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Year Number of Results
2006 1
2020 1
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Page 1
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.
Lee JW, Griffin M, Kim JS, Lee Lee LW, Piatek C, Nishimura JI, Carrillo Infante C, Jain D, Liu P, Filippov G, Sicre de Fontbrune F, Risitano A, Kulasekararaj AG; ALXN2040-PNH-301 Investigators. Lee JW, et al. Lancet Haematol. 2023 Dec;10(12):e955-e965. doi: 10.1016/S2352-3026(23)00315-0. Lancet Haematol. 2023. PMID: 38030318 Clinical Trial.
At data cutoff (June 28, 2022), 73 individuals were randomly assigned, received treatment, and were analysed for safety (danicopan, n=49; placebo, n=24). The protocol-prespecified interim efficacy analysis set included the first 63 participants (danicopan, n=42; pla …
At data cutoff (June 28, 2022), 73 individuals were randomly assigned, received treatment, and were analysed for safety (danicopan, n …
Breakthrough hemolysis in paroxysmal nocturnal hemoglobinuria throughout clinical trials: from definition to clinical practice.
Fattizzo B, Versino F, Barcellini W. Fattizzo B, et al. Blood. 2025 Jul 24;146(4):411-421. doi: 10.1182/blood.2024027574. Blood. 2025. PMID: 40233322 Review.
In this review article, we analyzed the definition, frequency, and severity of BTH events across phase 3 clinical trials with terminal (anti-C5 ravulizumab and crovalimab) and complement inhibitors upstream C5 (anti-C3 pegcetacoplan, alternative-pathway inhibitors iptacopan, and …
In this review article, we analyzed the definition, frequency, and severity of BTH events across phase 3 clinical trials with terminal (anti …
Paroxysmal Nocturnal Hemoglobinuria, Pathophysiology, Diagnostics, and Treatment.
Panse JP, Höchsmann B, Schubert J. Panse JP, et al. Transfus Med Hemother. 2024 Aug 21;51(5):310-320. doi: 10.1159/000540474. eCollection 2024 Oct. Transfus Med Hemother. 2024. PMID: 39371251 Free PMC article. Review.
New proximal complement inhibitors (CI) mainly targeting complement component C3 or factors of the amplification pathway such as pegcetacoplan, danicopan, and iptacopan became available and are meanwhile approved for marketing. Additional complement-inhibiting strategies a …
New proximal complement inhibitors (CI) mainly targeting complement component C3 or factors of the amplification pathway such as pegcetacopl …
Long-term efficacy and safety of danicopan as add-on therapy to ravulizumab or eculizumab in PNH with significant EVH.
Kulasekararaj A, Griffin M, Piatek C, Shammo J, Nishimura JI, Patriquin C, Schrezenmeier H, Barcellini W, Panse J, Gaya A, Patel Y, Liu P, Filippov G, Sicre de Fontbrune F, Risitano A, Lee JW. Kulasekararaj A, et al. Blood. 2025 Feb 20;145(8):811-822. doi: 10.1182/blood.2024026299. Blood. 2025. PMID: 39700502 Free PMC article. Clinical Trial.
In the phase 3 ALPHA trial, participants received oral factor D inhibitor danicopan (150 mg 3 times daily) or placebo plus ravulizumab or eculizumab during the 12-week, double-blind treatment period 1 (TP1); those receiving placebo switched to danicopan during the s …
In the phase 3 ALPHA trial, participants received oral factor D inhibitor danicopan (150 mg 3 times daily) or placebo plus ravulizuma …
Complement inhibition in paroxysmal nocturnal hemoglobinuria: From biology to therapy.
Versino F, Fattizzo B. Versino F, et al. Int J Lab Hematol. 2024 May;46 Suppl 1:43-54. doi: 10.1111/ijlh.14281. Epub 2024 Apr 15. Int J Lab Hematol. 2024. PMID: 38622956 Free article. Review.
Upstream inhibition of complement cascade was also explored with the anti-C3 pegcetacoplan, and with the alternative pathway inhibitors iptacopan (anti-factor B) and danicopan (anti-factor D). These drugs efficiently target EVH and are able to improve anemia and transfusio …
Upstream inhibition of complement cascade was also explored with the anti-C3 pegcetacoplan, and with the alternative pathway inhibitors ipta …
Danicopan: First Approval.
Kang C. Kang C. Drugs. 2024 May;84(5):613-618. doi: 10.1007/s40265-024-02023-6. Epub 2024 Mar 26. Drugs. 2024. PMID: 38528310 Review.
Danicopan recently received approval in Japan for the treatment of adults with paroxysmal nocturnal haemoglobinuria (PNH) when used in addition to a complement component 5 (C5) inhibitor. ...This article summarizes the milestones in the development of danicopan lead …
Danicopan recently received approval in Japan for the treatment of adults with paroxysmal nocturnal haemoglobinuria (PNH) when used i …
Danicopan.
[No authors listed] [No authors listed] Am J Health Syst Pharm. 2024 Aug 12;81(16):661-664. doi: 10.1093/ajhp/zxae142. Am J Health Syst Pharm. 2024. PMID: 38916137 No abstract available.
Danicopan.
[No authors listed] [No authors listed] 2025 Jan 15. Drugs and Lactation Database (LactMed®) [Internet]. Bethesda (MD): National Institute of Child Health and Human Development; 2006–. 2025 Jan 15. Drugs and Lactation Database (LactMed®) [Internet]. Bethesda (MD): National Institute of Child Health and Human Development; 2006–. PMID: 39836823 Free Books & Documents. Review.
No information is available on the use of danicopan during breastfeeding. Because danicopan is more than 91% bound to plasma proteins, the amount in milk is likely to be low. ...
No information is available on the use of danicopan during breastfeeding. Because danicopan is more than 91% bound to plasma p …
Complement factor D is a drug target for metabolic-associated fatty liver disease.
Guo R, Li Z, Wu J, Zhang Z, Shu J, Zhou X, Zhou W, Chen Y, Guan D, Wang Q, Shi Q, Yang H, Li G. Guo R, et al. Mol Immunol. 2025 Dec;188:192-200. doi: 10.1016/j.molimm.2025.11.006. Epub 2025 Nov 26. Mol Immunol. 2025. PMID: 41308544
Here, we assessed the role of CFD in MAFLD and danicopan therapy using HFD mice, patient sera, and hepatocytes via CRISPR knockout and pharmacological interventions. ...Mechanistically, danicopan suppressed NF-kappaB signaling, inhibiting lipid-related genes (CD36/F …
Here, we assessed the role of CFD in MAFLD and danicopan therapy using HFD mice, patient sera, and hepatocytes via CRISPR knockout an …
Advancements in complement inhibition for PNH and primary complement-mediated thrombotic microangiopathy.
Padilla Kelley T, King H, Malhotra A, DeLoughery TG, Martens K, Shatzel JJ. Padilla Kelley T, et al. Blood Adv. 2025 Aug 12;9(15):3937-3945. doi: 10.1182/bloodadvances.2024015777. Blood Adv. 2025. PMID: 40354320 Free PMC article. Review.
These agents currently include pegcetacoplan, a C3 inhibitor; iptacopan, an oral factor B inhibitor; danicopan, an oral factor D inhibitor; and crovalimab, an anti-C5 monoclonal antibody. This review highlights recent advances in complement-targeted therapies for PNH, incl …
These agents currently include pegcetacoplan, a C3 inhibitor; iptacopan, an oral factor B inhibitor; danicopan, an oral factor D inhi …
71 results