Voydeya (danicopan) / AstraZeneca - LARVOL DELTA

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

Successful retreatment of PNH with switch to ravulizumab and add-on danicopan after experiencing breakthrough hemolysis on pegcetacoplan: A case report (ASH 2025) - "Terminal complement inhibition with C5 inhibitors ravulizumab (Rav), the standard of care for PNH treatment, and eculizumab (Ecu) have provided effective control of terminal complement activity and IVH and decreased rates of thrombosis...The patient was treated intermittently with antithymocyte globulin and/or cyclosporine for ~5 years while receiving red blood cell transfusions... Hb, 11.8 g/dL; LDH, 351 U/L). The patient restarted Rav with 2 loading doses 2 weeks apart. The patient experienced 1 mild episode of BTH ~4 months after restarting Rav (Hb, 9.9 g/dL; LDH, 382 U/L), resolving rapidly with her scheduled dose of Rav."

Case report • Clinical • Cardiovascular • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • Thrombosis

Overview of treatment advances with complement inhibitors in patients with paroxysmal nocturnal hemoglobinuria (ASH 2025) - P3 | "The success of terminal C5 inhibitors emboldened researchers to investigate proximal complement inhibitors that have the potential to control both IVH and EVH, such as pegcetacoplan (C3/C3b inhibitor) and iptacopan (factor B inhibitor), as well as danicopan (factor D inhibitor) used as an add-on to C5 inhibitors...In the most recent analysis of the phase 3 ALPHA trial (NCT04469465), 57 patients (mean age 53 years) with PNH and residual clinically significant EVH (Hb ≤9.5 g/dL with ARC ≥120 × 10 9 /L) while receiving ravulizumab (63%) or eculizumab (37%) for a mean of 5 years were given danicopan in addition to their current C5 inhibitor for 12 weeks.[Kulasekararaj A, et al... Across clinical studies that included patients with PNH and Hb ≥10 g/dL after C5 inhibition, proximal complement inhibitors improved measures of residual anemia and quality of life to clinically significant degrees (increases of ≥2 g/dL in Hb levels and ≥3–5 points in FACIT-Fatigue..."

Clinical • Aplastic Anemia • Cardiovascular • Complement-mediated Rare Disorders • Hematological Disorders • Infectious Disease • Meningococcal Infections • Otorhinolaryngology • Paroxysmal Nocturnal Hemoglobinuria • Pneumonia • Rare Diseases • Respiratory Diseases • Thrombosis

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

Rapidly progressive acute chest syndrome is associated with complement-mediated cell injury in adults with sickle cell disease (ASH 2025) - "Additional testing on a single sample by flow cytometry showed that C5b9 deposition was fully blocked by adding eculizumab but only partially blocked by danicopan, suggesting a contribution of the classical complement pathway as a driver. However, due to the small sample size, our results should be considered as hypothesis-generating. Future studies with larger cohorts and newer functional complement assays, like the mHAM 2.0, should be considered to obtain definitive results."

Clinical • Cough • Genetic Disorders • Hematological Disorders • Pulmonary Disease • Respiratory Diseases • Sickle Cell Disease • Thrombocytopenia • CD55 • CD59

The use of danicopan as an add-on therapy in a ravulizumab-treated adult patient with paroxysmal nocturnal hemoglobinuria (PNH) and concomitant stage IV renal failure has induced a marked improvement of anemia and of clinically significant extravascular hemolysis in absence of deterioration of renal function (ASH 2025) - "In 2019, this patient was treated with biosimilar eculizumab (phase 3 trial- ABP959-Amgen) till spring 2022. In conclusion, danicopan has demonstrated high efficacy in increasing hemoglobin level (range 2.3-3 g/dL), and reducing markers of hemolysis in a PNH patient with concomitant stage IV renal failure, having an inadequate hematological response to either eculizumab or ravulizumab (hemoglobin <9•5 g/dL). Interestingly, renal function was not affected by the combined use of ravulizumab and danicopan, during a 11 months- follow-up observation period."

Clinical • Metastases • Anemia • Aplastic Anemia • Cardiovascular • Complement-mediated Rare Disorders • Endocrine Disorders • Hematological Disorders • Hypertension • Nephrology • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • Renal Disease • Thrombosis

Early access program for danicopan (ALXN2040) as add-on treatment to eculizumab or ravulizumab in patients with paroxysmal nocturnal hemoglobinuria (PNH): Preliminary data from the Italian real-world survey. (ASH 2025) - "Antithrombotic prophylaxis was reported in 4 patients (2warfarin and 2 low molecular weight heparin (LMWH)). to date, data from 10 of the treated patients are available (4 females, median age 41.5ys, range22-49, and 6 males, median age 58ys, range 33-86). Patients mainly suffered from classical PNH (80%),followed by PNH/aplastic anemia (PNH/AA, 20%). Median laboratory tests at the beginning of the C5iwere as follows: hemoglobin level (Hgb) 8.9 g/dl (range, 7.0-9.8), reticulocytes (Ret) count 180*10^6/L(range, 20-341), LDH value 5 x upper limit of normality (ULN) (range, 1.5-28)."

