Inaxaplin for the treatment of APOL1-associated kidney disease.

Chronic kidney disease (CKD) is highly prevalent World-Wide and it is an important cause of morbidity and mortality. In 2010, variants in apolipoprotein type 1 (APOL1) gene was identified as a major risk factor for higher prevalence of CKD in individuals of African ancestry. The mechanisms by which toxic gain of function APOL1 variants cause disease are the subjects of current investigations, and there is currently no effective targeted therapy for APOL1 associated kidney disease. Egbuna and others recently reported that an orally administered small molecule compound inaxaplin (VX-147) that binds APOL1 may be useful in the treatment of APOL1 associated kidney disease. This is a groundbreaking study, if confirmed in randomized control studies it may turn out to be one of the major advances in the therapy of proteinuric CKD this decade, and may also represent precision therapy for addressing health disparity in CKD. However, there is a need for a larger randomized control studies with stable eGFR and complete remission of proteinuria as end point, such studies should also be carried out in a more geographically diverse population.

Duke Scholars

Author Brandon Lane Pediatrics, Nephrology

Author Rasheed Adebayo Gbadegesin Pediatrics, Nephrology