STAT June 3, 2024
Agios Pharmaceuticals said Monday that its drug called mitapivat reduced the need for blood transfusions in patients with a severe form of beta-thalassemia, an inherited blood disorder. The results achieved the primary goal of a placebo-controlled Phase 3 clinical trial, and if the drug is eventually cleared by regulators, could accelerate sales.
In the study, 30% of participants responded to treatment with mitapivat compared to 12% of participants offered a placebo. Response was...
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