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 defined as a 50% reduction in the transfusion of red blood cell units in any 12-week period during the 48-week course of the study.
The placebo-adjusted reduction in transfusion burden shown by mitapivat is lower than what’s been seen with Reblozyl, a competing drug for transfusion-dependent beta-thalassemia marketed by Bristol Myers Squibb. The two drugs have not been compared directly against each other and Bristol’s study used a different definition of transfusion response.
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