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85 Diseases |  |
26 Trials |
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26 Trials |  |
4609 News |  |
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- Ultomiris (ravulizumab-cwvz) / AstraZeneca
Ravulizumab Provides Durable Control of Intravascular Hemolysis and Improves Survival in Patients with Paroxysmal Nocturnal Hemoglobinuria: Long-Term Follow-up of Study 301 and Comparisons with Patients of the International PNH Registry (Halls G-H (San Diego Convention Center)) - Nov 3, 2023 - Abstract #ASH2023ASH_3657;
P=N/A, P3
C5 inhibitor?na
- Soliris (eculizumab) / AstraZeneca
Efficacy and Safety of Subcutaneous Ravulizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Received Prior Intravenous Eculizumab: 2-Year Follow-up (SDCC - Halls G-H) - Nov 3, 2023 - Abstract #ASH2023ASH_3656;
P3
The efficacy of ravulizumab was maintained following the switch from IV to SUBQ ravulizumab and no new safety signals were identified through 2 years of treatment in patients with PNH. SUBQ ravulizumab demonstrated a favorable benefit
- danicopan (ALXN2040) / AstraZeneca, Soliris (eculizumab) / AstraZeneca, Ultomiris (ravulizumab-cwvz) / AstraZeneca
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 (Room 7 (San Diego Convention Center)) - Nov 3, 2023 - Abstract #ASH2023ASH_3251;
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.
- Soliris (eculizumab) / AstraZeneca, crovalimab (RG6107) / Roche
Safety of Crovalimab Versus Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH): Pooled Results from the Phase III COMMODORE 1, COMMODORE 2, and COMMODORE 3 Studies (Room 7 (San Diego Convention Center)) - Nov 3, 2023 - Abstract #ASH2023ASH_3250;
P3
The safety profile was consistent between pediatric and adult pts. Conclusions Overall, pooled safety data from COMMODORE 1, 2, and 3 showed that the safety profile of crova was consistent with that of ecu in pts with PNH, and comparable between pts who were C5i
- Fabhalta (iptacopan) / Novartis, Soliris (eculizumab) / AstraZeneca, crovalimab (RG6107) / Roche
The Change of Complement Deposition after Complement Inhibitor Treatment in Paroxysmal Nocturnal Hemoglobinuria Patients (Halls G-H (San Diego Convention Center)) - Nov 3, 2023 - Abstract #ASH2023ASH_1753;
Unlike C5 inhibitors, iptacopan also reduced the deposition of C3, C4b, and FB. C5b-9+/RBC may be correlated with the efficacy of inhibiting intravascular hemolysis.
- danicopan (ALXN2040) / AstraZeneca, Soliris (eculizumab) / AstraZeneca, Ultomiris (ravulizumab-cwvz) / AstraZeneca
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 (Halls G-H (San Diego Convention Center)) - Nov 3, 2023 - Abstract #ASH2023ASH_1749;
P=N/A, 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.
- Fabhalta (iptacopan) / Novartis
Clinical Breakthrough Hemolysis (BTH) during Monotherapy with the Oral Factor B Inhibitor Iptacopan Is Generally Not Severe and Managed without Treatment Discontinuation: 48-Week Data from the Phase III APPLY-PNH and APPOINT-PNH Trials in Paroxysmal Nocturnal Hemoglobinuria (PNH) (Halls G-H (San Diego Convention Center)) - Nov 3, 2023 - Abstract #ASH2023ASH_1741;
P3
Most clinical BTH events on iptacopan were associated with potential CACs; BTH was manageable with no intervention or RBC transfusion and without iptacopan discontinuation. The exposure-adjusted occurrence rates support our prior finding of a significantly lower adjusted annualized rate of clinical BTH with iptacopan vs anti-C5.
