Novartis: iptacopan provides clinically meaningful results in hemoglobinuria

 

• Phase III APPOINT-PNH study of investigational oral monotherapy iptacopan met its primary endpoint; second positive Phase III topline readout for iptacopan in paroxysmal nocturnal hemoglobinuria (PNH)1
  
  

• Topline APPOINT-PNH data were consistent with APPLY-PNH readout and showed a significant proportion of complement-inhibitor-naïve patients treated with iptacopan achieved clinically meaningful increases in hemoglobin levels vs. baseline without the need for blood transfusions1
  
  

• PNH has a significant unmet need not addressed by anti-C5 therapies; despite treatment with anti-C5s, a large proportion of people with PNH remain anemic, fatigued and dependent on blood transfusions2-5
  
  

• APPOINT-PNH data to be presented at upcoming medical meeting and included in iptacopan PNH global regulatory 2023 submissions

https://finance.yahoo.com/news/novartis-investigational-iptacopan-provides-clinically-061500164.html