PiaSky (crovalimab-akkz) / Roche - LARVOL DELTA

Improvement in anemia and symptoms after switching from crovalimab to iptacopan in paroxysmal nocturnal hemoglobinuria. (PubMed, Hematology) - "A 72-year-old man with long-standing PNH received eculizumab followed by crovalimab. This case demonstrates the feasibility of switching from crovalimab to iptacopan despite the absence of an established method, and pharmacokinetic considerations guided the timing. Switching to iptacopan led to rapid and durable improvement in anemia and symptoms, supporting proximal complement inhibition as a valuable option for PNH patients inadequately controlled with C5 inhibitors."

Journal • Anemia • Complement-mediated Rare Disorders • Gastrointestinal Disorder • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Pulmonary Disease • Rare Diseases

R&D activities: Hematology (Chugai Press Release) - "We decided to remove a pH-dependent binding humanized anti-complement (C5) monoclonal antibody SKY59/RG6107 (Product name: PiaSky) for the treatment of sickle cell disease from the pipeline following the decision made by Roche to discontinue the development, considering the results of overseas study."

Discontinued • Sickle Cell Disease

Domestic sales (Chugai Press Release) - "Specialty product sales were ¥225.8 billion (an increase of 5.8% year on year). This was primarily due to the strong sales of the mainstay product Vabysmo (an ophthalmic VEGF/Ang-2 inhibitor, anti-VEGF/anti Ang-2 humanized bispecific monoclonal antibody), Enspryng (pH-dependent binding humanized anti-IL-6 receptor monoclonal antibody), and Hemlibra (a blood coagulation factor VIII substitute/anti-coagulation factor IXa/X humanized bispecific monoclonal antibody), as well as the favorable market penetration of the new product PiaSky (a pH dependent binding humanized anti-complement (C5) monoclonal antibody), despite the market penetration of generic drugs and the effects of the NHI drug price revisions. Meanwhile, compared to the full year forecast announced on January 30, 2025, domestic sales increased by 2.1% to ¥472.4 billion, due to the increased sales of Hemlibra, Enspryng, Vabysmo, etc."

Commercial • Age-related Macular Degeneration • Diabetic Macular Edema • Hemophilia A • Neuromyelitis Optica Spectrum Disorder • Paroxysmal Nocturnal Hemoglobinuria • Retinal Vein Occlusion

Advancing treatment goals for paroxysmal nocturnal hemoglobinuria to align with quality of life improvements in the era of anti-complement therapy (PubMed, Rinsho Ketsueki) - "Eculizumab, a C5 inhibitor introduced in 2010, directly inhibits intravascular hemolysis, and thus not only resolves hemolysis-related symptoms but also prevents organ damage and improves survival...Various drugs have been developed to address these issues, including long-acting C5 inhibitors (ravulizumab and crovalimab) and proximal complement inhibitors capable of blocking extravascular hemolysis, such as a C3 inhibitor (pegcetacoplan), a factor B inhibitor (iptacopan), and a factor D inhibitor (danicopan). In particular, proximal complement inhibitors further enhance QOL because they inhibit both intravascular and extravascular hemolysis, resulting in greater improvement of hemoglobin levels and transfusion independence, and thereby further enhancing of QOL. Today, it is possible to achieve optimal improvement in QOL by appropriately selecting one of the three C5 inhibitors as first-line therapy or one of the three proximal complement inhibitors as second-line..."

HEOR • Journal • Cardiovascular • Chronic Kidney Disease • Complement-mediated Rare Disorders • Hematological Disorders • Nephrology • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • Renal Disease • Thrombosis

Therapeutic Antibodies in Hematology: Advances in Malignant and Non-Malignant Disorders. (PubMed, Cells) - "Besides cancer, complement inhibitors such as eculizumab, ravulizumab, and the recently approved crovalimab have redefined paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome management, and the bispecific antibody emicizumab has transformed prophylaxis in hemophilia A. Furthermore, novel antibody formats such as the trifunctional anti-CD38 × CD3 antibody (Tri-31C2) exhibit enhanced anti-myeloma activity compared to chimeric CD38 antibodies, underscoring the future potential of T-cell-redirecting designs. This review summarizes key developments in therapeutic antibodies for hematological disorders, their action mechanisms, and emerging strategies to further optimize their efficacy and safety."

