For iron deficiency anaemia, tolerability has improved with newer oral iron formulations (ferric maltol, sucrosomial iron, ferric citrate), and the recognition that alternate-day dosing enhances absorption while reducing side effects; modern intravenous iron preparations (ferric carboxymaltose, ferric derisomaltose) allow rapid, complete repletion for patients who are intolerant, malabsorptive, or need fast correction.
The bigger news is in thalassaemia, where two mechanisms now reduce transfusion burden. Luspatercept, a TGF-β ligand trap, is approved for beta-thalassaemia and reduces transfusion requirements (the BELIEVE trial). And the oral pyruvate kinase activator mitapivat — which improves red-cell energy metabolism and survival — was FDA-approved in December 2025 (marketed as AQVESME for this indication) for anaemia in adults with alpha- or beta-thalassaemia, whether or not they are transfusion-dependent, on the strength of the ENERGIZE and ENERGIZE-T trials; it carries a REMS for liver injury. A second PK activator, etavopivat, is in late-stage trials for thalassaemia and sickle cell disease.
Further reading: luspatercept BELIEVE in beta-thalassaemia (Cappellini et al., NEJM 2020); mitapivat ENERGIZE/ENERGIZE-T and 2025 approval; iron formulations review (Bazeley & Wish, AJKD 2022).