Vertex Pharmaceuticals has developed inaxaplin (VX-147) to treat APOL1-mediated focal segmental glomerulosclerosis, or FSGS.
The European Medicines Agency (EMA) also designated inaxaplin as a Priority Medicine (PRIME) for APOL1-mediated chronic kidney disease (AMKD).
Inaxaplin is the first experimental therapy designed to treat the underlying cause of AMKD.
APOL1-mediated FSGS is a chronic kidney disease caused by APOL1 gene mutations. People who have two mutations in the gene are more likely to develop kidney diseases, which cause podocyte injury.
Proteinuria and rapidly progressive kidney disease result from this injury, which disrupts filtration. Dialysis, kidney transplantation, or death can all result from progressive kidney disease.
The FDA designation was based on Vertex's Phase II clinical trial results.
Inaxaplin is an experimental drug that aims to inhibit the function of the APOL1 protein, which is responsible for kidney cell injury, cell death, and glomerular damage in people with APOL1-mediated kidney diseases.
The drug is the first genetically targeted approach to treating kidney disease and the first therapy designed to target the underlying cause of APOL1-mediated kidney diseases.
The EMA has designated Inaxaplin as the second nephrology product to receive PRIME status.