Journal • Review • Atypical Hemolytic Uremic Syndrome • Complement-mediated Rare Disorders • Hematological Disorders • Hematological Malignancies • Hemophilia • Hemophilia A • Multiple Myeloma • Nephrology • Oncology • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

Crovalimab Rescue Therapy in a Case With Genetic Complement Mediated Thrombotic Microangiopathy. (PubMed, Kidney Med) - "Current treatments include the complement C5 blockers eculizumab and ravulizumab as well as plasma therapy. Hemolysis improved immediately and kidney function recovered after 3 months of dialysis treatment and improved continuously during 1 year of therapy with crovalimab. The excellent and rapid response to crovalimab potentially suggests that the engineering of crovalimab, facilitating also subcutaneous administration, may result in a different pharmacokinetic and pharmacodynamic profile of crovalimab as compared with standard C5 inhibitors in patient with nephrotic range proteinuria."

Journal • Acute Kidney Injury • Atypical Hemolytic Uremic Syndrome • Complement-mediated Rare Disorders • Hematological Disorders • Nephrology • Rare Diseases • Renal Disease

The Ministery of Food and Drug Safety said on the 24th that it approved the rare drug Piascai injection (hereafter Piascai), used to treat paroxysmal nocturnal hemoglobinuria in children 12 and older and adults who weigh at least 40 kilograms. (Chosun Biz)

Korea approval • Paroxysmal Nocturnal Hemoglobinuria

Optimizing PNH treatment with the complement inhibitor pegcetacoplan: A case report (ASH 2025) - "The current treatment landscape includes 6 approved complement cascade inhibitors: 3 C5 inhibitors (eculizumab, ravulizumab, crovalimab), 1 C3/C3b inhibitor (pegcetacoplan), 1 factor B inhibitor (iptacopan), and 1 factor D inhibitor used as add-on treatment (danicopan)...Concomitant medications included apixaban, penicillin, and folic acid. In November 2020, her platelets count declined, and a bone marrow evaluation was diagnostic for moderate aplastic anemia (55-65% cellularity for age) and she was started on eltrombopag and cyclosporin. Despite ravulizumab and eltrombopag treatments, the patient developed significant anemia related to extravascular hemolysis (hemoglobin, 5.7 g/dL; LDH, 495 U/L; C5, 26.1 mg/dL [high]; complement hemolytic activity 50 [CH50], 7 U/mL [low])...After receiving both pegcetacoplan and iptacopan for 1 week and rivaroxaban 10 mg once daily for 48 hours, pegcetacoplan treatment ended on May 16, 2024... For this patient with PNH, the..."

Case report • Clinical • Anorexia • Aplastic Anemia • Complement-mediated Rare Disorders • Hematological Disorders • Infectious Disease • Meningococcal Infections • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

Low risk for meningococcal and other encapsulated bacteria infections with systemically administered pegcetacoplan in paroxysmal nocturnal hemoglobinuria and C3 glomerulopathies (ASH 2025) - P3 | "Clinical benefits of the initially available C5 inhibitors that blocked terminal complement activation (eculizumab, ravulizumab, crovalimab) paved the way for the development of proximal inhibitors, including the C3/C3b inhibitor pegcetacoplan, the factor B inhibitor iptacopan, and the add-on (to C5 inhibitors) factor D inhibitor danicopan. Understanding the safety profile of pegcetacoplan and other complement-targeted therapies, especially the risk for meningococcal and other encapsulated bacteria infections, will help physicians and patients make informed treatment decisions for individuals with complement-mediated conditions. For nearly over 7 years, systemically administered pegcetacoplan has had a consistently low rate of encapsulated bacteria infections in patients with PNH, C3G, or primary IC-MPGN. These findings may reflect effective risk mitigation strategies."

