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EHA 2025 | Exploring mitapivat in thalassemia & SCD: the potential upcoming approval of the agent
In this video, Maria Domenica Cappellini, MD, University of Milan, Milan, Italy, comments on clinical trials exploring mitapivat in sickle cell disease (SCD) and thalassemia, noting that the results to date have been encouraging. Prof. Cappellini expects mitapivat to be registered soon for patients with thalassemia, with the approval in SCD likely to come later. This interview took place at the 30th Congress of the European Hematology Association (EHA) in Milan, Italy.
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Transcript
Mitapivat is a pyruvate kinase inducer. It was approved already a few years ago for treating patients with PK deficiency and getting the results on PK patients, the idea was can we use this compound to improve also the energy of let’s say thalassemia or sickle cell red cells. The idea was a bit you know fancy, but after data coming from a Phase II trial in thalassemia and in sickle, the results were really very exciting...
Mitapivat is a pyruvate kinase inducer. It was approved already a few years ago for treating patients with PK deficiency and getting the results on PK patients, the idea was can we use this compound to improve also the energy of let’s say thalassemia or sickle cell red cells. The idea was a bit you know fancy, but after data coming from a Phase II trial in thalassemia and in sickle, the results were really very exciting. So now I’ve been presented the results of ENERGIZE and ENERGIZE-T, the two Phase three trials in thalassemia, transfusion-dependent ENERGIZE-T and in non-transfusion-dependent thalassemia. And the results are extremely promising. So we are expecting to have the compound registered soon because for transfusion dependent we saw a reduction of transfusion burden of equal or more than 50 percent in a large proportion of patients and in non-transfusion dependent we saw an increase of hemoglobin at least of one gram or even more. So very promising.
In sickle cell disease, the outcome should be the reduction of vaso-occlusive crisis and data have been presented and even in sickle the results are quite good. Probably the registration for this indication will come a bit later than thalassemia, but for thalassemia we are expecting to have the registration quite soon. I have to mention that there are similar compounds to mitapivat, such as etavopivat, which confirm the data either in thalassemia and in SCD.
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