- |||||||||| cemdisiran (ALN-CC5) / Alnylam, Regeneron, Veopoz (pozelimab) / Regeneron
Trial completion date, Trial termination, Trial primary completion date, Combination therapy: ACCESS 2: Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (clinicaltrials.gov) - Aug 16, 2023 P3, N=2, Terminated, R. Trial completion date: Sep 2026 --> Jul 2023 | Active, not recruiting --> Terminated | Trial primary completion date: Jun 2025 --> Jul 2023; Sponsor decision not related to efficacy or safety.
- |||||||||| Review, Journal: Biologic drugs in the treatment of juvenile dermatomyositis: a literature review. (Pubmed Central) - Aug 15, 2023
The most frequently used biologic drugs were rituximab (RTX, 50%) and tumor necrosis factor (TNF) inhibitors (34.8%). In a few cases, abatacept (4.3%), anti-interleukin-1 agents (0.9%), tocilizumab (0.9%), bortezomib (0.4%), ustekinumab (0.2%), eculizumab (0.2%), and golimumab (0.2%) were used...Infliximab was most frequently preferred in calcinosis (43.3%), while adalimumab in skin involvement (50%) and etanercept in resistant/recurrent diseases (80%)...
- |||||||||| Soliris (eculizumab) / AstraZeneca
Review, Journal: New findings in preventing recurrence and improving renal function in AHUS patients after renal transplantation treated with eculizumab: a systemic review and meta-analyses. (Pubmed Central) - Aug 15, 2023 In a few cases, abatacept (4.3%), anti-interleukin-1 agents (0.9%), tocilizumab (0.9%), bortezomib (0.4%), ustekinumab (0.2%), eculizumab (0.2%), and golimumab (0.2%) were used...Infliximab was most frequently preferred in calcinosis (43.3%), while adalimumab in skin involvement (50%) and etanercept in resistant/recurrent diseases (80%)... Furthermore, the drug was effective in improving platelet counts (
- |||||||||| Soliris (eculizumab) / AstraZeneca, Rituxan (rituximab) / Biogen, Zenyaku Kogyo, Roche
Journal: Focused Jurisdictional Scan of Glomerulonephritis Medication Access in Canada: A Program Report. (Pubmed Central) - Aug 15, 2023 Interprovincial differences in the drugs funded, funding criteria, and application process may affect timely and equitable access to GN medications across Canada. Given the rarity of GN, a pan-Canadian funding approach may be warranted to improve the current state.
- |||||||||| Journal: Advancements in Targeted Therapies for Generalized Acetylcholine Receptor Antibody Positive Myasthenia Gravis: Beginnings of a Paradigm Shift. (Pubmed Central) - Aug 14, 2023
Compared to traditional immune modulatory therapies (steroids, non-steroidal immunosuppressive agents), these agents have more specific target engagement via inhibition of complement activation (eculizumab, ravulizumab, zilucoplan) or blockade of IgG recycling via the neonatal Fc receptor (FcRn; efgartigimod, rozanolixizumab)...B-cell targeting with rituximab was safe and well-tolerated but with less impressive efficacy in two phase 2 studies...A paradigm shift is afoot. The challenge is figuring out where these therapies may fit in gMG treatment algorithms.
- |||||||||| Soliris (eculizumab) / AstraZeneca, Rituxan (rituximab) / Biogen, Zenyaku Kogyo, Roche
Review, Journal: Antiphospholipid Syndrome: State of the Art of Clinical Management. (Pubmed Central) - Aug 12, 2023 Finally, it has been observed that SARS COV2 infection may produce vascular complications mimicking the clinical and pathophysiological features of APS and particularly of CAPS. From this point of view, attention has been focused on the "protective" role of anticoagulant therapy in preventing thrombotic complication when these clinical conditions coexist.
- |||||||||| Voydeya (danicopan) / AstraZeneca, Soliris (eculizumab) / AstraZeneca, Ultomiris (ravulizumab-cwvz) / AstraZeneca
New trial: Danicopan Early Access Program (clinicaltrials.gov) - Aug 9, 2023 P=N/A, N=0, Available,
- |||||||||| Soliris (eculizumab) / AstraZeneca
Review, Journal, PD(L)-1 Biomarker, IO biomarker: Antibody-mediated rejection in xenotransplantation: Can it be prevented or reversed? (Pubmed Central) - Aug 7, 2023 Potential therapies include (i) inhibition of complement activation (e.g., by IVIg, C1 INH, or an anti-C5 antibody), but some complement inhibitors are not effective in NHPs, for example, eculizumab...We suggest that treatment with a systemic complement inhibitor is likely to be most effective, possibly combined with anti-inflammatory agents (if these are not already being administered). Ultimately, it may require further genetic engineering of the organ-source pig to resolve the problem entirely, for example, knockout or knockdown of SLA, and/or expression of PD-L1, HLA E, and/or HLA-G.
- |||||||||| Soliris (eculizumab) / AstraZeneca, Ultomiris IV (ravulizumab-cwvz IV) / AstraZeneca
Journal, HEOR, Real-world evidence, Cost of care, Real-world: Descriptive, real-world treatment patterns, resource use, and total cost of care among eculizumab- and ravulizumab-treated members with paroxysmal nocturnal hemoglobinuria. (Pubmed Central) - Aug 7, 2023 Dr Burke reports employment by Prime Therapeutics, LLC, which has received research funding from Apellis Pharmaceuticals. Dr Gleason reports employment by Prime Therapeutics, LLC, which has received research funding from Apellis Pharmaceuticals; serves on the advisory committee at the Institute for Clinical and Economic Review; and has served on the Board of Directors at the Academy of Managed Care Pharmacy.