CNS Disorders • Complement-mediated Rare Disorders • Glomerulonephritis • Hematological Disorders • Immunology • Infectious Disease • Influenza • Lupus Nephritis • Meningococcal Infections • Nephrology • Paroxysmal Nocturnal Hemoglobinuria • Pneumococcal Infections • Pneumonia • Primary Immunodeficiency • Rare Diseases • Respiratory Diseases • Septic Shock

Direct comparison of crovalimab versus eculizumab in paroxysmal nocturnalhemoglobinuria: A systematic review and meta-analysis of randomized controlled trials (ASH 2025) - "Crovalimab offers comparable efficacy and safety to eculizumab in the treatment of PNH, both in C5-inhibitor–naive patients and those transitioning from prior therapy. With no rise in SAEs, crovalimab was linked to notable improvements in hemoglobin levels, fatigue scores, hemolysis control, and transfusion avoidance. Due to its subcutaneous mode of administration and long-lasting therapeutic benefit, crovalimab offers an adequate prospective alternative for conventional intravenous C5 inhibitors."

Retrospective data • Review • Aplastic Anemia • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

Paroxysmal nocturnal hemoglobinuria in pediatric patients: A UK experience (ASH 2025) - "Approved treatments for those under 18 years of age include eculizumab (andbiosimilars), ravulizumab and latterly crovalimab (over 12 years)...One additional patient had a myelodysplasia diagnosis.Treatment of aplastic anemia in the 53 patients included no treatment for 9/53 (17%), anti-thymocyteglobulin and ciclosporin in 18/53 (34%), ciclosporin 1/53 (2%), oxymethalone 1/53 (2%) and hematopoieticstem cell transplant (HSCT) for 24/53 (45%) (either first or second line).Five thrombotic events occurred in 4 patients at diagnosis: 2 cerebral vein thromboses, 1 Budd Chiari and1 pulmonary embolism (PE) and portal vein thrombosis.Management of patients with PNH requiring complement inhibition (41/65):The majority of patients experienced at least 2 symptoms from PNH (30/41), including fatigue (26/41),thrombosis (2/41), abdominal pain (5/41), shortness of breath (7/41), hemoglobinuria (7/41), dysphagia(1/41), bruising/bleeding (18/41) and anemia (27/41)...Currently the PNH..."

Clinical • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Cardiovascular • Complement-mediated Rare Disorders • Gastrointestinal Disorder • Hematological Disorders • Infectious Disease • Meningococcal Infections • Myelodysplastic Syndrome • Paroxysmal Nocturnal Hemoglobinuria • Pediatrics • Pulmonary Embolism • Rare Diseases • Respiratory Diseases

Study design of A phase 3, open-label trial for pozelimab and cemdisiran combination therapy in patients with paroxysmal nocturnal hemoglobinuria with inadequate control of intravascular hemolysis (ASH 2025) - "Treatment for PNH includes C5 inhibitorssuch as eculizumab/biosimilar, ravulizumab, and crovalimab, however, patients under terminalcomplement inhibition can experience residual intravascular hemolysis due to incomplete C5 blockade.The combination of pozelimab (a monoclonal antibody that prevents activation of C5) and cemdisiran (asilencing RNA that reduces production of circulating C5) is a novel approach being investigated for itsability to achieve durable inhibition of the terminal complement pathway. During the ext period, the secondary endpoints willinclude percent change in LDH from baseline to ext week 24 and ext week 52, normalization of LDH ateach visit through ext week 52, inclusive, and adequate control of hemolysis at each visit through extweek 52. Recruitment for this study is expected to begin around November 2025."

Clinical • Combination therapy • P3 data • Bone Marrow Transplantation • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

Correlation between efficacy, clonal dynamics evolution, and development of bone marrow failure in PNH/AA syndrome treated with complement inhibitors: A single-center cohort study (ASH 2025) - "Objective:To investigate the correlation between therapeutic response patterns to complement inhibitors, clonaldynamics changes, and progression of bone marrow failure (BMF) in patients with PNH/AA (paroxysmalnocturnal hemoglobinuria/aplastic anemia).A retrospective analysis was conducted on 92 PNH/AA patients treated at our center from 2021 to 2025.All patients received complement inhibitor therapy (eculizumab, crovalimab, or iptacopan), with a medianfollow-up of 29 months.Parameters monitored included PNH clone size, next-generation sequencing (NGS), lactatedehydrogenase (LDH) levels, hematologic response, breakthrough hemolysis (BTH), and dynamicchanges in hematopoietic function (reticulocytes, absolute neutrophil count, platelets). Complement inhibitors effectively control hemolysis in PNH/AA patients but provide limitedimprovement in BMF. Residual hemolysis, the emergence of new subclones, and an expanding PNH cloneare associated with BMF progression. Timely..."