- Soliris (eculizumab) / AstraZeneca
Anticoagulation Use and Patient Outcomes in Paroxysmal Nocturnal Hemoglobinuria: A Province-Wide Retrospective Review (SDCC - Halls G-H) - Nov 3, 2023 - Abstract #ASH2023ASH_1681;
Our practice patterns indicate that DOACs are being used in our province as primary prophylaxis, even in patients who are concurrently being treated with a complement inhibitor. DOACs are also being used more frequently than other anticoagulants as secondary prophylaxis, despite there not being robust data for DOAC use in this patient population.
- || Soliris (eculizumab) / AstraZeneca, Ultomiris IV (ravulizumab-cwvz IV) / AstraZeneca
Review, Journal: Eculizumab Versus Ravulizumab for the Treatment of Atypical Hemolytic Uremic Syndrome: A Systematic Review. (Pubmed Central) - Oct 31, 2023
Treatment with a monoclonal antibody should be initiated as soon as a genetic mutation is identified. Blinded, double-arm, clinical trials preferably with larger sample sizes are needed to effectively compare both the monoclonal antibodies.
- | Soliris (eculizumab) / AstraZeneca
Journal, Real-world evidence, Real-world: Long-Term Efficacy and Safety of Eculizumab in Patients With Paroxysmal Nocturnal Hemoglobinuria and High Disease Burden: Real-World Data From Korea. (Pubmed Central) - Oct 30, 2023
Blinded, double-arm, clinical trials preferably with larger sample sizes are needed to effectively compare both the monoclonal antibodies. These data provided evidence for the long-term efficacy and safety of eculizumab in Korean PNH patients with high disease burdens.
- || Benlysta (belimumab) / GSK, Soliris (eculizumab) / AstraZeneca, Rituxan (rituximab) / Biogen, Zenyaku Holdings, Roche
Review, Journal: Clinical advances in immunotherapy for immune-mediated glomerular diseases. (Pubmed Central) - Oct 27, 2023
Certain immunotherapeutic drugs, such as rituximab, belimumab, and eculizumab, have exhibited notable efficacy in treating specific immune-mediated glomerular diseases, thereby providing novel therapeutic approaches for patients. Nonetheless, the efficacy of numerous immunotherapeutic drugs remains to be substantiated.
- || Ultomiris IV (ravulizumab-cwvz IV) / AstraZeneca
Biomarker, Clinical, Review, Journal: Non-capsulated Neisseria meningitidis sepsis in a paroxysmal nocturnal hemoglobinuria patient treated with ravulizumab: case report and review of the literature. (Pubmed Central) - Oct 24, 2023
Increasing awareness regarding septic risk in PNH patients on complement inhibitors despite vaccinations is pivotal. A warning about serotypes generally not pathogenetic and not covered by vaccination, such as non-capsulated forms, is emerging.
- || Soliris (eculizumab) / AstraZeneca, Ultomiris IV (ravulizumab-cwvz IV) / AstraZeneca, Zilbrysq (zilucoplan) / UCB
Review, Journal: Role of complement in myasthenia gravis. (Pubmed Central) - Oct 24, 2023
Complement therapies were initially studied and approved for paroxysmal nocturnal hemoglobinuria and in the past decade, those have also been studied in myasthenia gravis. The currently available randomized control trial and real-world data on the efficacy and safety of the approved and investigational complement therapies are summarized in this review.
