Empaveli (pegcetacoplan SC) / Apellis, SOBI - LARVOL DELTA

Report from the COMPLETE Study on Pegcetacoplan Real-World-Effectiveness in Patients with Paroxysmal Nocturnal Haemoglobinuria (PNH) (BSH 2025) - No abstract available

Clinical • Real-world • Real-world effectiveness • Real-world evidence • Complement-mediated Rare Disorders • Paroxysmal Nocturnal Hemoglobinuria

Pegcetacoplan for Adolescents With Complement 3 Glomerulopathy or Primary Immune Complex-Membranoproliferative Glomerulonephritis: VALIANT Phase 3 Placebo-Controlled Trial Subgroup Analysis (PAS 2025) - No abstract available

Clinical • P3 data • Glomerulonephritis • Lupus Nephritis • Nephrology

COMPLETE: A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) (clinicaltrials.gov) - P=N/A | N=200 | Recruiting | Sponsor: Swedish Orphan Biovitrum | Trial completion date: Jul 2027 ➔ Aug 2029 | Trial primary completion date: Jan 2026 ➔ Aug 2026

Real-world evidence • Trial completion date • Trial primary completion date • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • HP

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

Efficacy of Pegcetacoplan in Children with C3 Glomerulopathy [WITHDRAWN] (PAS 2025) - No abstract available

Clinical • Complement-mediated Rare Disorders • Glomerulonephritis

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

Real-world evidence for efficacy and safety of Iptacopan and Pegcetacoplan in patients with primary membranoproliferative glomerulonephritis (MPGN) (ERA 2025) - No abstract available

Clinical • HEOR • Real-world • Real-world evidence • Glomerulonephritis • Lupus Nephritis • Nephrology

FPT2002: Pegcetacoplan in Action: Transforming Paroxysmal Nocturnal Haemoglobinuria (PNH) Care with C3 Inhibition (EHA 2025) - "Sponsored by Swedish Orphan Biovitrum AB"

Complement-mediated Rare Disorders • Paroxysmal Nocturnal Hemoglobinuria

Horizon Scanning for Paroxysmal Nocturnal Hemoglobinuria in Brazil: Insights on Past Trends and Future Prospects (ISPOR 2025) - "Reimbursement decisions were obtained from the Conitec website in Brazil, and the reimbursement status in the UK for drugs not yet approved in Brazil was verified on the NICE website. We identified 10 treatments for PNH, six of which had FDA approval: eculizumab (2007, intravenous C5 inhibitor), ravulizumab (2018, intravenous C5 inhibitor), pegcetacoplan (2021, subcutaneous C5 inhibitor), iptacopan (2023, oral factor B inhibitor), danicopan (2024, oral factor D inhibitor used with eculizumab or ravulizumab), and crovalimab (2024, intravenous/subcutaneous C5 inhibitor). Recent cooperation between ANVISA and the FDA promises to accelerate drug approvals in Brazil. Three new therapies, including an oral monotherapy currently under ANVISA evaluation, could significantly improve pharmaceutical services by enhancing distribution and addressing geographical disparities."

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

Apellis Announces FDA Acceptance and Priority Review of the Supplemental New Drug Application for EMPAVELI (pegcetacoplan) for C3G and Primary IC-MPGN (GlobeNewswire) - "Apellis Pharmaceuticals, Inc...announced that the U.S. Food and Drug Administration (FDA) has accepted and granted Priority Review designation of the supplemental New Drug Application (sNDA) for EMPAVELI (pegcetacoplan) for C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN), which are severe and rare kidney diseases. The Prescription Drug User Fee Act (PDUFA) target action date is July 28, 2025....sNDA submission supported by positive Phase 3 VALIANT results at Week 26."

