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Iptacopan Demonstrates Clinical Efficacy in Cold Agglutinin Disease While Falling Short in Immune Thrombocytopenia: Results from a Phase 2 Basket Trial
Posted inHematology-Oncology Internal Medicine news

Iptacopan Demonstrates Clinical Efficacy in Cold Agglutinin Disease While Falling Short in Immune Thrombocytopenia: Results from a Phase 2 Basket Trial

Posted by By MedXY 01/19/2026
This Phase 2 basket trial highlights the divergent efficacy of the oral factor B inhibitor iptacopan, showing significant hemoglobin improvements in Cold Agglutinin Disease but failing to meet platelet response endpoints in Immune Thrombocytopenia.
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Oral Iptacopan Monotherapy in Paroxysmal Nocturnal Haemoglobinuria: 48-Week Outcomes from APPLY-PNH and APPOINT-PNH Phase 3 Trials
Posted inClinical Updates Internal Medicine news Pediatrics Specialties

Oral Iptacopan Monotherapy in Paroxysmal Nocturnal Haemoglobinuria: 48-Week Outcomes from APPLY-PNH and APPOINT-PNH Phase 3 Trials

Posted by By MedXY 08/03/2025
Oral iptacopan provides sustained haemolysis control and improved haemoglobin in PNH patients over 48 weeks, with a favorable safety profile, supporting its role as a new therapeutic option.
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