- | Soliris (eculizumab) / AstraZeneca
New trial: m-TMA: Study of Patients With Thrombotic Microangiopathy Associated With Mitomycin C, Treated or Not With Eculizumab (clinicaltrials.gov) - Oct 23, 2023
P=N/A, N=30, Recruiting, Sponsor: University Hospital, Strasbourg, France
- | Uplizna (inebilizumab) / Mitsubishi Tanabe, Amgen, Soliris (eculizumab) / AstraZeneca, Enspryng (satralizumab) / Roche
FDA event, Journal: Utilization of FDA approved treatments for neuromyelitis optica spectrum disorder in clinical practice: A survey study of academic neuroimmunologists. (Pubmed Central) - Oct 23, 2023
Neuromyelitis optica spectrum disorder (NMOSD) is a rare autoimmune condition for which three treatments have been approved since 2019: eculizumab, inebilizumab, and satralizumab. Following relapse, respondents were dichotomized, either switching 75-100
- | Soliris (eculizumab) / AstraZeneca
Journal: Intermediate-term Outcomes of Complement Inhibition for Prevention of Antibody-Mediated Rejection in Immunologically High-risk Heart Allograft Recipients. (Pubmed Central) - Oct 23, 2023
P3
Our results support the utility of complement inhibition for high-immunological risk recipients. DATA AVAILABILITY STATEMENT: The data that support the findings of this study are available from the corresponding author upon reasonable request.
- | Soliris (eculizumab) / AstraZeneca
Journal: Eculizumab use in patients with pneumococcal-associated hemolytic uremic syndrome and kidney outcomes. (Pubmed Central) - Oct 22, 2023
Kidney outcomes are strongly correlated with the duration of dialysis and mechanical ventilation duration. A higher resolution version of the Graphical abstract is available as Supplementary information.
- | Soliris (eculizumab) / AstraZeneca, Ultomiris IV (ravulizumab-cwvz IV) / AstraZeneca
Journal, HEOR, Cost-effectiveness, Cost effectiveness: Cost-effectiveness of ravulizumab compared with eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria in the Netherlands. (Pubmed Central) - Oct 22, 2023
A higher resolution version of the Graphical abstract is available as Supplementary information. The cost reduction and QALY gain associated with the lower rates of BTH and less frequent administration make ravulizumab a cost-saving and clinically beneficial substitute for eculizumab for adults with PNH in the Netherlands.
- | Soliris (eculizumab) / AstraZeneca
Journal: Thrombotic microangiopathy due to primary antiphospholipid syndrome: successful treatment with eculizumab. (Pubmed Central) - Oct 17, 2023
Whereas in secondary antiphospholipid syndrome, e.g. to systemic lupus erythematosus, there is emerging evidence of a benefit from complement blockade with eculizumab, optimal treatment of primary antiphospholipid syndrome-associated thrombotic microangiopathy is currently unknown. We report the case of a 36-year-old male patient with primary antiphospholipid syndrome-associated thrombotic microangiopathy, presenting with a clinical picture of atypical hemolytic-uremic syndrome with frequent relapses, treated with eculizumab (four 900
- ||||| Soliris (eculizumab) / AstraZeneca, Ultomiris IV (ravulizumab-cwvz IV) / AstraZeneca, Ultomiris SC QW (ravulizumab-cwvz SC) / AstraZeneca
Trial completion, Head-to-Head: Ravulizumab Subcutaneous (SC) Versus Ravulizumab Intravenous (IV) in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab (clinicaltrials.gov) - Oct 13, 2023
P3, N=136, Completed, Sponsor: Alexion
We report the case of a 36-year-old male patient with primary antiphospholipid syndrome-associated thrombotic microangiopathy, presenting with a clinical picture of atypical hemolytic-uremic syndrome with frequent relapses, treated with eculizumab (four 900 Active, not recruiting --> Completed
- | Uplizna (inebilizumab) / Horizon Therapeutics, Mitsubishi Tanabe
Journal, HEOR: Inebilizumab for neuromyelitis optica spectrum disorder in Italy: a budget impact model. (Pubmed Central) - Oct 5, 2023
Inebilizumab reduces the INHS expenditure for NMOSD drugs. Future research should explore the cost-effectiveness of inebilizumab vs other NMOSD-targeting drugs in Italy.
- | Enjaymo (sutimlimab) / Sanofi, Soliris (eculizumab) / AstraZeneca
Journal: Novel therapeutic agents for hemolytic anemia (Pubmed Central) - Oct 4, 2023
In addition, the anti-C1s antibody sutimlimab was approved last year for the treatment of cold agglutinin disease, a form of autoimmune hemolytic anemia. This presentation overviews novel anti-complement agents for these hemolytic anemias.