Clinical • Anemia • Aplastic Anemia • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

The role of red blood cell lifespan in evaluating the condition of patients with paroxysmal nocturnal hemoglobinuria treated with complement inhibitors (ASH 2025) - "Four patients were treated with Eculizumab: 1 achieved CR, 2achieved PR, and 1 achieved minimal partial remission (MIPR).Following treatment, the median LDH level decreased by 1689 U/L (820–3114.6), and all patients had LDHlevels below twice the upper limit of normal (ULN) within three weeks, with 20 maintaining this level.Median RBC lifespan increased by 30.5 days (-16–71), with the following changes by treatment group:Crovalimab: -3.5 days (-16–3); Iptacopan: +61 days (24–71); KP104 cohort 1: +37 days (26–62); KP104cohort 2: +53 days (31–60); Eculizumab: -2 days (-7–12). Different complement inhibitors, whether targeting upstream or downstream components ofthe complement system, demonstrate varying effects on PNH disease control. Hemoglobin levels duringtreatment are an independent factor influencing therapeutic outcomes, and RBC lifespan is closelyassociated with hemoglobin dynamics. RBC lifespan provides a quantitative measure of extravascularhemolysis and serves as..."

Clinical • Aplastic Anemia • Cardiovascular • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • Thrombosis

Pregnancy outcomes after crovalimab exposure: Analysis of animal model and clinical trial data suggest no adverse effects in paroxysmal nocturnal hemoglobinuria (ASH 2025) - P1, P1/2, P3 | "Adverse outcomes, including hypertensive disorders such as pre-eclampsia, are common incomplement-mediated diseases; therefore, pregnant patients being treated for such diseases should bemonitored. Although data on crovalimab treatment during pregnancy are limited in patients with PNH,data from the reproductive toxicity study in pregnant animals and clinical trial data suggest no adverseeffect of crovalimab treatment on pregnant mothers or fetuses, consistent with data from other C5inhibitors. As untreated PNH in pregnancy is associated with adverse outcomes, continuation ofcrovalimab treatment in patients with PNH who become pregnant should be considered."

Adverse events • Preclinical • Atypical Hemolytic Uremic Syndrome • Complement-mediated Rare Disorders • Genetic Disorders • Gynecology • Hematological Disorders • Hypertension • Nephrology • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • Sickle Cell Disease • PIAS4

Switching between complement inhibitors in patients with PNH: A real-world analysis of strategy, efficacy, and safety. (ASH 2025) - "In 2025, 3 C5 inhibitors (C5i) are approved (eculizumab (ECU), ravulizumab (RAV), crovalimab) & 3proximal inhibitors (PI) (pegcetacoplan (PEG), ipatacopan (IPTA), danicopan (DAN) plus C5i). Some clinicaltrial therapies have not continued development (vermicopan (VERM), BCX9930 (BCX), other C5i).PI clinical trials have protocols for changing from terminal to PI, based on drug half-life... Sixty-two pts from 8 countries were included with mean age at diagnosis 38.4 years (range 16-79)& mean Hb 87.8 g/L (missing data, n=17). Where reported indications for CI were hemolysis (49/62),hemolysis and thrombosis (5/62), thrombosis (3/62). Mean time on CI was 103.6 months (range 23-276; missing n=3) & mean granulocyte clone 86% (range 31-99; missing n=9).First-line CI were ECU/RAV (50/62), VERM (9/62) & investigational C5i (3/62)."