- |||||||||| Review, Journal: Molecular pharmacology in complement-mediated hemolytic disorders. (Pubmed Central) - Aug 7, 2023
Finally, aHUS is an indication for complement inhibitors. Eculizumab and ravulizumab have been approved, whilst other C5 inhibitors, and novel lectin pathway inhibitors are under active investigation in this disease.
- |||||||||| Soliris (eculizumab) / AstraZeneca, Rituxan (rituximab) / Biogen, Zenyaku Kogyo, Roche
Preclinical, Journal: Anti-complement 5 antibody ameliorates antibody-mediated rejection after liver transplantation in rats. (Pubmed Central) - Jul 7, 2023 Antibody-mediated rejection (AMR) remains a refractory rejection after donor-specific antibody (DSA)-positive or blood-type incompatible liver transplantation (LT), even in the era of pre-transplant rituximab desensitization...Notably, just two doses of Anti-C5 only on PTD-0 and -3 significantly improved biliary injury and liver fibrosis up to PTD-100, leading to better long-term animal survival (P =0.02). We newly developed a rat model of LT-AMR that meets all the Banff diagnostic criteria and demonstrated the efficacy of Anti-C5 antibody for LT-AMR.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Journal, HEOR: Eculizumab for paroxysmal nocturnal haemoglobinuria: catastrophic health expenditure in Nepalese patients. (Pubmed Central) - Jul 5, 2023 Eculizumab is a terminal complement inhibitor that blocks this complement activation and has revolutionized the treatment of PNH but comes with an enormous price which can have catastrophic health expenditure in low-middle income countries (LMIC) like Nepal. Here, we discuss the potential way forwards in the treatment of PNH in Nepal and other LMICs.
- |||||||||| Soliris (eculizumab) / AstraZeneca, Empaveli (pegcetacoplan SC) / Apellis, SOBI
Review, Journal: Treatment of eculizumab refractory paroxysmal nocturnal hemoglobinuria: A systematic review about current treatment options and future direction. (Pubmed Central) - Jun 30, 2023 C5 inhibitors such as eculizumab and ravulizumab are the first-line treatment in the management of paroxysmal nocturnal hemoglobinuria (PNH)...Two studies were about pegcetacoplan, one was about danicopan, and one was about iptacopan...More studies using study designs such as randomized controlled trials comparing multiple drugs should be performed to accurately assess the different medications and aid in designing guidelines of the management of eculizumab refractory PNH. Level I.
- |||||||||| Tavneos (avacopan) / Kissei, Amgen, Otsuka, Soliris (eculizumab) / AstraZeneca
Journal: C5a-licensed phagocytes drive sterilizing immunity during systemic fungal infection. (Pubmed Central) - Jun 26, 2023 Lower serum C5a concentrations or a C5 polymorphism that decreases leukocyte C5 expression correlated independently with poor patient outcomes. Thus, local, phagocyte-derived C5 production licenses phagocyte antimicrobial function and confers innate protection during systemic fungal infection.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Trial completion date, Trial primary completion date: The Use of Eculizumab in HELLP Syndrome (clinicaltrials.gov) - Jun 23, 2023 P1, N=3, Suspended, Thus, local, phagocyte-derived C5 production licenses phagocyte antimicrobial function and confers innate protection during systemic fungal infection. Trial completion date: Jun 2023 --> Sep 2023 | Trial primary completion date: May 2023 --> Aug 2023
- |||||||||| Soliris (eculizumab) / AstraZeneca, Opdivo (nivolumab) / Ono Pharma, BMS, Rituxan (rituximab) / Biogen, Zenyaku Kogyo, Roche
Journal: Global research hotspots and frontiers of myasthenia gravis from 2002 to 2021: A bibliometric study. (Pubmed Central) - Jun 20, 2023 The study also identified pathogenesis, eculizumab, thymic epithelial cells, immune checkpoint inhibitors, thymectomy, MuSK antibodies, risk, diagnosis, and management as the current hot research topics in MG, while burst keywords like quality of life, immune-related adverse events (irAEs), rituximab, safety, nivolumab, cancer, and classification indicated the frontiers of MG research. This study effectively identifies the hotspots and frontiers of MG research, and offers valuable references for researchers interested in this area.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Review, Journal: Caring for two in the ICU: pharmacologic management of pregnancy-related complications. (Pubmed Central) - Jun 16, 2023 Brief descriptions of non-pharmacological (e.g., cesarean or vaginal delivery of the baby) interventions are provided. Mainstays of pharmacotherapy highlighted include oxytocin for obstetric hemorrhage, methotrexate for ectopic pregnancy, magnesium and antihypertensive agents for preeclampsia and eclampsia, eculizumab for atypical hemolytic uremic syndrome, corticosteroids and immunosuppressive agents for thrombotic thrombocytopenic purpura, diuretics, metoprolol and anticoagulation for peripartum cardiomyopathy, and pulmonary vasodilators for amniotic fluid embolism.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Journal: The Effect of Respiratory Viral Infections on Breakthrough Hemolysis in Patients with Paroxysmal Nocturnal Hemoglobinuria. (Pubmed Central) - Jun 14, 2023 We retrospectively analyzed 34 patients with PNH disease between 2016 and 2018, who were on eculizumab treatment and who presented with respiratory symptoms and were subsequently tested for 10 respiratory viruses (influenza A, influenza B, parainfluenza, respiratory syncytial virus, adenovirus, rhinovirus, and human metapneumovirus)...Our data indicate that respiratory virus infections pose a significant risk for BTH in PNH patients on complement inhibitor treatment, underlining the need for regular screening and close monitoring of patients with respiratory symptoms. Furthermore, it implies a higher risk for patients who are not established on complement inhibitors, suggesting the necessity for greater vigilance in these patients.
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