Ultomiris (ravulizumab-cwvz) / AstraZeneca, Xencor - LARVOL DELTA

Advancing Landscape of Paroxysmal Nocturnal Hemoglobinuria Treatment. (PubMed, Turk J Haematol) - "With an improved understanding of PNH biology, a focused effort on complement inhibitors led to the discovery of eculizumab, a C5 inhibitor initially approved by the FDA in 2007. Further advancements in drug development for PNH include improved pharmacokinetics with ravulizumab in 2018 and the introduction of proximal complement inhibitors such as pegcetacoplan (2021), iptacopan (2023), and danicopan (2024), and crovalimab (2024) to enhance patient outcomes. With these new proximal and distal complement inhibitors in the treatment landscape, it is timely for clinicians to review the evolving landscape of PNH treatments and patient selection."

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

Long-Term Efficacy and Safety of Ravulizumab in Adults With Anti-Acetylcholine Receptor Antibody-Positive Generalized Myasthenia Gravis: Final Results From the Phase 3 CHAMPION MG Open-Label Extension. (PubMed, Eur J Neurol) - "Ravulizumab demonstrated clinically meaningful and durable efficacy and safety in adults with AChR-Ab+ gMG."

Clinical • Journal • P3 data • CNS Disorders • Fatigue • Infectious Disease • Meningococcal Infections • Myasthenia Gravis

Breakthrough hemolysis in paroxysmal nocturnal hemoglobinuria throughout clinical trials: from definition to clinical practice. (PubMed, Blood) - "In particular, BTH may occur with all complement inhibitors, with a frequency of 10-15% over 6 months with eculizumab, crovalimab, and pegcetacoplan, and <5% with ravulizumab, iptacopan, and danicopan plus anti-C5. Complement amplifying conditions were observed in about half of cases and were more frequently infections. Treatment adherence, optimization of the administration schedule, anticoagulant prophylaxis, as well as education of patients and physicians remain important factors to prevent BTH and its complications."

Journal • Complement-mediated Rare Disorders • Hematological Disorders • Infectious Disease • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • Thrombosis

ISS1.10 How terminal complement inhibition continues to change the landscape of atypical haemolytic uraemic syndrome (aHUS) (ERA 2025) - "Currently approved C5i include eculizumab and ravulizumab, which directly block the terminal complement pathway. Case reports from the faculty will provide a basis for an interactive dialogue with the audience around these and other patient types, and how to optimize their treatment. Finally, the audience will be invited to ask additional questions around how to manage patients with this debilitating and multifaceted disease."

Atypical Hemolytic Uremic Syndrome • Complement-mediated Rare Disorders • Nephrology • Pediatrics • Rare Diseases • Renal Disease

Ravulizumab in the ongoing evolution of aHUS management (ERA 2025) - No abstract available

Complement-mediated Rare Disorders

A PK/PD Analysis and Population PK Modeling of Ravulizumab Using Data from the Phase 2 Trial in Adults with IgA Nephropathy (SANCTUARY) (ERA 2025) - No abstract available

Clinical • P2 data • PK/PD data • Glomerulonephritis • IgA Nephropathy • Renal Disease

Efficacy and safety of ravulizumab for the maintenance of aHUS remission after kidney transplant (ERA 2025) - No abstract available

Clinical • Complement-mediated Rare Disorders • Transplantation

Safety and mid-term efficacy of switching from Eculizumab to Ravulizumab in a cohort of adult patients with atypical hemolytic uremic syndrome (ERA 2025) - No abstract available

Clinical • Atypical Hemolytic Uremic Syndrome • Complement-mediated Rare Disorders • Nephrology

Real-world effectiveness of ravulizumab in adults with aHUS who switched to ravulizumab within 3 months of eculizumab treatment (aHUS IMPACT) (ERA 2025) - No abstract available

Clinical • Real-world • Real-world effectiveness • Real-world evidence • Complement-mediated Rare Disorders

A Phase 3, Open-Label, Multicenter Study of Ravulizumab in Pediatric Patients With IgA Nephropathy or IgA Vasculitis-Associated Nephritis (ERA 2025) - No abstract available

Clinical • P3 data • Glomerulonephritis • IgA Nephropathy • Nephrology • Pediatrics • Renal Disease • Vasculitis

Ravulizumab in atypical haemolytic uraemic syndrome: analysis of quality of life outcomes in adult and paediatric phase 3 trials (ERA 2025) - No abstract available

Clinical • HEOR • P3 data • Atypical Hemolytic Uremic Syndrome • Complement-mediated Rare Disorders • Pediatrics

Ravulizumab in atypical hemolytic uremic syndrome: final analysis of efficacy and safety outcomes in two phase 3 trials (UKKW 2025) - No abstract available

Clinical • P3 data • Atypical Hemolytic Uremic Syndrome • Complement-mediated Rare Disorders • Nephrology

Atypical Hemolytic Uremic Syndrome: A Review of Complement Dysregulation, Genetic Susceptibility and Multiorgan Involvement. (PubMed, J Clin Med) - "C5 inhibitors, such as eculizumab and ravulizumab, have revolutionized treatment but necessitate prophylactic vaccination and ongoing clinical surveillance. Progress in genetic profiling and biomarker discovery is essential for earlier diagnosis, individualized therapy and relapse prevention. This review highlights recent advances in the understanding of aHUS pathophysiology, clinical features and evolving therapeutic strategies aimed at improving patient outcomes."

