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IPIG 2025 | Safety & efficacy of iptacopan in PNH and identifying the patients that may benefit from this agent
Yasutaka Ueda, MD, PhD, Osaka University, Suita, Japan, discusses the safety and efficacy of iptacopan in paroxysmal nocturnal hemoglobinuria (PNH), highlighting its potential to control both intravascular and extravascular hemolysis. Dr Ueda notes that iptacopan is an attractive option for patients who are anemic and those who prefer an oral route of administration. This interview took place at the 2nd International PNH Interest Group (IPIG) Conference in Paris, France.
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Transcript
Iptacopan from clinical trials shows quite an efficacy in both controlling intravascular hemolysis and extravascular hemolysis. And another unique feature is that it is monotherapy of a proximal inhibitor. So this drug shows that in PNH, in the complement activation pathway, alternative pathway, is a major cause of PNH intravascular hemolysis. The safety has been shown in clinical trials, and it’s quite safe...
Iptacopan from clinical trials shows quite an efficacy in both controlling intravascular hemolysis and extravascular hemolysis. And another unique feature is that it is monotherapy of a proximal inhibitor. So this drug shows that in PNH, in the complement activation pathway, alternative pathway, is a major cause of PNH intravascular hemolysis. The safety has been shown in clinical trials, and it’s quite safe. Of course, the patients are vaccinated against HIV or meningitis, but there was no reported meningitis infection in the trial. So it’s a good choice for those PNH patients on C5 inhibitors who are still anemic because iptacopan shows superior improvement of hemoglobin and quality of life. So if the patients are still anemic, iptacopan could be a good choice for those patients. And of course it’s oral monotherapy. So those patients with sick veins or they want to avoid injections, it would be a good choice.
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