Source
Hematology Advisor
Topline data were announced from a phase 3 trial evaluating etavopivat in adults and pediatric patients with sickle cell disease (SCD).
Etavopivat is a once-daily, oral pyruvate kinase-R activator designed to decrease 2,3-diphosphoglycerate (2,3-DPG) levels and increase adenosine triphosphate (ATP) in SCD. This dual mechanism is expected to increase hemoglobin-oxygen affinity to prevent polymerization while also preserving red blood cell membrane integrity and deformability to prolong cell survival.
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