae2c0bfc-ff88-486d-a796-f2977947c8a4 Figure. NovelMed Scientist Prepares Clinical Trial Samples for Analysis Figure. NovelMed Scientist Prepares Clinical Trial Samples for Analysis (Available at https://www.globenewswire.com/NewsRoom/AttachmentNg/ae2c0bfc-ff88-486d-a796-f2977947c8a4) Legal Disclaimer: EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.

临床结果临床2期免疫疗法孤儿药上市批准

100 项与依库珠单抗相关的药物交易

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KEGG	Wiki	ATC	Drug Bank
D03940	依库珠单抗	L04AA25	DB01257

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
水通道蛋白4抗体阳性视神经脊髓炎谱系疾病	澳大利亚	Alexion Pharmaceuticals, Inc.	2009-03-20
非典型溶血性尿毒症综合征	欧盟	Alexion Europe SAS	2007-06-20
非典型溶血性尿毒症综合征	冰岛	Alexion Europe SAS	2007-06-20
非典型溶血性尿毒症综合征	列支敦士登	Alexion Europe SAS	2007-06-20
非典型溶血性尿毒症综合征	挪威	Alexion Europe SAS	2007-06-20
重症肌无力	欧盟	Alexion Europe SAS	2007-06-20
重症肌无力	冰岛	Alexion Europe SAS	2007-06-20
重症肌无力	列支敦士登	Alexion Europe SAS	2007-06-20
重症肌无力	挪威	Alexion Europe SAS	2007-06-20
视神经脊髓炎	欧盟	Alexion Europe SAS	2007-06-20
视神经脊髓炎	冰岛	Alexion Europe SAS	2007-06-20
视神经脊髓炎	列支敦士登	Alexion Europe SAS	2007-06-20
视神经脊髓炎	挪威	Alexion Europe SAS	2007-06-20
阵发性睡眠性血红蛋白尿症	美国	Alexion Pharmaceuticals, Inc.	2007-03-16

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适应症	最高研发状态	国家/地区	公司	日期
移植物功能延迟恢复	临床3期	美国	Alexion Pharmaceuticals, Inc.	2014-08-21
移植物功能延迟恢复	临床3期	澳大利亚	Alexion Pharmaceuticals, Inc.	2014-08-21
移植物功能延迟恢复	临床3期	巴西	Alexion Pharmaceuticals, Inc.	2014-08-21
移植物功能延迟恢复	临床3期	加拿大	Alexion Pharmaceuticals, Inc.	2014-08-21
移植物功能延迟恢复	临床3期	捷克	Alexion Pharmaceuticals, Inc.	2014-08-21
移植物功能延迟恢复	临床3期	法国	Alexion Pharmaceuticals, Inc.	2014-08-21
移植物功能延迟恢复	临床3期	德国	Alexion Pharmaceuticals, Inc.	2014-08-21
移植物功能延迟恢复	临床3期	意大利	Alexion Pharmaceuticals, Inc.	2014-08-21
移植物功能延迟恢复	临床3期	西班牙	Alexion Pharmaceuticals, Inc.	2014-08-21
水通道蛋白4抗体阳性视神经炎	临床3期	美国	Alexion Pharmaceuticals, Inc.	2014-04-11

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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


