Vertex Pharmaceuticals (NASDAQ:VRTX) received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for its drug candidate inaxaplin (VX-147) for the treatment of patients with APOL1-mediated focal segmental glomerulosclerosis (FSGS).
The FDA grant was supported by the Phase 2 clinical study of inaxaplin in patients with APOL1-mediated FSGS, a form of AMKD.