- | Soliris (eculizumab) / AstraZeneca
Journal: Optimizing the Long-Term Therapy with Eculizumab (ECU) for Atypical Hemolytic Uremic Syndrome (aHUS). (Pubmed Central) - Sep 30, 2023
This presentation overviews novel anti-complement agents for these hemolytic anemias. No abstract available
- Ultomiris IV (ravulizumab-cwvz IV) / AstraZeneca
Long-term ravulizumab treatment in complement inhibitor-experienced patients with pnh provides durable control of intravascular hemolysis with low incidence of major adverse vascular events and death (Y1) - Sep 30, 2023 - Abstract #DGHO2023DGHO_1479;
P3
To date, this study reports the longest duration of ravulizumab treatment exposure in C5 inhibitor-experienced patients with PNH (527.04 patient-years). Effective control of intravascular hemolysis with ravulizumab was reflected by LDH levels being maintained at <1.5
- Soliris (eculizumab) / AstraZeneca, crovalimab (RG6107) / Roche
The phase iii, randomized, multicenter commodore 2 trial: results from the study of crovalimab vs eculizumab in paroxysmal nocturnal hemoglobinuria (PNH) patients naive to complement inhibitors (Y1) - Sep 30, 2023 - Abstract #DGHO2023DGHO_1468;
P3
The COMMODORE 2 study demonstrated that crova was non-inferior to ecu in both co-primary efficacy endpoints and had a comparable safety profile. These data highlight the overall favorable benefit
- Soliris (eculizumab) / AstraZeneca, crovalimab (RG6107) / Roche
The phase iii randomized, multicenter commodore 1 trial: comparison of crovalimab vs eculizumab in complement inhibitor-experienced patients with paroxysmal nocturnal hemoglobinuria (PNH) (Hall F) - Sep 30, 2023 - Abstract #DGHO2023DGHO_616;
P3
The safety profile of crova in C5i-experienced patients with PNH is consistent with that in C5i-naive patients, except for transient, self-limiting T3H events unique to patients switching between crova and ecu. The COMMODORE 1 data comparing crova vs ecu in C5i-experienced patients support the favorable benefit
- iptacopan (LNP023) / Novartis, Soliris (eculizumab) / AstraZeneca, Ultomiris IV (ravulizumab-cwvz IV) / AstraZeneca
Efficacy and safety of oral therapy with iptacopan compared to standard intravenous therapy with eculizumab or ravulizumab in the treatment of paroxysmal nocturnal hemoglobinuria (PNH): Results of the randomized controlled open-label multicenter phase III study APPLY-PNH (X4) - Sep 30, 2023 - Abstract #DGHO2023DGHO_388;
P3
In general, iptacopan was well tolerated. It could be a viable alternative in the future for patients who respond inadequately to SoC treatment.
- | Soliris (eculizumab) / AstraZeneca, Rituxan (rituximab) / Biogen, Zenyaku Holdings, Roche
Journal: Immunosuppressive Therapy of Antibody-Mediated aHUS and TTP. (Pubmed Central) - Sep 28, 2023
In the aHUS cases diagnosed and treated before the eculizumab era, CFH-ab was detected using the ELISA assay...Treatment protocol consisted of plasma exchanges (PE) and steroid pulses, followed by the combination of cyclophosphamide and rituximab to achieve long-term immunosuppression...In the cases of antibody-mediated TMAs, PE and early immunosuppressive treatment may result in sustained remission with preserved kidney function. Further data are needed to establish optimal treatment of anti-FH antibody-associated HUS.
