Emerging treatments
Mitavipat
Mitapivat, an oral small molecule pyruvate kinase activator, has been granted orphan drug designation by the US Food and Drug Administration (FDA) for the treatment of thalassaemia. One phase 3, double-blind, randomised controlled trial, comparing mitapivat therapy with placebo in adults with non-transfusion-dependent alpha-thalassaemia (n=62) or beta-thalassaemia (n=132), reported that patients in the mitapivat group had a significantly higher haemoglobin response rate compared with those in the placebo group (55 of 130 [42%] patients vs. 1 of 64 [2%] patients, respectively).[108] 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%).[108] 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 non-transfusion-dependent thalassaemia, including alpha-thalassaemia.[109] The FDA has granted orphan drug designation to etavopivat for the treatment of thalassaemia.
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