Researchers have published the results of a clinical trial that led the U.S. Food and Drug Administration to recently approve mitapivat for the treatment of adults with pyruvate kinase deficiency—a rare genetic condition that leads to the destruction of red blood cells, or hemolytic anemia. The primary results from the global, phase 3, randomized, placebo-controlled ACTIVATE trial, which was conducted by an international team including investigators at Massachusetts General Hospital (MGH), are published in the New England Journal of Medicine.
