Voydeya (danicopan)
Research Synopsis
- - Danicopan (brand name Voydeya) is an oral complement factor D inhibitor primarily targeting paroxysmal nocturnal hemoglobinuria (PNH) and is now approved for use in Japan and recommended for authorization in Europe.
- - Recent research indicates that danicopan effectively enhances treatment outcomes when added to existing therapies like eculizumab for patients experiencing significant extravascular hemolysis.
- - In phase 2 trials, danicopan demonstrated a meaningful increase in hemoglobin levels and a reduction in blood transfusion requirements among PNH patients already treated with other medications.
- - Studies have shown that danicopan is generally well-tolerated by patients, although some adverse effects did lead to discontinuation of treatment in certain cases.
- - There is promising preclinical research supporting the potential of danicopan for other conditions involving excessive complement activation, such as atypical hemolytic uremic syndrome and membranoproliferative glomerulonephritis.
- - Despite its benefits, findings from clinical trials on C3 glomerulopathy showed that danicopan's effectiveness might be inconsistent, suggesting that further enhancements or alternative strategies might be necessary for optimal outcomes.
- - Ongoing trials, including the ALPHA study, are evaluating the safety and efficacy of danicopan in conjunction with C5 inhibitors, further underlining its role in managing PNH.
- - Research also highlights the emerging role of complement factor D inhibitors in oncology, particularly against early-onset colorectal cancer, indicating a broader therapeutic potential beyond hematology.
- - Overall, danicopan is positioned as an important addition to complement-based therapies, aiming to improve the quality of life for patients with specific hematologic conditions despite ongoing challenges in optimizing treatment responses.
Voydeya (danicopan)
Rise of the planet of rare anemias: An update on emerging treatment strategies.
London, UK
2 hours ago
1 Received
- There are new treatments being developed for rare types of anemia and other blood disorders, helping patients need fewer blood transfusions and feel better.
- Some treatments, like gene therapy and new medications, are showing promise, but scientists still need to make sure they are safe for patients in the long run.
- New drugs are being created to target specific problems in different types of anemia, which could lead to better care and help doctors find the right treatment for each patient.
$100 - $150
Hourly Rate
[Novel anti-complement therapeutics for hemolytic anemia].
London, UK
2 hours ago
1 Received
- Eculizumab was the first anti-complement drug approved in 2007 to help treat a blood disorder called PNH, and now it can help with other diseases too.
- An improved version, called ravulizumab, allows patients to get treatment every 2 to 8 weeks instead of more often, making life easier.
- New treatments like pegcetacoplan, iptacopan, danicopan, and sutimlimab are being developed or approved to help with different types of blood issues, focusing on hemolytic anemia.
$100 - $150
Hourly Rate
Discovery and Development of the Oral Complement Factor D Inhibitor Danicopan (ACH-4471).
London, UK
2 hours ago
1 Received
- - Complement is crucial for the innate immune system's defense against infections, but improper activation can lead to chronic diseases.
- - Complement Factor D (CFD) is key for activating the Alternative Pathway of complement, which amplifies responses but isn't necessary for other pathways, making it a target for selective inhibition.
- - Recent advancements in CFD inhibitors, especially reversible small molecules like danicopan, show promise for modulating complement activity without increasing infection risk.
$100 - $150
Hourly Rate
Factor D Inhibition Blocks Complement Activation Induced by Mutant Factor B Associated With Atypical Hemolytic Uremic Syndrome and Membranoproliferative Glomerulonephritis.
London, UK
2 hours ago
1 Received
- Complement factor B (FB) mutant variants lead to excessive complement activation associated with kidney diseases like aHUS, C3 glomerulopathy, and MPGN, with current treatments mainly focused on aHUS.
- This study investigates three FB missense mutations (D371G, E601K, I242L) found in aHUS and MPGN patients, revealing that certain mutations can cause hemolysis of sheep red blood cells.
- The FD inhibitor danicopan successfully reduced complement overactivation and hemolysis by blocking the cleavage of FB in patient serum and preventing release of harmful components from glomerular endothelial cells, suggesting it could be a potential treatment strategy for these kidney diseases.
$100 - $150
Hourly Rate
Phase 2 study of danicopan in patients with paroxysmal nocturnal hemoglobinuria with an inadequate response to eculizumab.
London, UK
2 hours ago
1 Received
- Paroxysmal nocturnal hemoglobinuria (PNH) causes uncontrolled complement activation leading to hemolysis, but treatment with C5 inhibitors may not fully prevent blood transfusion needs.
- A phase 2 trial involving 12 eculizumab-treated patients tested danicopan, an oral drug targeting an alternative complement pathway, showing a significant increase in hemoglobin levels and a reduction in transfusions over 24 weeks.
- Danicopan was well-tolerated with manageable side effects and demonstrated improved fatigue scores, indicating its potential as an effective treatment option for PNH patients.
