Agios Pharmaceuticals Inc. is preparing to present a mixed bag of phase III Rise Up data to the U.S. FDA in hopes of “full approval” for mitapivat in sickle cell disease (SCD), which would make it its third indication in rare hematology.
The pressure to replace animal testing with human-relevant assays that are more predictive of human-drug responses has now reached a tipping point, and there is a movement toward greater acceptance of these potentially more translatable tests.