Clinical • Real-world • Real-world evidence • Cardiovascular • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • Thrombosis

Comparative efficacy of complement inhibitors in complement Inhibitor–Naïve PNH: A network meta-analysis of randomized trials (ASH 2025) - "A frequentist model network meta-analyses were conducted in RStudio (v5.4.1) using acommon-effects model. A total of 4 randomized controlled trials evaluating 4 complement inhibitor agents (Ravulizumab,Crovalimab, Eculizumab & Pegcetacoplan) were included in this meta-analysis, involving 589 complementinhibitor–naïve adults with PNH. We found no single agent being consistently superior to others across all clinically relevantoutcomes. Notably, the treatment of choice should be individualized based on the goals of care andpriorities of the patients such as transfusion independence or quality of life. Post-market real-worldanalysis and comparison of these agents may guide optimal sequencing or cost-effective strategies in themanagement of PNH."

Retrospective data • Anemia • Aplastic Anemia • Complement-mediated Rare Disorders • Hematological Disorders • Hematological Malignancies • Myelodysplastic Syndrome • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

Efficacy and Safety of the C5 Inhibitor Crovalimab in Patients With Paroxysmal Nocturnal Hemoglobinuria: A Systematic Review and Meta-Analysis. (PubMed, Eur J Haematol) - "Crovalimab showed encouraging efficacy and an acceptable safety profile in patients with PNH, supporting its role as a promising treatment option in PNH."

Journal • Retrospective data • Review • Complement-mediated Rare Disorders • Hematological Disorders • Infectious Disease • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

Patient Preferences and Treatment Satisfaction in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Treated with Crovalimab and Approved C5 Inhibitors in the Phase III Randomized COMMODORE 1 and COMMODORE 2 Trials (ASH 2023) - P3 | "Introduction Existing C5 inhibitor (C5i) therapies for the management of PNH, eculizumab (ecu) and ravulizumab (ravu), are typically given by regular intravenous (IV) administration (every 2 weeks [q2w] and every 8 weeks [q8w], respectively) in a hospital. Pt perceptions of convenience around treatment frequency and mode of administration largely drove preference. With SC injection q4w and the option for self-administration outside of a supervised healthcare setting, crova has the potential to offer a new treatment option for pts with PNH that is less burdensome than existing therapies for this chronic lifelong disease."

Clinical • P3 data • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

Evaluating the Value Elements Considered in Health Technology Assessments of Paroxysmal Nocturnal Hemoglobinuria Treatments: A Targeted Review (ISPOR-EU 2025) - "This analysis assessed how value elements were considered in HTAs for PNH treatments and their impact on HTA decision-making. A targeted search was conducted in June 2025 for PNH treatments approved since 2020 (iptacopan, pegcetacoplan, danicopan, crovalimab) across five HTA body websites (NICE, GBA, HAS, TLV, Medicinrådet). Novel ISPOR value elements were included in CSs and CRs with qualitative supporting data, however costs and QALYs remained the primary focus. Research and efforts from companies/HTA bodies are needed to generate supporting data and facilitate adoption of broader value elements in decision-making, particularly in rare diseases."

Review • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

Real-World Adherence with Pegcetacoplan in Paroxysmal Nocturnal Hemoglobinuria Compared with Previously Reported Oral Medication Adherence Rates (ASH 2024) - "Several complement inhibitors are approved for PNH, including the intravenous or subcutaneous C5 inhibitors (C5is) eculizumab, ravulizumab, and crovalimab; the subcutaneous self-administered C3 inhibitor pegcetacoplan; and the oral factor B inhibitor iptacopan and factor D inhibitor danicopan (as add-on therapy to a C5i)...OAC adherence rates in AF range widely (~40% to ~90%), differing across countries, patient populations (incident AF vs. post cardiovascular event), OAC types (warfarin, direct OACs), and follow-up period lengths...2020; 26 : 186].Pegcetacoplan adherence for PNH in the US postmarketing setting from launch (2021) to date (2024) was estimated at 97%, well above the 80% threshold defining medication adherence in the literature.Conclusions : Real-world patients with PNH who self-administer pegcetacoplan subcutaneously have adherence rates exceeding the reported real-world adherence rates for oral medications in chronic conditions, especially those with high..."

