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EHA 2025 | The value of pyruvate kinase activators in the treatment landscape of SCD
Caterina Minniti, MD, Albert Einstein College of Medicine, New York City, NY, comments on the value of pyruvate kinase activators in the treatment landscape of sickle cell disease (SCD). Dr Minniti highlights the dual benefits of these agents as they affect both the hemolytic and vasoocclusive pathways, with promising early data showing a decrease in pain episodes. This dual effect, combined with a favorable safety profile, has generated excitement in the SCD community, Dr Minniti notes. This interview took place at the 30th Congress of the European Hematology Association (EHA) in Milan, Italy.
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Transcript
Yes, we are very excited about the pyruvate kinase activators and there are more than one company that is developing them. And the reason we’re so excited is because it seems to hit both major pathophysiological pathways that lead to sickle cell complications. The hemolytic pathways by increasing hemoglobin and decreasing hemolysis, and the vasoocclusive pathway, as it seemed that early data showed a decrease in pain episodes...
Yes, we are very excited about the pyruvate kinase activators and there are more than one company that is developing them. And the reason we’re so excited is because it seems to hit both major pathophysiological pathways that lead to sickle cell complications. The hemolytic pathways by increasing hemoglobin and decreasing hemolysis, and the vasoocclusive pathway, as it seemed that early data showed a decrease in pain episodes. And it’s this dual effect that makes us in the community very excited, linked with what seems to be a very favorable safety profile, which of course safety is always at the heart of everything that we do. And so having a drug that seems at least pretty safe to date and seems to be affecting both hemolysis and vasoocclusion is certainly something we look forward to.
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Disclosures
Consultancy: Pfizer, Novo Nordisk, Fulcrum.