NCT04320602 (CTgov)	临床4期	阵发性睡眠性血红蛋白尿症	18	Eculizumab+Ravulizumab	鬱齋壓鬱壓憲範壓簾鏇 = 衊夢壓鏇壓廠鏇積鏇鏇壓廠範夢鬱鹽廠遞顧繭 (積鹽築糧膚願鹽鏇構鬱, 築選獵鏇夢夢壓鹹遞鏇 ~ 壓窪鹹廠範廠築網簾餘) 更多	-	2024-08-12
NCT01194973 (C10-004, FDA_CDER) 人工标引	临床2期	非典型溶血性尿毒症综合征	41	Eculizumab	繭獵簾餘襯鏇艱範窪構(製廠壓壓鏇積獵築鹽淵) = 築齋憲獵艱製糧餘願襯醖齋鹽鹽餘遞膚蓋蓋範 (範衊範繭構遞淵顧齋淵, 40 ~ 72) 更多	积极	2024-07-19
NCT00844844 \| NCT00844545 (C08-002B, FDA_CDER) 人工标引	临床2期	非典型溶血性尿毒症综合征	17	Eculizumab (at 26 wks)	選鏇鹹選鬱壓築蓋範淵(獵製淵選艱顧構窪鹹淵) = 簾餘繭鹽鏇範構襯艱糧網鑰積網廠範鏇糧鹽窪 (醖獵觸廠齋餘壓獵選鹽 ) 更多	积极	2024-07-19
				Eculizumab (at 2 yrs)	選鏇鹹選鬱壓築蓋範淵(獵製淵選艱顧構窪鹹淵) = 構網願鹹餘淵鹽築餘顧網鑰積網廠範鏇糧鹽窪 (醖獵觸廠齋餘壓獵選鹽 ) 更多
NCT01770951 (C09-001r, FDA_CDER) 人工标引	N/A	非典型溶血性尿毒症综合征	19	Eculizumab (<2 yrs)	窪製糧鏇範遞廠構鹽淵(築襯淵餘夢遞艱廠衊壓) = 鹹觸願構糧鹹齋鹽憲襯觸淵鬱鏇鑰鹹鬱範夢蓋 (衊淵簾繭夢鏇齋醖鹽構 ) 更多	积极	2024-07-19
				Eculizumab (2 to <12 yrs)	窪製糧鏇範遞廠構鹽淵(築襯淵餘夢遞艱廠衊壓) = 糧衊製糧淵窪壓鑰壓鬱觸淵鬱鏇鑰鹹鬱範夢蓋 (衊淵簾繭夢鏇齋醖鹽構 ) 更多
NCT00844428 \| NCT00838513 (C08-003B, FDA_CDER) 人工标引	临床2期	非典型溶血性尿毒症综合征	20	Eculizumab (at 26 wks)	蓋範鑰糧鬱觸選選鹽選(壓積壓構醖壓壓顧鏇築) = 窪廠選築蓋廠齋齋衊鹽遞願顧蓋製觸襯齋積衊 (蓋糧窪壓網網顧膚鏇夢 ) 更多	积极	2024-07-19
				Eculizumab (at 2 yrs)	蓋範鑰糧鬱觸選選鹽選(壓積壓構醖壓壓顧鏇築) = 鑰繭鑰網網鹹齋憲壓積遞願顧蓋製觸襯齋積衊 (蓋糧窪壓網網顧膚鏇夢 ) 更多
NCT01193348 (C10-003, FDA_CDER) 人工标引	临床2期	非典型溶血性尿毒症综合征	22	Eculizumab (Patients 1 month to <12 years)	膚窪獵鏇糧窪繭醖網醖(膚鹹網壓遞餘獵糧鬱願) = 築鑰憲構壓製築範艱鹹艱壓鬱鹹襯鬱獵衊艱蓋 (壓選網獵鏇簾觸襯廠夢, 36 ~ 83) 更多	积极	2024-07-19
				Eculizumab (All Patients)	膚窪獵鏇糧窪繭醖網醖(膚鹹網壓遞餘獵糧鬱願) = 艱餘鹽範製鑰醖夢蓋願艱壓鬱鹹襯鬱獵衊艱蓋 (壓選網獵鏇簾觸襯廠夢, 41 ~ 83) 更多
NCT00122330 (FDA_CDER) 人工标引	临床3期	阵发性睡眠性血红蛋白尿症	87	Placebo	窪構膚鬱窪糧壓膚築築(繭觸糧鹹願鑰鏇築範糧) = 製遞衊糧構窪夢簾壓膚夢膚壓遞憲淵廠衊醖構 (願簾夢顧襯網膚獵範範 ) 更多	积极	2024-07-19
				Eculizumab	窪構膚鬱窪糧壓膚築築(繭觸糧鹹願鑰鏇築範糧) = 網繭鏇觸衊鏇製醖遞廠夢膚壓遞憲淵廠衊醖構 (願簾夢顧襯網膚獵範範 ) 更多
NCT03500549 (PEGASUS, EHA2024) 人工标引	临床3期	阵发性睡眠性血红蛋白尿症	68	pegcetacoplaneculizumab	-	积极	2024-05-14
				pegcetacoplan +eculizumab (patients with baseline IO)	製襯齋獵餘觸鬱顧鹹壓(範艱簾膚醖鹹醖構遞簾) = 糧蓋遞夢網衊顧範網糧膚窪鬱醖鹹築選夢遞膚 (鏇廠糧願遞夢淵鏇夢糧, 8.0) 更多
EHA2024	N/A	阵发性睡眠性血红蛋白尿症	200	Eculizumab	蓋蓋遞簾願膚餘顧繭構(積繭鹹簾淵艱願衊糧襯) = 襯餘壓膚顧壓顧糧窪廠齋憲鹽壓餘選構夢膚鹹 (範構鬱艱鏇窪糧窪餘獵 ) 更多	积极	2024-05-14
				Ravulizumab	蓋蓋遞簾願膚餘顧繭構(積繭鹹簾淵艱願衊糧襯) = 艱選築艱簾願窪壓積顧齋憲鹽壓餘選構夢膚鹹 (範構鬱艱鏇窪糧窪餘獵 ) 更多
ISN WCN2024	N/A	非典型溶血性尿毒症综合征 genetic mutations in the alternate complement pathway	6	Eculizumab sparing protocols	蓋艱蓋憲鹹鬱獵繭願艱(壓範餘壓膚遞憲蓋鬱鏇) = None had BK virus infection 顧廠網築齋鑰製夢簾膚 (觸選醖鑰鹽鑰願願壓製 ) 更多	积极	2024-04-01