BSH 2023 | The changing treatment landscape of PNH: novel agents and combinations

So I think it’s really important to emphasize that we have now in countries where treatment is available, PNH used to be a life-threatening disease with a significant mortality. Treatment with eculizumab has now changed that to patients having a near normalized life expectancy when treated. So I think we are now in a position to address more unmet needs within PNH, and that’s certainly, we now have ravulizumab available in the UK, which is every eight week treatment IV instead of every two weeks, and pegcetacoplan which has just recently been approved for patients with extravascular hemolysis as well. There’s lots of clinical studies going on with proximal complement inhibitors. But to address extra vascular hemolysis where patients on C5 inhibitor, have got C3 optimization, and reduced red cell lifespan, and this includes all factor B and factor D inhibitors and other more early compounds. We’re also looking at alternative options at blocking at C5, with either a combination of pozelimab and cemdisiran, or crovalimab to name but a few. So these are self-injection subcutaneous options. So having gone from a single option for patients with PNH as an IV treatment option, we’re now moving towards a much more patient focused and delivery of treatment and a more personalized approach depending on the patient needs. But I would emphasize it’s really important to involve the patients within all of these discussions and certainly the PNH patient support groups around the world are very involved with this.