FDA filing • PDUFA • Priority review • Glomerulonephritis

[ICKSH 2025] Paroxysmal nocturnal hemoglobinuria drug Empaveli targets extravasation hemolysis (Korea Biomedical Review) - P3 | N=80 | PEGASUS (NCT03500549) | Sponsor: Apellis Pharmaceuticals, Inc. | "In the PEGASUS trial, a phase 3 study in patients previously treated with eculizumab, Empaveli demonstrated superiority over eculizumab in terms of change in hemoglobin concentration over 16 weeks...In the Empaveli arm, mean hemoglobin levels increased by more than 2.37 g/dL from baseline, and 85 percent of patients remained transfusion-free for 16 weeks in the Empaveli arm, compared to 15 percent in the eculizumab arm. Empaveli also showed significant improvements in absolute reticulocyte count, bilirubin, and LDH levels, with benefits lasting up to 48 weeks."

P3 data • Paroxysmal Nocturnal Hemoglobinuria

Pegcetacoplan for post-transplant recurrent C3 glomerulopathy or idiopathic immune complex-mediated glomerulonephritis: the VALIANT trial (NKF-SCM 2025) - P3 | "There were no deaths, rejection episodes, or graft losses in this post-transplant population.Conclusion In this small group of pts with post-transplant recurrent C3G or IC-MPGN, PEG showed improvement in the 3 key efficacy measures of proteinuria reduction, C3 deposit clearance, and stabilized eGFR. *On behalf of the VALIANT Investigators"

Post-transplantation • Complement-mediated Rare Disorders • Glomerulonephritis • Lupus Nephritis • Nephrology • Transplantation

Switch to Empaveli shows good efficacy in real-world Italian study (PNH News) - "In this report, scientists described the experiences of 22 Italian people with PNH, all adults, who switched to Empaveli after having a suboptimal response to treatment with Soliris or Ultomiris...Within two months of switching to Empaveli, 54.5% of the patients had a complete response, essentially meaning that their disease was fully under control. All but one of the remaining patients had a good response to treatment. At six months after starting Empaveli, all but one of the patients still had a response judged as either complete or good...No unexpected safety issues documented in real-world study."

Real-world evidence • Paroxysmal Nocturnal Hemoglobinuria

VALIANT: Phase III Study Assessing the Efficacy and Safety of Pegcetacoplan in Patients With C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis (clinicaltrials.gov) - P3 | N=124 | Completed | Sponsor: Apellis Pharmaceuticals, Inc. | Active, not recruiting ➔ Completed | N=90 ➔ 124

Enrollment change • Trial completion • Complement-mediated Rare Disorders • Glomerulonephritis • Lupus Nephritis • Nephrology

Pegcetacoplan for Breakthrough Hemolysis in an Adolescent With Classical Paroxysmal Nocturnal Hemoglobinuria on Eculizumab. (PubMed, Pediatr Blood Cancer) - No abstract available

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

Meningococcal Sepsis in Patient with Paroxysmal Nocturnal Hemoglobinuria during Pegcetacoplan Therapy. (PubMed, Emerg Infect Dis) - "A novel complement C3 inhibitor, pegcetacoplan, was recently approved to treat paroxysmal nocturnal hemoglobinuria, a condition commonly treated with complement C5 inhibitors. We present a case of meningococcal sepsis in a pegcetacoplan-treated patient with aplastic anemia and paroxysmal nocturnal hemoglobinuria."

Journal • Aplastic Anemia • Complement-mediated Rare Disorders • Hematological Disorders • Infectious Disease • Meningococcal Infections • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • Septic Shock

Apellis Pharmaceuticals Reports Fourth Quarter and Full Year 2024 Financial Results (GlobeNewswire) - "On track to initiate two Phase 3 trials of EMPAVELI in focal segmental glomerulosclerosis (FSGS) and in delayed graft function (DGF) in 2H 2025...Total revenue was $212.5 million for the fourth quarter of 2024, which consisted of $167.8 million in U.S. net product revenue of SYFOVRE, $23.4 million in U.S. net product revenue of EMPAVELI and $21.4 million in licensing and other revenue associated with the Sobi collaboration...For the full year 2024, total revenue was $781.4 million, which consisted of $611.8 million in U.S. net product revenue of SYFOVRE, $98.1 million in U.S. net product revenue of EMPAVELI and $71.4 million in licensing and other revenue associated with the Sobi collaboration."