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

Real-world data on breakthrough hemolysis in patients with paroxysmal nocturnal hemoglobinuria treated with proximal and terminal complement inhibitors. (ASH 2025) - "Four patients were treated with factor D inhibitor danicopan(n=3) or iptacopan (n=1) concurrently with ravulizumab.A total of 49 BTH events were identified; while on eculizumab (26 events, 12 patients), ravulizumab (14events, 7 patients), ravulizumab and danicopan (6 events, 3 patients), pegcetacoplan (1 event, 1 patient),zilucoplan (2 events, 2 patients), and no events while on iptacopan. Twelve BTH events were incidentally detected during routine CIadministration; more frequent lab draws may be beneficial for patients with chronic hemolysis. Moredata is needed to understand the BTH rates of different CIs and guide clinicians in BTH management."

Clinical • Real-world • Real-world evidence • Anemia • Aplastic Anemia • Breast Cancer • Complement-mediated Rare Disorders • Infectious Disease • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • Solid Tumor • CD55 • CD59 • HP

Danicopan add-on therapy demonstrates positive efficacy and safety outcomes in advanced age adults with paroxysmal nocturnal hemoglobinuria and clinically significant extravascular hemolysis: A sub-analysis of the phase 3 ALPHA trial (ASH 2025) - P3 | "During 12 weeks ofdanicopan add-on therapy, these patients reported meaningful improvements in Hb concentration andARC, while maintaining transfusion avoidance and a low rate of AEs. Overall, this analysis supports theuse of danicopan add-on therapy in patients with advanced age with PNH and csEVH treated withravulizumab or eculizumab, with no new safety signals."

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

Iptacopan monotherapy demonstrated improved clinical outcomes in a real-world cohort with paroxysmal nocturnal hemoglobinuria: Evidence from a managed access program (ASH 2025) - "The proportion of pts previously treated witheculizumab, ravulizumab, pegcetacoplan, and danicopan was 71.6%, 37.9%, 13.7%, and 6.3%,respectively. The updated data from this MAP confirm hematological and clinical improvements withiptacopan in Ci-naive and Ci-experienced pts with PNH, with approximately 6 months of iptacopantherapy. Oral iptacopan monotherapy led to normalization of Hb and LDH level, and RLC, along with ahigher proportion of pts achieving transfusion avoidance at retreatment visits compared to BL. Pts alsoreported notable improvements in fatigue and tx satisfaction."

Clinical • Clinical data • Monotherapy • Real-world • Real-world evidence • Complement-mediated Rare Disorders • Hematological Disorders • Infectious Disease • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • CFB

Treatment-requiring paroxysmal nocturnal hemoglobinuria in association with myeloproliferative neoplasms (MPNs): Clinical correlations and outcomes (ASH 2025) - "Cytogeneticanalysis (n =10) demonstrated an abnormal karyotype in 3 patients (30%), all of which were categorizedas favorable risk.First-line treatment for PNH included complement inhibitors in 9 patients (82%), with eculizumab (n=8), orravulizumab (n=1)...Notably, one patient with PNH clone size of 91.19 % in granulocytes, had ongoing hemolysisdespite dual complement blockade with ravulizumab and danicopan.Treatment for MPN included hydroxyurea (n=5), ruxolitinib (n=4), pacritinib (n=1), and erythropoiesisstimulating agents (n=4)...The current study represents the largest series of patients with treatment-requiring PNH and MPN. Itreveals a predominance of MF patients, particularly among those harboring CALR mutations, andunderlines suboptimal efficacy of complement inhibitor therapy in this patient population."