Journal • Review • Atypical Hemolytic Uremic Syndrome • Cardiovascular • Complement-mediated Rare Disorders • Hematological Disorders • Immunology • Infectious Disease • Ischemic stroke • Nephrology • Thrombocytopenia • Thrombocytopenic Purpura • Thrombosis

Patient Preferences for Treatment Features in Neuromyelitis Optica Spectrum Disorder (NMOSD): Results From a Discrete Choice Experiment (DCE) (P7-8.013). (PubMed, Neurology) - "To quantify preferences and predict treatment choices between ravulizumab and other approved treatments (eculizumab, inebilizumab, satralizumab) among US adults with anti-aquaporin-4 antibody-positive (AQP4-Ab+) NMOSD. Conway has received research support from Biogen. Disclaimer: Abstracts were not reviewed by Neurology® and do not reflect the views of Neurology® editors or staff."

Journal • CNS Disorders • Infectious Disease • Meningococcal Infections • Neuromyelitis Optica Spectrum Disorder • Rare Diseases

Effectiveness and Safety of Ravulizumab in Generalized Myasthenia Gravis (gMG): Updated Analysis from a Global Registry (P1-11.014). (PubMed, Neurology) - P | "MG-ADL total scores and MGFA classification were assessed in patients enrolled in the MG SPOTLIGHT Registry who received ravulizumab, including those who received ravulizumab only (ravu-only subgroup) or transitioned from eculizumab to ravulizumab (ecu-to-ravu subgroup) with data available prior to C5IT initiation ("pre-C5IT") and ≥1 assessment post-ravulizumab initiation ("post-ravu")...Pulley has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Cabaletta...Howard has a non-compensated relationship as a Committee member with American Assoc Neuromuscular and Electrodiagnostic Medicine that is relevant to AAN interests or activities. Disclaimer: Abstracts were not reviewed by Neurology® and do not reflect the views of Neurology® editors or staff."

Journal • CNS Disorders • Infectious Disease • Meningococcal Infections • Myasthenia Gravis • Rare Diseases

Switching to Subcutaneous Zilucoplan from Intravenous Complement Component 5 Inhibitors in Myasthenia Gravis: Patient Preference and Satisfaction from a Phase 3b Study (P1-11.009). (PubMed, Neurology) - P3 | "Twenty-six patients enrolled and received zilucoplan; 16 switched from eculizumab and 10 from ravulizumab. Howard has a non-compensated relationship as a Committee member with American Assoc Neuromuscular and Electrodiagnostic Medicine that is relevant to AAN interests or activities. Disclaimer: Abstracts were not reviewed by Neurology® and do not reflect the views of Neurology® editors or staff."

Journal • P3 data • CNS Disorders • Myasthenia Gravis

Clinical Characteristics Associated With High Baseline Glial Fibrillary Acidic Protein (GFAP) and Neurofilament Light Chain (NfL) Levels in Patients With Anti-Aquaporin-4 Antibody-Positive (AQP4-Ab+) Neuromyelitis Optica Spectrum Disorder (NMOSD) From the PREVENT and CHAMPION-NMOSD Trials (S38.001). (PubMed, Neurology) - P3 | "The placebo-controlled PREVENT and externally controlled CHAMPION-NMOSD trials evaluated the efficacy and safety of eculizumab and ravulizumab, respectively, in adults with AQP4-Ab+ NMOSD. Pittock has received publishing royalties from a publication relating to health care. Disclaimer: Abstracts were not reviewed by Neurology® and do not reflect the views of Neurology® editors or staff."

Biomarker • Clinical • Journal • CNS Disorders • Hematological Malignancies • Leukemia • Lymphoma • Neuromyelitis Optica Spectrum Disorder • Oncology • Rare Diseases • GFAP • NEFL

Change in Concomitant Immunosuppressive Therapies for Generalized Myasthenia Gravis in Patients Receiving Complement C5 Inhibitor Therapies: A Retrospective Analysis of Registry Data (P7-11.028). (PubMed, Neurology) - "The current analysis included registry patients who transitioned from eculizumab to ravulizumab, had available con-IST (azathioprine, mycophenolate mofetil, intravenous immunoglobulin/plasma exchange, oral corticosteroid [OCS]) data, and received eculizumab and ravulizumab for ≥1 year...Pulley has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Cabaletta...Narayanaswami has a non-compensated relationship as a Member, Board of Directors with AANEM that is relevant to AAN interests or activities. Disclaimer: Abstracts were not reviewed by Neurology® and do not reflect the views of Neurology® editors or staff."

