IPIG 2025 | A case report on the use of pegcetacoplan in pregnancy for PNH: efficacy, safety, & pharmacokinetics

I was very excited to present this morning one of the first case reports of pegcetacoplan use in pregnancy. So to our knowledge there was one other case reported in the literature and so we were excited to add to that with two cases now reported in the literature to our knowledge, but what’s also unique is that our case adds data on pharmacokinetics of the drug in pregnancy including maternal plasma levels, cord blood levels as well as breast milk levels. So this data was not previously available in humans and so we’re excited to be able to add that. 

So the case itself is a young woman with a diagnosis of classical PNH who unfortunately didn’t have an optimal response to frontline treatment with eculizumab so was transitioned to pegcetacoplan as another option, had a remarkable response and was feeling better, and ultimately ended up conceiving on this drug. So we ended up switching her back to eculizumab as this is a drug with more established safety and efficacy in pregnancy but owing to the fact that she still had clinically significant extravascular hemolysis throughout the pregnancy the decision was made, and this really required a multidisciplinary care team involving experts in maternal fetal medicine and clinical pharmacology, we made the decision at 28 weeks to switch her to pegcetacoplan, to switch her back. This resulted in transfusion independence and she ended up having improvement in symptoms as well. 

We, in discussion with the team, planned for a cesarean section at 37 weeks and this was because of certain maternal risk factors for prolonged labor. She delivered a healthy infant with no constitutional or developmental concerns to date, and had no PNH complications throughout pregnancy and delivery. 

In terms of the data that we were able to add in this case, so we measured the drug levels like I was saying and we found that there was no detectable pegcetacoplan levels in the cord blood, so suggesting undetectable or no transfer, and in the breast milk as well it was below the lower limit of quantification for the test. So this was exciting data to be able to present today. Obviously this is very preliminary, it’s only now the second case report and so really what it shows though is that we need more evidence in this space to be able to expand our therapeutic options. 

So to your question about unmet needs for patients with PNH in pregnancy, this is really one of them. We know that a significant portion of patients may not respond to frontline C5 inhibition, may require other therapeutic options. PNH is a disease that affects people across the age spectrum including young women or women of childbearing age who would be interested in conceiving and so it’s great to be able to have a range of therapeutic options available. So I would really applaud IPIG and the group here in you know having PNH registries and being able to collect more data on this, and I hope this is something that we’ll have in the near future.

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