FDA Grants Breakthrough Therapy Designation To Vertex Kidney Disease Treatment

The U.S. Food and Drug Administration (FDA) granted Vertex Pharmaceuticals Breakthrough Therapy Designation for inaxaplin, its kidney disease treatment in development. Inaxaplin is an investigational drug to treat APOL1-mediated focal segmental glomerulosclerosis, an underlying cause of APOL1-mediated chronic kidney disease (AMKD). In addition to gaining the Breakthrough Therapy Designation from the FDA, the European Medicines Agency (EMA) has granted inaxaplin Priority Medicines (PRIME) designation.

The FDA and EMEA designations enable Vertex to continue testing inaxaplin in the Inaxaplin (VX-147) Pivotal Program, a randomized, double-blind, placebo-controlled Phase 2/3 adaptive study. The program will first . . .