Journal • Retrospective data • CNS Disorders • Myasthenia Gravis • Rare Diseases

Assessment of Hospitalizations in Patients with Generalized Myasthenia Gravis (gMG) Before and During Treatment with Ravulizumab: Results from a Global Registry (P1-11.016). (PubMed, Neurology) - P | "Reductions in hospitalizations with ravulizumab were most pronounced in patients not previously treated with eculizumab, whereas the decrease in hospitalizations associated with eculizumab was maintained after switching to ravulizumab...Pulley has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Cabaletta...Pulley has received personal compensation in the range of $50,000-$99,999 for serving as an Expert Witness for Multiple law firms. Disclaimer: Abstracts were not reviewed by Neurology® and do not reflect the views of Neurology® editors or staff."

Journal • CNS Disorders • Myasthenia Gravis • Rare Diseases

A Phase 3, Open-Label, Single-Arm, Multicenter Study to Evaluate Ravulizumab Administered Intravenously in Pediatric Patients With Anti-Acetylcholine Receptor Antibody-Positive (AChR-Ab+) Generalized Myasthenia Gravis (gMG) (P7-6.014). (PubMed, Neurology) - P3 | "Frick has stock in AstraZeneca/Alexion Rare Disease. Disclaimer: Abstracts were not reviewed by Neurology® and do not reflect the views of Neurology® editors or staff."

Clinical • Journal • P3 data • CNS Disorders • Immunology • Myasthenia Gravis • Pediatrics • Rare Diseases

Outcomes for Patients with Generalized Myasthenia Gravis Prescribed Ravulizumab or Efgartigimod Treatment: A Retrospective Medical Record Analysis (P1-11.001). (PubMed, Neurology) - "Pulley has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Cabaletta. Pulley has received personal compensation in the range of $50,000-$99,999 for serving as an Expert Witness for Multiple law firms. Disclaimer: Abstracts were not reviewed by Neurology® and do not reflect the views of Neurology® editors or staff."

Journal • Retrospective data • CNS Disorders • Myasthenia Gravis

Ravulizumab in Patients with Generalized Myasthenia Gravis (gMG) from Japan: Real-world Outcomes (P7-11.023). (PubMed, Neurology) - "In the effectiveness analysis set, 24 (53.3%) patients were female, and 38 (84.4%) patients had treatment history with eculizumab, a terminal complement inhibitor, during the 12 months prior to initiating ravulizumab. Murai has received research support from Japan Blood Products Organization. Disclaimer: Abstracts were not reviewed by Neurology® and do not reflect the views of Neurology® editors or staff."

Journal • Real-world evidence • CNS Disorders • Genetic Disorders • Infectious Disease • Meningococcal Infections • Muscular Dystrophy • Myasthenia Gravis • Myotonic Dystrophy • Novel Coronavirus Disease • Pneumonia • Rare Diseases • Respiratory Diseases

Efficacy and Safety of Ravulizumab in Adults With Anti-Aquaporin-4 Antibody-Positive (AQP4-Ab+) Neuromyelitis Optica Spectrum Disorder (NMOSD): Interim Analysis From the Ongoing Phase 3 CHAMPION-NMOSD Trial (S38.007). (PubMed, Neurology) - P3 | "Kim has received personal compensation in the range of $5,000-$9,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Multiple Sclerosis Journal, Journal of Clinical Neurology. Disclaimer: Abstracts were not reviewed by Neurology® and do not reflect the views of Neurology® editors or staff."

Journal • P3 data • P3 data: top line • Cardiovascular • CNS Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Lymphoma • Meningococcal Infections • Multiple Sclerosis • Neuromyelitis Optica Spectrum Disorder • Oncology • Rare Diseases

NMO SPOTLIGHT Registry: Real-World Clinical Outcomes With Eculizumab and Ravulizumab in Anti-Aquaporin-4 Antibody-Positive (AQP4-Ab+) Neuromyelitis Optica Spectrum Disorder (NMOSD) (P11-8.013). (PubMed, Neurology) - P | "Sami Fam has stock in Astra Zeneca. Disclaimer: Abstracts were not reviewed by Neurology® and do not reflect the views of Neurology® editors or staff."

Clinical data • Journal • Real-world evidence • CNS Disorders • Infectious Disease • Meningococcal Infections • Multiple Sclerosis • Neuromyelitis Optica Spectrum Disorder • Rare Diseases

Warren Alpert Medical School of Brown University: Clinicopathologic Conference: September 20th, 2024. A Woman in her 20s with Abdominal Pain, Anemia and Thrombocytopenia. (PubMed, J Brown Hosp Med) - "A case conference describing the presentation of a woman in her 20's with anemia, abdominal pain and thrombocytopenia who was diagnosed with paroxysmal nocturnal hemoglobinuria and likely aplastic anemia."

Journal • Anemia • Aplastic Anemia • Complement-mediated Rare Disorders • Hematological Disorders • Pain • Paroxysmal Nocturnal Hemoglobinuria • Pulmonary Disease • Rare Diseases • Thrombocytopenia