Novartis iptacopan shows benefit in blood disorder paroxysmal nocturnal hemoglobinuria

-- First-in-class, oral, targeted factor B inhibitor iptacopan substantially 
      reduced both intra- and extravascular hemolysis when given as monotherapy 
      in a Phase II study of anti-C5 naïve paroxysmal nocturnal 
      hemoglobinuria (PNH) patients1 
 
   -- New results are promising for potential use of iptacopan as monotherapy 
      in PNH, a rare and life-threatening blood disorder2,3; results from a 
      previous Phase II study showed iptacopan substantially improved 
      hematological response as add-on to standard-of-care (eculizumab)4 
 
   -- The FDA has granted Breakthrough Therapy Designation to iptacopan for 
      PNH5; it also has received orphan drug designation for PNH from both the 
      FDA and EMA6 
 
   -- Iptacopan is also in development for several rare renal conditions with 
      complement system (part of the innate immune system) involvement, 
      targeting a key driver of these diseases7,8 
 
   -- Recently presented Phase II data showed iptacopan reduced proteinuria and 
      stabilized kidney function in IgA nephropathy (IgAN)9, and improved 
      estimated glomerular filtration rate (eGFR) slope and stabilized kidney  

function in C3 glomerulopathy (C3G)10

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