- | Sputnik-V (Gam-COVID-Vac Lyo) / Gamaleya Research Institute
Journal: The Features of COVID-19's Course and the Efficacy of the Gam-COVID-Vac Vaccine in Patients with Paroxysmal Nocturnal Hemoglobinuria. (Pubmed Central) - Sep 27, 2023
The study included data from 52 patients with PNH aged 18 to 75 years, 38 of whom received background therapy with eculizumab (Elizaria) between March 2020 and January 2022...After the second dose, no AEs were reported. The performed study suggests the possible efficacy and demonstrates the safety of Gam-COVID-Vac (Sputnik V) for the prophylaxis of COVID-19 in patients with PNH who experience immunosuppression due to target therapy.
- | Soliris (eculizumab) / AstraZeneca, Rituxan (rituximab) / Biogen, Zenyaku Holdings, Roche
Journal: Diagnosis and treatment of glomerular diseases with a membranoproliferative glomerulonephritis (MPGN) pattern of injury (Pubmed Central) - Sep 24, 2023
Rapidly progressive MPGN should be treated like ANCA-associated vasculitis. The recurrence rates after kidney transplantation are very high and treatment is challenging.
- Soliris (eculizumab) / AstraZeneca
Understanding Sickle Cell Pathophysiology (Room 33 (San Diego Convention Center)) - Sep 23, 2023 - Abstract #ASH2023ASH_155;
We will also present data on cases collection of SCD patients with severe sickle cell related clinical manifestations treated with eculizumab, a complement inhibitor...Jeffrey Chalmers notes that while a variety of cell therapy approaches are being pursued to cure SCD, there are still significant needs, which will continue to exist, for improved technology monitor, and evaluate SCD patients. Multiple novel testing techniques in the field including RBC health evaluation, Gazelle, and measurement of adhesion will be presented/discussed.
- | Journal: Complement inhibitors in pediatric kidney diseases: new therapeutic opportunities. (Pubmed Central) - Sep 21, 2023
Many of these molecules have been shown to be effective in reducing proteinuria and stabilizing kidney function in different complement-mediated kidney diseases. Thanks to their efficacy and target specificity, these novel drugs may radically improve the outcome of complement-mediated kidney diseases, contributing to an improvement in our understanding of their underlying pathophysiology.
- | Soliris (eculizumab) / AstraZeneca
Journal, Cytokine storm: Eculizumab suppresses zymosan-induced release of inflammatory cytokines IL-1?, IL-1?, IFN-? and IL-2 in autologous serum-substituted PBMC cultures: Relevance to cytokine storm in Covid-19. (Pubmed Central) - Sep 18, 2023
The clinically relevant findings include the abundant formation of the chemokine IL-8, which was stimulated by C5a, and the suppression of numerous inflammatory cytokines by eculizumab, which explains its therapeutic efficacy in severe Covid-19. These data strengthen the clinical relevance of the applied PBMC model for drug screening against CS, enabling the separation of complex innate immune cross-talks.
- | Soliris (eculizumab) / AstraZeneca
Journal: Long-term effectiveness of eculizumab: Data from the International PNH Registry. (Pubmed Central) - Sep 15, 2023
P=N/A
These data strengthen the clinical relevance of the applied PBMC model for drug screening against CS, enabling the separation of complex innate immune cross-talks. Using data from the largest Registry of patients with PNH, with ?14?years of overall follow-up, we demonstrate that treatment with eculizumab conferred a 49% relative benefit in survival and an approximately 60% reduction in TE/MAVE risk.
- || Soliris (eculizumab) / AstraZeneca
Review, Journal: The Rationale of Complement Blockade of the MCP Haplotype following Atypical Hemolytic Uremic Syndrome of Three Southeastern European Countries with a Literature Review. (Pubmed Central) - Sep 13, 2023
The median number of relapses before CBT initiation (Eculizumab) was two...However, the duration of such a blockade remains uncertain. If complement inhibition is not applied within 4-5 relapses, proteinuria and chronic renal failure will eventually occur.