Commercial • New P3 trial • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Focal Segmental Glomerulosclerosis • Macular Degeneration • Paroxysmal Nocturnal Hemoglobinuria

Ravulizumab and other complement inhibitors for the treatment of autoimmune disorders. (PubMed, Mult Scler Relat Disord) - "Based on the importance of intermediate and terminal components of the complement cascade during disease pathogenesis, in the last few years, a mAb targeting the C5 complement factor (anti-C5, eculizumab) has already been in use for treating AQP4-IgG positive NMOSD. Here, we discuss the main findings obtained during the phase 3 clinical trial (CHAMPIONNMOSD [NCT0420126]) for ravulizumab and new developments in anti-complement therapy, with other complement inhibitors for the treatment of autoimmune diseases and paroxysmal nocturnal hemoglobinuria (PNH) (zilucoplan, avacopan and, pegcetacoplan). The approval of the new long-acting anti-C5 mAb adds another therapeutic option to the already existing inhibitors of complement currently in use, increasing therapeutic options for inflammatory and autoimmune diseases."

Journal • Review • CNS Disorders • Complement-mediated Rare Disorders • Hematological Disorders • Immunology • Neuromyelitis Optica Spectrum Disorder • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

Apellis and Sobi Announce EMA Validation of Indication Extension Application for Aspaveli (pegcetacoplan) for C3G and Primary IC-MPGN (GlobeNewswire) - "Apellis Pharmaceuticals...today announced the European Medicines Agency (EMA) has validated an indication extension application for Aspaveli (pegcetacoplan) for the treatment of C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN), which are rare, chronic kidney diseases with no approved treatments....The submission includes positive data from the Phase 3 VALIANT study....'Additionally, we continue to advance the regulatory process in the U.S., with a potential launch of pegcetacoplan for C3G and IC-MPGN in the second half of 2025, if approved.'"

EMA filing • Launch US • Glomerulonephritis • Rare Diseases

Pegcetacoplan for the treatment of pediatric complement 3 Glomerulonephritis: A case report (IPNA 2025) - "The patient continued to have nephrotic range proteinuria despite treatment (prednisone 40 mg/day and mycophenolate mofetil 500 mg twice daily for immunosuppression; amlodipine, lisinopril, hydrochlorothiazide, and fluid restriction for difficult-to-manage edema). Conclusions In this pediatric patient, pegcetacoplan treatment was associated with rapid clinical improvement without adverse effects. Clinical effectiveness was confirmed by laboratory and histologic results within 6 months of treatment initiation."

Case report • Clinical • Acute Kidney Injury • Complement-mediated Rare Disorders • Glomerulonephritis • Hematological Disorders • Lupus Nephritis • Nephrology • Paroxysmal Nocturnal Hemoglobinuria • Pediatrics • Rare Diseases • Renal Disease

Pegcetacoplan for adolescents with complement 3 glomerulopathy or primary immune complex-membranoproliferative glomerulonephritis: VALIANT phase 3 double-blind placebo-controlled trial subgroup analysis (IPNA 2025) - P3 | "Conclusions In VALIANT, pegcetacoplan, a novel C3/C3b inhibitor that precisely targets complement dysregulation and overactivation of C3 breakdown driving complement-mediated glomerulopathies, was well tolerated in adolescent patients with C3G and primary IC-MPGN. Pegcetacoplan was the first treatment to induce clinically meaningful proteinuria reduction and eGFR stabilization compared with placebo in adolescent patients with C3G or primary IC-MPGN."

