FDA Greenlights Clinical Trial for ADPKD Drug

The US Food and Drug Administration approved a clinical trial for VX-407, an investigational drug from Vertex Pharmaceuticals. VX-407 is intended to treat autosomal dominant polycystic kidney disease, a hereditary form of kidney disease that can require dialysis and transplantation and may ultimately lead to death.

Vertex, which is known for its cystic fibrosis therapies, is planning a phase 1 trial for the small-molecule candidate in healthy volunteers. The company also has an oral experimental therapy in its pipeline. That drug, inaxaplin, targets APOL1-mediated kidney disease, a chronic kidney disease caused by mutations in the APOL1 gene.