Emerging treatments
Mitavipat
Mitapivat, an oral small molecule pyruvate kinase activator, has been granted orphan drug designation by the Food and Drug Administration (FDA) for the treatment of thalassemia. One phase 3, double-blind, randomized controlled trial, comparing mitapivat therapy with placebo in adults with nontransfusion-dependent alpha-thalassemia (n=62) or beta-thalassemia (n=132), reported that patients in the mitapivat group had a significantly higher hemoglobin response rate compared with those in the placebo group (55 of 130 [42%] patients vs. 1 of 64 [2%] patients, respectively).[111] The most common adverse events in the mitapivat group were headache, initial insomnia, nausea, and upper respiratory tract infection. Treatment-related adverse events leading to discontinuation of mitapivat occurred in four patients (3%).[111] An open-label, 5-year extension study is ongoing.
Etavopivat
An investigational pyruvate kinase activator, etavopivat, is being studied in a phase 2 trial for transfusion-dependent and nontransfusion-dependent thalassemia, including alpha-thalassemia.[112] The FDA has granted orphan drug designation to etavopivat for the treatment of thalassemia.
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