Mitapivat shows haemoglobin response in sickle cell disease trial
In the RISE UP phase 3 trial, 56 (41%) of 138 patients on mitapivat achieved a haemoglobin response versus 2 (3%) of 69 on placebo, an adjusted difference of 38% (95% CI 29-47). The annualised rate of pain crises was 2·6 for mitapivat and 3·1 for placebo (p=0·1213).
Primary source
PubMed record of a Phase III journal article, October 23, 2026
Efficacy and safety of mitapivat in patients with sickle cell disease (RISE UP): a phase 3, double-blind, global, randomised, placebo-controlled trial.
Read the source: Efficacy and safety of mitapivat in patients with sickle cell disease (RISE UP): a phase 3, double-blind, global, randomised, placebo-controlled trial. pubmed.ncbi.nlm.nih.gov
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