Adherence • Clinical • HEOR • Real-world • Real-world evidence • Atrial Fibrillation • Cardiovascular • Complement-mediated Rare Disorders • Diabetes • Hematological Malignancies • Metabolic Disorders • Oncology • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • Thrombosis • Type 2 Diabetes Mellitus

Terminal Complement Inhibition Exhibiting Higher Response in Patients with Paroxysmal Nocturnal Hemoglobinuria: A Multicentric Real-World Evidence in Brazil (ASH 2024) - "The majority of hemolytic patients (n=93, 53%) was treated with terminal complement inhibitor, with 87 patients receiving eculizumab and 6 patients receiving crovalimab after a median time of 24 months, and the median duration of the treatment was 86 months. Lower thrombosis and chronic kidney disease rates were reported in this cohort and require further investigation. Terminal complement inhibition was safe and effectively controlled hemolysis, improved symptoms, and reduced complications, with normalization of hemoglobin and transfusion- avoidance rate slowly higher than previously reported."

Clinical • HEOR • Real-world • Real-world evidence • Acute Kidney Injury • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Cardiovascular • Chronic Kidney Disease • Complement-mediated Rare Disorders • Fatigue • Gastrointestinal Disorder • Hematological Disorders • Hematological Malignancies • Infectious Disease • Myelodysplastic Syndrome • Nephrology • Oncology • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • Renal Disease • Venous Thromboembolism

Characteristics and Long-Term Efficacy of 25 Patients with Paroxysmal Nocturnal Hemoglobinuria Treated with Eculizumab or Ravulizumab in Taiwan (ASH 2024) - "The median LDH level was 6.9 (range 1.67-14.74) times the upper limit of normal (ULN) at baseline.Before receiving eculizumab or ravulizumab, thirteen patients (52%) had previously received other therapies, including steroids in nine patients (36%), cyclosporine and/or anti-thymocyte globulin (ATG) in five (20%), danazol in five (20%), and crovalimab in one (4%) who had been enrolled in a trial...Except for one patient who achieved remission of PNH and three patients who crossed over to clinical trials of iptacopan or pozelimab/cemdisiran, seven patients discontinued eculizumab...Eculizumab and ravulizumab were well-tolerated, and no cases of meningococcal disease were reported.Conclusion : Our experiences demonstrated the clinical characteristics and long-term efficacy and safety of eculizumab and ravulizumab in Taiwanese PNH patients with high disease burdens. However, a major reason for discontinuing treatment was the need for frequent transfusions, which did not meet..."

Clinical • Anemia • Aplastic Anemia • Complement-mediated Rare Disorders • Hematological Disorders • Hematological Malignancies • Infectious Disease • Meningococcal Infections • Myelodysplastic Syndrome • Oncology • Paroxysmal Nocturnal Hemoglobinuria • Pulmonary Disease • Rare Diseases • Renal Disease

Safety and Efficacy of Corvalimab in Paroxysmal Nocturnal Hemoglobinuria (PNH): A Systematic Review and Single-Arm Meta-Analysis (ASH 2024) - "Crovalimab, a novel FDA-approved C5 inhibitor, appeared topromising therapy for C5 mutation patients with less effective outcomes on conventionaltherapies.Objective : To evaluate the safety and efficacy of Crovalimab in paroxysmal nocturnalhemoglobinuria (PNH).Methods : The PubMed, Embase, Scopus and Cochrane databases were systematically searchedusing relevant keywords from inception until July 2024...Moreover, only a smallproportion of patients had breakthrough hemoglobin. More robust studies with larger samplesizes are required to establish conclusive evidence."

Retrospective data • Review • Cardiovascular • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • Thrombosis

Current Real-World Treatment Landscape for Patients with Paroxysmal Nocturnal Hemoglobinuria in the United States (ASH 2024) - "CI therapies included eculizumab, ravulizumab, pegcetacoplan, iptacopan, danicopan, and crovalimab. Following its approval, uptake of iptacopan among patients with PNH has been rapid in US real-world clinical practice, with 14% of CI-treated patients currently receiving iptacopan as their most recent therapy. Nonetheless, the majority of included patients with PNH did not have a claim for a CI therapy during the study period, despite the availability of six approved CI therapies."

Clinical • HEOR • Real-world • Real-world evidence • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases