inaxaplin

Inaxaplin (formerly known as VX-147) is an oral, small molecule inhibitor of apolipoprotein L1 (APOL1) channel function. It is being developed as a precision medicine for APOL1-mediated kidney disease (AMKD), a genetic condition characterized by proteinuric nephropathies and accelerated progression to end-stage kidney disease. Inaxaplin binds directly to the APOL1 protein and blocks its channel activity, thereby reducing cationic flux associated with pathogenic APOL1 variants. This targeted inhibition reduces glomerular damage, lowers proteinuria, and may slow the progression of renal dysfunction in individuals with two risk variants of the APOL1 gene. Clinical studies have shown that inaxaplin significantly reduces proteinuria in patients with AMKD and has demonstrated a favorable safety profile through phase 2a trials. The drug is currently being evaluated in a global phase 2/3 pivotal trial for both adults and adolescents with AMKD[1][2][3][4][5][6][7][8].

Brand names

Inaxaplin

Other names

inaxaplin