$100 - $150
Hourly Rate
Danicopan: an oral complement factor D inhibitor for paroxysmal nocturnal hemoglobinuria.
London, UK
2 hours ago
1 Received
- Paroxysmal nocturnal hemoglobinuria (PNH) is a blood disorder caused by a lack of certain complement regulators, leading to serious hemolysis (destruction of red blood cells).
- Danicopan, an innovative oral medication, targets a specific factor in the complement system to help reduce hemolysis in PNH patients.
- In a phase 2 trial with 10 patients, danicopan was shown to significantly lower lactate dehydrogenase levels (an indicator of hemolysis) and was well-tolerated, although two patients discontinued the study for various reasons.
$100 - $150
Hourly Rate
Complement Factor D Is a Novel Biomarker and Putative Therapeutic Target in Cutaneous Squamous Cell Carcinoma.
London, UK
2 hours ago
1 Received
- Cutaneous squamous cell carcinoma (cSCC) is the most common type of skin cancer that can spread to other parts of the body, and it shows a link between complement components and cancer growth.* -
- Research found that factor D (FD), an enzyme in the alternative complement pathway, is significantly more active and produced in cSCC cells compared to normal skin cells, indicating its role in cSCC development.* -
- The study highlights FD as a potential new biomarker and target for therapy, showing that blocking FD can reduce cSCC cell growth by affecting key signaling pathways.*
$100 - $150
Hourly Rate
Complement System as a New Target for Hematopoietic Stem Cell Transplantation-Related Thrombotic Microangiopathy.
London, UK
2 hours ago
1 Received
- Thrombotic microangiopathy (TMA) can occur after stem cell transplants, known as transplant-associated thrombotic microangiopathy (TA-TMA), but its causes and effective diagnosis are still unclear, leading to a high risk of mortality.
- Key symptoms of TA-TMA include low platelet levels, hemolysis, and organ damage, especially to the kidneys, which can also cause high blood pressure, but diagnosing it is complicated by other potential health issues.
- Recent research indicates that the complement system plays a role in TA-TMA, suggesting that complement inhibition therapy might help some patients, particularly if they show clear signs of complement activation; two medications, eculizumab and narsoplimab, have
$100 - $150
Hourly Rate
Danicopan, an Oral Complement Factor D Inhibitor, Exhibits High and Sustained Exposure in Ocular Tissues in Preclinical Studies.
London, UK
2 hours ago
1 Received
- Danicopan is a new medication being tested for treating geographic atrophy in age-related macular degeneration by targeting a specific enzyme involved in the alternative pathway of the immune response.
- Research showed that after oral dosing, danicopan was widely distributed in rat tissues and had a prolonged presence in the pigmented rat eye, while it was less measurable in other tissues after a day.
- In rabbit studies, danicopan was found to reach higher concentrations in the eye tissues, especially in those with melanin, suggesting better and longer-lasting treatment effects in pigmented animals compared to albino ones.
$100 - $150
Hourly Rate
Clinical Outcomes of Patients with C3G or IC-MPGN Treated with the Factor D Inhibitor Danicopan: Final Results from Two Phase 2 Studies.
London, UK
2 hours ago
1 Received
- * Two phase 2 clinical studies were conducted on the orally active factor D inhibitor danicopan, aiming to assess its impact on C3G and related conditions.
- * Although danicopan was found to be safe, it failed to provide consistent treatment benefits or sufficiently inhibit the alternative pathway, indicating that stronger and sustained action is needed for effective patient outcomes.
$100 - $150
Hourly Rate
Baseline Clinical Characteristics and Complement Biomarkers of Patients with C3 Glomerulopathy Enrolled in Two Phase 2 Studies Investigating the Factor D Inhibitor Danicopan.
London, UK
2 hours ago
1 Received
- C3 glomerulopathy (C3G) is a rare kidney disease caused by issues in the complement system, specifically the alternative pathway, and the study aimed to analyze biomarkers in patients participating in phase 2 trials of danicopan, a factor D inhibitor.
- Twenty-nine patients were evaluated, revealing systemic activation of the complement pathway with significant correlations between various complement biomarkers and kidney health metrics, such as eGFR and proteinuria.
- The findings highlight strong relationships between complement biomarkers and kidney function/histology, potentially enhancing the understanding and classification of C3G patients.
$100 - $150
Hourly Rate
Complement Inhibitors in the Management of Complement-Mediated Hemolytic Uremic Syndrome and Paroxysmal Nocturnal Hemoglobinuria.
London, UK
2 hours ago
1 Received
- CM-HUS and PNH are rare blood disorders linked to the malfunction of the complement system, traditionally treated with plasma exchange or supportive care, but emerging monoclonal antibodies offer better options.
- Eculizumab has been the main treatment for over ten years, but its administration challenges have led to the development of new therapies that enhance patient quality of life.
- Recent advancements include ravulizumab, which requires less frequent treatment, and other novel therapies currently in trials, showing promise for easier management of these conditions.
