Vertex Advances Kidney Disease Pipeline With FDA Breakthrough Therapy And Multiple Clinical Milestones

Vertex Pharmaceuticals has reported key advancements across its kidney disease pipeline, highlighting progress in immunoglobulin A nephropathy (IgAN), APOL1-mediated kidney disease (AMKD), and autosomal dominant polycystic kidney disease (ADPKD). The updates reflect the company’s commitment to delivering first-in-class and best-in-class therapies aimed at the root causes of serious renal conditions.

For IgAN, Vertex’s investigational therapy povetacicept (pove), a dual antagonist of BAFF and APRIL, has received Breakthrough Therapy Designation from the U.S. FDA. The drug is being evaluated in the global Phase 3 RAINIER trial, which includes a pre-planned interim analysis assessing reduction in proteinuria. Vertex aims to seek accelerated approval in the U.S. during the first half of 2026 if the interim data support its efficacy. 

“Our pursuit of breakthrough science and serial innovation has changed the treatment landscape for cystic fibrosis, acute pain, sickle cell disease and beta thalassemia by advancing transformative therapies that target the underlying cause of these diseases. We are excited to bring the same level of pioneering science and potentially transformative benefits to patients with serious kidney diseases. Today’s announcements on BTD for pove in IgAN, completion of enrollment in the IA cohort in our AMKD study, setting up the potential for accelerated approval of inaxaplin and initiation of the VX-407 POC study in ADPKD mark significant milestones toward bringing the next wave of promising medicines to patients who are waiting,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. 

In parallel, the company has completed enrollment of the interim analysis cohort in the AMPLITUDE trial for inaxaplin, its APOL1 inhibitor targeting AMKD, with results expected to support a potential accelerated approval pathway.

Additionally, Vertex has initiated the AGLOW Phase 2 proof-of-concept study for VX-407 in ADPKD patients with specific PKD1 variants. The small molecule corrector is designed to restore protein folding and function, potentially reducing kidney volume and delaying disease progression. With these milestones, Vertex strengthens its position in renal innovation, advancing therapies that could significantly reshape treatment options for underserved patient populations.