Vertex Pharmaceuticals Receives FDA Approval for Inaxaplin in APOL1-Mediated Kidney Disease

Vertex Pharmaceuticals today announced that the FDA has approved inaxaplin for the treatment of adults with APOL1-mediated kidney disease (AMKD) with proteinuria. This marks the first targeted therapy approved for AMKD, a progressive kidney disease caused by high-risk APOL1 variants that disproportionately affects people of African ancestry. In the Phase 3 AMPLITUDE trial, inaxaplin demonstrated a statistically significant reduction in proteinuria compared to placebo. Inaxaplin is a first-in-class oral APOL1 inhibitor that targets the underlying genetic cause of AMKD.