$100 - $150
Hourly Rate
Treatment of eculizumab refractory paroxysmal nocturnal hemoglobinuria: A systematic review about current treatment options and future direction.
London, UK
2 hours ago
1 Received
- Eculizumab is a primary treatment for paroxysmal nocturnal hemoglobinuria (PNH), but some patients may become resistant to it, leading to a condition known as eculizumab refractory PNH.
- The study systematically reviewed 70 relevant studies on treatments for eculizumab refractory PNH, ultimately identifying four multicenter clinical trials that focused on drugs like pegcetacoplan, danicopan, and iptacopan.
- The findings suggest that treatment plans should be tailored to individual patient needs, and further randomized controlled trials are necessary to improve treatment guidelines for eculizumab refractory PNH.
$100 - $150
Hourly Rate
Addition of danicopan to ravulizumab or eculizumab in patients with paroxysmal nocturnal haemoglobinuria and clinically significant extravascular haemolysis (ALPHA): a double-blind, randomised, phase 3 trial.
London, UK
2 hours ago
1 Received
- The study evaluates the safety and effectiveness of danicopan, an oral complement factor D inhibitor, as an add-on treatment for patients with paroxysmal nocturnal haemoglobinuria (PNH) experiencing extravascular haemolysis while on C5 inhibitors (ravulizumab or eculizumab).
- It is an ongoing, phase 3 trial called ALPHA, which randomly assigns eligible adult patients to receive danicopan or a placebo alongside their current PNH treatment for 12 weeks.
- The primary goal of the study is to measure changes in haemoglobin concentration from baseline to week 12, with interim analysis conducted once around 75% of participants completed the trial up to that point.
$100 - $150
Hourly Rate
Complement inhibition in paroxysmal nocturnal hemoglobinuria: From biology to therapy.
London, UK
2 hours ago
1 Received
- Complement inhibitors are key treatments for paroxysmal nocturnal hemoglobinuria (PNH), with eculizumab being the first drug that helped improve symptoms, though it requires lifelong intravenous infusions every two weeks.
- Ravulizumab, a newer drug, offers convenience by being administered every 8 weeks and helps improve hemolysis control, while other anti-C5 drugs and upstream inhibitors are under study and aim to address limitations of current treatments.
- Despite advancements, there remain challenges like drug adherence and managing breakthrough hemolysis (BTH) due to various physical stressors, highlighting the need for a more tailored treatment approach for PNH patients.
$100 - $150
Hourly Rate
Danicopan: First Approval.
London, UK
2 hours ago
1 Received
- * Recently approved in Japan, it is specifically indicated for adults with paroxysmal nocturnal hemoglobinuria (PNH) when used alongside a complement component 5 (C5) inhibitor.
- * The European Medicines Agency also recommended marketing authorization for danicopan, highlighting its role for PNH patients who still suffer from anemia despite C5 inhibitor treatments; this article outlines its development journey leading to this approval.
$100 - $150
Hourly Rate
Complement inhibitors in pediatric kidney diseases: new therapeutic opportunities.
London, UK
2 hours ago
1 Received
- The complement system is essential in the development of various kidney diseases, with significant activation evident through the consumption of complement proteins in serum and deposition in kidney tissues.
- The introduction of eculizumab has highlighted the potential of complement inhibitors to positively impact the progression of certain kidney diseases, leading to a surge in research and available treatments.
- This review discusses several key complement inhibitors, including eculizumab and others that have shown promise in clinical studies, demonstrating their effectiveness in reducing protein levels and stabilizing kidney function in a range of complement-related kidney conditions.
$100 - $150
Hourly Rate
Exploring treatment strategies for paroxysmal nocturnal hemoglobinuria: an overview of registered clinical trials.
London, UK
2 hours ago
1 Received
- * New treatments like terminal complement inhibitors have improved patient outcomes by blocking hemolysis and reducing the need for blood transfusions, but issues like extravascular hemolysis still persist.
- * Proximal complement inhibitors have emerged as a promising solution to prevent both intra- and extravascular hemolysis, with multiple FDA-approved drugs and ongoing clinical trials aimed at optimizing PNH treatment strategies.
$100 - $150
Hourly Rate
Iptacopan and danicopan for paroxysmal nocturnal hemoglobinuria.
London, UK
2 hours ago
1 Received
$100 - $150
Hourly Rate
Combinatorial Inhibition of Complement Factor D and BCL2 for Early-Onset Colorectal Cancer.
London, UK
2 hours ago
1 Received
- The study investigates differences in gene expression between early-onset and late-onset colorectal cancer, focusing on immune genes like complement factor D.
- Researchers analyzed tumor samples from 121 patients using advanced techniques to validate these gene expressions and tested drugs that target these genes in mouse models.
- Findings showed that genes like complement factor D and BCL2 are more active in early-onset cases, and using drugs like danicopan and venetoclax in mice significantly reduced tumor size and weight.
$100 - $150
Hourly Rate