Clinical • P3 data • Complement-mediated Rare Disorders • Glomerulonephritis • Lupus Nephritis • Nephrology • Renal Disease

Thrombosis at Unusual Sites: Focus on Myeloproliferative Neoplasms and Paroxysmal Nocturnal Hemoglobinuria. (PubMed, Hamostaseologie) - "Specific treatments for MPN may include phlebotomy or cytoreductive drugs such as hydroxyurea, anagrelide, pegylated interferon-alpha, or Janus kinase inhibitors. Drugs used for PNH treatment include terminal complement inhibitors, such as eculizumab and ravulizumab, as well as proximally acting inhibitors such as pegcetacoplan or iptacopan. Patients with MPN and PNH are at high risk for thrombosis during their entire lifetime and should thus be followed by specialists experienced in the care of these diseases."

Journal • Cardiovascular • Complement-mediated Rare Disorders • Hematological Disorders • Myeloproliferative Neoplasm • Oncology • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • Thrombosis

Sobi publishes Q4 2024 report: A solid ending to a strong year (PRNewswire) - "Haematology revenue increased 22 per cent at CER to SEK 4,487 M (3,640), mainly driven by strong sales of Doptelet of SEK 1,147 M (727), Vonjo of SEK 416 M (322), Aspaveli/Empaveli of SEK 269 M (186) and launch sales of Altuvoct of SEK 302 M (2); Immunology revenue decreased 12 per cent at CER to SEK 2,564 M (2,905), explained by low Synagis sales of 68 M (897), partially offset by Beyfortus royalty of SEK 1,207 M (890) and sales of Kineret of SEK 777 M (621)."

Sales • Hemophilia A • Idiopathic Arthritis • Immune Thrombocytopenic Purpura • Immunology • Infectious Disease • Inflammatory Arthritis • Myelofibrosis • Myeloproliferative Neoplasm • Paroxysmal Nocturnal Hemoglobinuria • Rheumatoid Arthritis • Thrombocytopenia • Thrombocytopenic Purpura

Pegcetacoplan for the Treatment of Paediatric C3 Glomerulonephritis: A Case Report. (PubMed, Nephrology (Carlton)) - "Despite treatment with intense immunosuppression (high-dose steroids, mycophenolate mofetil and calcineurin inhibitor), he continued to have high disease activity with low C3 levels (35 mg/dL), hypertension, symptomatic oedema, anaemia, and nephrotic-range proteinuria (e.g., urine protein-to-creatinine ratio [uPCR], 10 g/g; serum creatinine, 0.4 mg/dL). A kidney biopsy after 6 months of pegcetacoplan treatment showed mesangial and focal endocapillary proliferative glomerulonephritis with isolated C3c deposition by immunofluorescence, consistent with previous C3GN diagnosis. In this paediatric patient, compassionate use of pegcetacoplan was associated with rapid clinical improvement without adverse effects, and clinical effectiveness was confirmed by laboratory and histologic results within 6 months of treatment initiation."

Journal • Cardiovascular • Complement-mediated Rare Disorders • Glomerulonephritis • Hematological Disorders • Hypertension • Lupus Nephritis • Nephrology • Pediatrics • Renal Disease

C3 GLOMERULOPATHY RARE DISEASE AND NEW THERAPUTIC APPROACH (ISN-WCN 2025) - "Subsequently the patient initiated on supportive treatment in the form maximum tolerated dose of angiotensin receptor blockade also sGLT2i added .patient also received prednisolone and Mycophenolate Mofetile MMF...Many cases seriessupportthe use of anti-C5, Eculizumab, as a therapeutic approach in a subset of patients...Clinical trials which showed promising results in treating C3 GN with Iptacopan and Pegcetacoplan...Treatment is still not optimal with MMF and steroid widely used with poor clinical responses. New anti-complement medications are in the near horizon, however more studies will be needed for treatment standardization."

Cardiovascular • Chronic Kidney Disease • Complement-mediated Rare Disorders • Diabetes • Diabetic Nephropathy • Fatigue • Glomerulonephritis • Hepatitis B • Hepatitis C • Hepatology • Hypertension • Immunology • Infectious Disease • Inflammation • Inflammatory Arthritis • Lipodystrophy • Lupus Nephritis • Metabolic Disorders • Monoclonal Gammopathy • Pain • Rare Diseases • Renal Calculi • Retinal Disorders • Rheumatology