- ||| Elizaria (eculizumab biosimilar) / Generium
Trial completion: NAP: Prospective Observational Study of Long-term Pathogenic Treatment of Elizaria (clinicaltrials.gov) - Sep 13, 2023
P=N/A, N=44, Completed, Sponsor: AO GENERIUM
If complement inhibition is not applied within 4-5 relapses, proteinuria and chronic renal failure will eventually occur. Active, not recruiting --> Completed
- | Soliris (eculizumab) / AstraZeneca
Trial completion date, Trial initiation date, Trial primary completion date: HYPERSHU: Eculizumab in Hypertensive Emergency-associated Hemolytic Uremic Syndrome (clinicaltrials.gov) - Sep 11, 2023
P3, N=66, Not yet recruiting, Sponsor: Assistance Publique - H
Active, not recruiting --> Completed Trial completion date: Jun 2026 --> Oct 2027 | Initiation date: Jun 2023 --> Oct 2023 | Trial primary completion date: Dec 2025 --> Apr 2026
- | NM8074 / NovelMed
Trial completion date, Trial initiation date, Trial primary completion date: Study of NM8074 in Adult PNH Patients With Inadequate Response to Soliris (clinicaltrials.gov) - Sep 11, 2023
P2, N=12, Not yet recruiting, Sponsor: NovelMed Therapeutics
Trial completion date: Jun 2026 --> Oct 2027 | Initiation date: Jun 2023 --> Oct 2023 | Trial primary completion date: Dec 2025 --> Apr 2026 Trial completion date: Nov 2024 --> Dec 2025 | Initiation date: Sep 2023 --> Mar 2024 | Trial primary completion date: Oct 2024 --> Aug 2025
- ||| Soliris (eculizumab) / AstraZeneca, Rituxan (rituximab) / Biogen, Zenyaku Holdings, Roche
Journal, Adverse events, Biopsy: The need for kidney biopsy in the management of side effects of target and immunotherapy. (Pubmed Central) - Sep 8, 2023
A patient with severe thrombotic microangiopathy requiring dialysis received a treatment with eculizumab for 3 months...Cancer treatment was resumed without change in 21 out of 30 patients. Kidney biopsy is critical for the management of kidney toxicities and should be strongly encouraged for patients showing adverse kidney effects of novel cancer agents.
- | Soliris (eculizumab) / AstraZeneca
Journal: COVID-19 associated thrombotic microangiopathy. (Pubmed Central) - Sep 7, 2023
In conclusion, TMA may be associated with COVID-19 infection. Treatment with eculizumab may be beneficial in selected patients because of the potential activation of the complement system.
- The safety and efficacy advantage of blocking C5a vs C5 in critically ill, COVID-19 patients: Results from PANAMO, a Phase 3 randomized controlled trial (102 AB) - Sep 6, 2023 - Abstract #IDWeek2023IDWeek_767;
- | Soliris (eculizumab) / AstraZeneca
Journal: Severe hemolytic exacerbations of Chinese PNH patients infected SARS-CoV-2 Omicron. (Pubmed Central) - Sep 3, 2023
Treatment with eculizumab may be beneficial in selected patients because of the potential activation of the complement system. Hemolytic exacerbation of PNH with COVID-19 is severe and eculizumab may be an effective treatment.
- | Soliris (eculizumab) / AstraZeneca, Avastin (bevacizumab) / Roche
Journal: Bevacizumab-Associated Thrombotic Microangiopathy Treated with Eculizumab: A Case Report. (Pubmed Central) - Sep 3, 2023
CONCLUSIONS Bevacizumab-associated TMA can be reversed or attenuated in some patients with the use of eculizumab (inhibiting complement system overactivation), possibly reducing time to recovery, with fewer long-term sequelae. This additional case encourages future clinical trials to evaluate the safety and efficacy of eculizumab in cases of TMA associated with bevacizumab.
