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The Thalassemia Channel on VJHemOnc is an independent medical education platform, supported with funding from Agios (Gold). Supporters have no influence on the production of content. The levels of sponsorship listed are reflective of the amount of funding given.
ASH 2024 | The potential of mitapivat for treating thalassemia
Maria Domenica Cappellini, MD, University of Milan, Milan, Italy, comments on the value of mitapivat, a pyruvate kinase (PK) activator, for treating thalassemia. Prof. Cappellini notes that the agent’s oral formulation and favorable safety profile make it a promising treatment option, and she hopes it will be approved soon. This interview took place at the 66th ASH Annual Meeting and Exposition, held in San Diego, CA.
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Transcript (AI-generated)
Well, mitapivat that now has been already used since 2014 in a quite large cohort of patients, starting from PK deficiency patients and then thalassemia, either non-transfusion dependent and transfusion dependent. The results were all positive and what is the advantage? It is an oral molecule and that’s probably is impacting on the quality of life of the patients...
Well, mitapivat that now has been already used since 2014 in a quite large cohort of patients, starting from PK deficiency patients and then thalassemia, either non-transfusion dependent and transfusion dependent. The results were all positive and what is the advantage? It is an oral molecule and that’s probably is impacting on the quality of life of the patients. It’s quite safe because based on quite a good body of data we may say that patients tolerate quite well the treatment without serious adverse events. So whenever the drug will be approved, and hopefully after the results of the Phase III that I hope they will open the approval in different countries, I think that we should really be able to identify the patients who are good for being treated with mitapivat compared to other possibilities that are now in the scenario of novel treatment for thalassemia.
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Disclosures
Membership on an entity’s Board of Directors or advisory committees: Novo Nordisk, Vertex Pharmaceuticals, Pfizer, Silence, Sanofi-Genzyme, Pharmacosmos, Bristol Myers Squibb (Celgene), Vifor, Agios Pharmaceuticals, Inc.
