iptacopan PNH
Selected indexed studies
- Iptacopan Efficacy and Safety to Treat Paroxysmal Nocturnal Hemoglobinuria (PNH): A Systematic Review and Meta-Analysis. (Cureus, 2024) [PMID:39323666]
- Oral iptacopan monotherapy in paroxysmal nocturnal haemoglobinuria: final 48-week results from the open-label, randomised, phase 3 APPLY-PNH trial in anti-C5-treated patients and the open-label, single-arm, phase 3 APPOINT-PNH trial in patients previously untreated with complement inhibitors. (Lancet Haematol, 2025) [PMID:40447351]
- Oral Iptacopan Monotherapy in Paroxysmal Nocturnal Hemoglobinuria. (N Engl J Med, 2024) [PMID:38477987]
_Worker-drafted node — pending editorial review._
Connections
iptacopan PNH is a side effect of
Sources
- Oral Iptacopan Monotherapy in Paroxysmal Nocturnal Hemoglobinuria. (2024) pubmed
- Iptacopan: First Approval. (2024) pubmed
- Iptacopan Efficacy and Safety to Treat Paroxysmal Nocturnal Hemoglobinuria (PNH): A Systematic Review and Meta-Analysis. (2024) pubmed
- Factor B inhibitor iptacopan for the treatment of paroxysmal nocturnal hemoglobinuria. (2024) pubmed
- Iptacopan monotherapy in patients with paroxysmal nocturnal hemoglobinuria: a 2-cohort open-label proof-of-concept study. (2022) pubmed
- Navigating the Complement Pathway to Optimize PNH Treatment with Pegcetacoplan and Other Currently Approved Complement Inhibitors. (2024) pubmed
- Oral iptacopan monotherapy in paroxysmal nocturnal haemoglobinuria: final 48-week results from the open-label, randomised, phase 3 APPLY-PNH trial in anti-C5-treated patients and the open-label, single-arm, phase 3 APPOINT-PNH trial in patients previously untreated with complement inhibitors. (2025) pubmed
- Patient-reported improvements in paroxysmal nocturnal hemoglobinuria treated with iptacopan from 2 phase 3 studies. (2025) pubmed
- PMID:40294188 (2025) pubmed
- Patient-Reported Meaningful Change in Symptoms and Impacts of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Three Phase III Clinical Trials of Iptacopan. (2025) pubmed