- | Empaveli (pegcetacoplan SC) / Apellis, SOBI
Journal, HEOR, Cost-effectiveness, Cost effectiveness: The cost-effectiveness of pegcetacoplan in complement treatment-na (Pubmed Central) - Sep 1, 2023
P3
Pegcetacoplan was associated with lifetime cost savings of USD1,176,808 and USD213,062 relative to eculizumab and ravulizumab, respectively (largely attributed to reduced drug costs and blood transfusions), and additional quality-adjusted life years (QALYs) of 0.25 and 0.24. In patients with PNH who are treatment-naive, the base-case cost-effectiveness analysis, scenario analysis and sensitivity analysis showed both lifetime cost savings and increased QALYs associated with pegcetacoplan compared with eculizumab or ravulizumab in the USA.
- || Review, Journal: A Clinical Approach to Existing and Emerging Therapeutics in Neuromyelitis Optica Spectrum Disorder. (Pubmed Central) - Aug 31, 2023
Choosing a maintenance therapy remains nuanced depending on patient factors and accessibility. With over 100 putative agents in trials, disease-free survival is now a realistic goal for NMOSD patients.
- | zilucoplan (RA101495) / UCB
Journal: Zilucoplan, a macrocyclic peptide inhibitor of human complement component 5, uses a dual mode of action to prevent terminal complement pathway activation. (Pubmed Central) - Aug 29, 2023
Our findings demonstrate that zilucoplan uses a dual mode of action to potently inhibit the activation of C5 and terminal complement pathway including wild-type and clinical R885 variants that do not respond to eculizumab treatment. These data may be relevant to the clinically demonstrated benefits of zilucoplan.
- Soliris (eculizumab) / AstraZeneca
HELLP As a Trigger For Atypical Hemolytic Uremic Syndrome (Exhibit Hall) - Aug 23, 2023 - Abstract #ASA2023ASA_4129;
37F G6P5 with a PMH of methamphetamine use and no prenatal care BIBA for decompensation found to have acute renal failure, elevated liver enzymes, and hemolysis concerning for HELLP syndrome...Renal biopsy confirmed TMA, and she underwent empirical treatment with PLEX and eculizumab for aHUS...These disorders overlap and can be difficult to distinguish. There are few case reports describing HELLP as a trigger for aHUS.
- | Empaveli (pegcetacoplan SC) / Apellis, SOBI
Journal: Normalisation of haemoglobin and control of breakthrough haemolysis with increased frequency pegcetacoplan dosing in treated paroxysmal nocturnal haemoglobinuria. (Pubmed Central) - Aug 21, 2023
Whilst almost all patients treated with terminal complement inhibitors develop extravascular haemolysis, only a small proportion of these results in symptomatic anaemia limiting their activities and requiring red cell transfusion. This case highlights the potential role for the C3 inhibitor, pegcetacoplan, in controlling both intravascular and extravascular haemolysis, and is the first case to report on the use of additional doses of pegcetacoplan to control breakthrough haemolysis.
- || cemdisiran (ALN-CC5) / Alnylam, Regeneron
P1/2 data, Journal: Results of a phase 1/2 study of cemdisiran in healthy subjects and patients with paroxysmal nocturnal hemoglobinuria. (Pubmed Central) - Aug 21, 2023
Cemdisiran and eculizumab combination therapy reduced the dose of eculizumab required to provide adequate control of intravascular hemolysis. These results demonstrate a potential benefit of cemdisiran coadministration in patients who are inadequate responders to eculizumab alone.
- | Soliris (eculizumab) / AstraZeneca
Journal, Gene therapy: Adopting Model-Informed Precision-Dosing for Eculizumab in Transplant Associated-Thrombotic Microangiopathy to Gene Therapies. (Pubmed Central) - Aug 21, 2023
These results demonstrate a potential benefit of cemdisiran coadministration in patients who are inadequate responders to eculizumab alone. No abstract available