Clinical • Cardiovascular • Complement-mediated Rare Disorders • Hematological Malignancies • Infectious Disease • Myelodysplastic Syndrome • Myelofibrosis • Paroxysmal Nocturnal Hemoglobinuria • Polycythemia Vera • Rare Diseases • Septic Shock • Thrombocytosis • Thrombosis • ASXL1 • CALR • JAK2 • SF3B1 • ZRSR2

Complement-induced procoagulant platelets in patients with antiphospholipid antibodies (ASH 2025) - "On certain occasions sera were heat inactivated at 56º C for30 min or pre-treated with IdeS (IgG cleaving enzyme) (10000 U/ml at 37º C for 30 min), eculizumab (1mg/ml), sutimlimab (C1s inhibitor) (1.5 mg/ml) and/or danicopan (Factor D inhibitor) (100 µM). Also, onother occasions platelets were pre-incubated with antagonists of C3aR (SB290157), FcgRIIa (IV.3) or C5aR1(Avacopan) (10 µM) before serum.ResultsWe found that although activation and responsiveness to agonists of platelets from aPL+/APS patientswere like those from healthy individuals, unstimulated platelets from APS patients exhibited higherphosphatidylserine (PS) exposure (19.6±3.9% vs 4.0±1.0% controls) and P-selectin expression (15.9±2.3%vs 8.6±1.8% controls) than controls suggesting induction of procoagulant activity...Pre-incubating platelets with FcgRIIa, C3aR or C5aR1 antagonistsdid not reverse the procoagulant activity induced by APS serum.ConclusionWe demonstrate..."

Clinical • Cardiovascular • Genetic Disorders • Hematological Disorders • Immunology • Thrombosis • ANXA5 • CASP3 • CASP7 • SELP

Management of a real-world cohort of patients with paroxysmal nocturnal hemoglobinuria treated with iptacopan: A multi-institutional analysis (ASH 2025) - "In the 50 patients who switched, 60%transitioned from ravulizumab, 30% from pegcetacoplan, 8% from eculizumab, and 2% fromravulizumab and danicopan. In this real-world cohort, iptacopan provided substantial and durable hemoglobinimprovement across naive and previously treated patients with PNH, with meaningful reduction inhemolytic parameters, absence of thrombosis, and low BTH rate predominantly linked to infection.Patients with previous satisfactory responses also showed improved Hgb levels. The favorable safetyprofile and high treatment persistence underscore iptacopan's role as an effective first-line or switchtherapy and support its incorporation into evidence-based sequencing algorithms in the evolving PNHtreatment landscape."

Clinical • Real-world • Real-world evidence • Cardiovascular • CNS Disorders • Complement-mediated Rare Disorders • Congestive Heart Failure • Heart Failure • Hematological Disorders • Infectious Disease • Pancreatitis • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • Thrombosis • Vascular Neurology

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

AstraZeneca advances its ambition to redefine haematology care with new data from its diverse pipeline and portfolio at the 67th American Society of Hematology (ASH) Annual Meeting and Exposition, 6-9 December 2025. (AstraZeneca Press Release) - "New data will further support clinical benefits of Ultomiris in paroxysmal nocturnal haemoglobinuria and its potential to improve outcomes for paediatric patients with haematopoietic stem cell transplant-associated thrombotic microangiopathy."

Clinical data • Paroxysmal Nocturnal Hemoglobinuria

Complement factor D is a drug target for metabolic-associated fatty liver disease. (PubMed, Mol Immunol) - "These results establish CFD as a novel MAFLD mediator, validating FDA-approved danicopan's therapeutic efficacy and translational potential. This work provides critical evidence for targeting the CFD pathway in MAFLD management."

Journal • Complement-mediated Rare Disorders • Hematological Disorders • Hepatology • Inflammation • Metabolic Disorders • Metabolic Dysfunction-Associated Steatotic Liver Disease • Obesity • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • CD36 • FASN • IL6 • SCARB1 • SLC27A2 • TNFA

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

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

Optimizing Complement Inhibitor Monitoring in PNH and Beyond (ASH 2024) - "Results : In vitro addition of C5 inhibitors (eculizumab, ravulizumab, crovalimab) to healthy control serum demonstrated a dose-dependent increase of complement blockade in GVB++ buffer. In isolated AP buffer, we observed dose-dependent inhibition with increasing concentrations of eculizumab, danicopan (factor D inhibitor), iptacopan (factor B inhibitor), and pegcetacoplan (C3 inhibitor)...Interestingly, in the baseline and 1-week serum, either danicopan or sutimlimab completely inhibited AP activity...Using this assay, EVH can be more precisely defined as full blockade of complement activity with persistent evidence of hemolysis. Furthermore, the assay has applications for not only PNH but also other diseases in which complement inhibitors are indicated, such as atypical hemolytic uremic syndrome."

Anemia • Atypical Hemolytic Uremic Syndrome • Complement-mediated Rare Disorders • Hematological Disorders • Nephrology • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • CD46 • CD55 • CD59

Advances in Complement Inhibition Therapies for Paroxysmal Nocturnal Hemoglobinuria and Autoimmune Hemolytic Disorders. (PubMed, J Blood Med) - "The terminal complement inhibitor, eculizumab, was the initial drug available which significantly reduced hemolysis and thrombotic events but failed to resolve residual extravascular hemolysis and transfusion requirements...This review highlights the evolving therapeutic landscape in complement inhibition by summarizing clinical evidence for the terminal complement inhibitors, as well as pegcetacoplan, iptacopan, and danicopan as emerging agents for treatment of PNH and autoimmune hemolytic anemias-warm AIHA and cold agglutinin disease (CAD). The review also examines ongoing clinical trials and proposes future directions to optimize therapeutic outcomes to address remaining clinical challenges."

Journal • Review • Aplastic Anemia • Autoimmune Hemolytic Anemia • Cardiovascular • Complement-mediated Rare Disorders • Hematological Disorders • Immunology • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • Thrombosis

Baseline Characteristics of Individuals with Paroxysmal Nocturnal Hemoglobinuria in an App-Based Home-Reported Outcomes Study to Evaluate Disease Burden (ASH 2024) - P | "Participants will track symptoms and treatment use, including recently approved treatments (e.g., iptacopan, danicopan, crovalimab)...PNH treatments selected for tracking include iptacopan (n=9), ravulizumab (n=5), eculizumab (n=4), and pegcetacoplan (n=3)...Conclusions : Initial interim baseline data of recently enrolled participants with PNH show a variable disease experience. This app-based, observational study aims to establish a new real-world data source to understand patient priorities related to symptom variability, treatment patterns and preference, and impacts of condition management on HRQoL."

Clinical • Anemia • Cardiovascular • Complement-mediated Rare Disorders • Fatigue • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Pulmonary Disease • Rare Diseases • Thrombosis

Danicopan As Add-on Therapy to Ravulizumab or Eculizumab Versus Placebo in Patients with Paroxysmal Nocturnal Hemoglobinuria and Clinically Significant Extravascular Hemolysis: Phase 3 Long-Term Data (ASH 2023) - P3 | "Conclusions Danicopan as add-on to Rav or Ecu significantly improves Hgb and ARC levels and reduces the need for transfusion by addressing cs-EVH while maintaining control of IVH through 48 wks of treatment. Danicopan demonstrated a favorable benefit-risk profile with no deaths, meningococcal infections, or discontinuations due to hemolysis."

Clinical • P3 data • Cardiovascular • Complement-mediated Rare Disorders • Gastroenterology • Gastrointestinal Disorder • Hematological Disorders • Infectious Disease • Meningococcal Infections • Novel Coronavirus Disease • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • Thrombosis

Patient-Reported Outcomes: Danicopan As Add-on Therapy to Ravulizumab or Eculizumab Versus Placebo in Patients with Paroxysmal Nocturnal Hemoglobinuria and Clinically Significant Extravascular Hemolysis (ASH 2023) - P, P3 | "In this phase 3, randomized, controlled superiority trial, improvements in clinically relevant PROs were observed in the Dan vs Pbo arm in the first double-blind 12 wks of treatment, and FACIT-Fatigue and EORTC-QLQ-C30 scores were maintained during the open-label period to wk 24 in the Dan-Dan arm and improved at wk 24 in Pbo-Dan arm to levels similar to those of the general public. Comparable EQ-5D-3L scores were maintained between arms across the study."

Clinical • Patient reported outcomes • Anemia • Anorexia • Complement-mediated Rare Disorders • Constipation • Fatigue • Gastroenterology • Gastrointestinal Disorder • Hematological Disorders • Infectious Disease • Meningococcal Infections • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

Complement Biosensors Can be Used to Identify Classical Pathway and Alternative Pathway Dysregulation in Complement-Mediated Thrombotic Microangiopathy (ASH 2024) - "This stimulus is blocked by sutimlimab and eculizumab, but not by alternative pathway inhibitors including, AMY-101 (C3 inhibitor), iptacopan (factor B inhibitor, FBi), danicopan (factor D inhibitor, FDi), or even a combination of all three. In summary, these biosensors can be used as a sensitive method for investigating individual mechanisms of complement pathway activation in CM-TMA and help monitor therapeutic complement blockade. Identification of a CP stimulus in CM-TMA provides a potential explanation for ~50% of CM-TMA patients who lack an AP "driving" variant and suggests at least a subset of CM-TMA is characterized by a breakdown of IgM immunologic tolerance."

Atypical Hemolytic Uremic Syndrome • Complement-mediated Rare Disorders • Hematological Disorders • Nephrology • Thrombocytopenic Purpura • CD46 • CD55 • CD59