Press release
Vertex Announces Key Advancements Across Kidney Portfolio
- Food and Drug Administration granted Breakthrough Therapy Designation for povetacicept in IgA nephropathy; on track to file for accelerated approval in the
About this update from Vertex Pharmaceuticals Incorporated
[{"type":"text","content":"\n- Food and Drug Administration granted Breakthrough Therapy Designation for povetacicept in IgA nephropathy; on track to file for accelerated approval in the U.S. in H1 2026 if 36-week interim analysis data positive -\n\n\n- Enrollment completed for interim analysis cohort of AMPLITUDE global Phase 2/3 trial evaluating inaxaplin in APOL1-mediated kidney disease; potential to file for accelerated approval in the U.S. if 48-week interim analysis data positive -\n\n\n- Phase 2 proof-of-concept study initiated for VX-407 in patients with autosomal dominant polycystic kidney disease -\n\n\n BOSTON--(BUSINESS WIRE)--\nVertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced several important advancements across its programs in immunoglobulin A Nephropathy (IgAN), APOL1-mediated kidney disease (AMKD) and autosomal dominant polycystic kidney disease (ADPKD). These updates represent significant progress toward reaching the Company’s goal of bringing forward first-in-class or best-in-class therapies that target the underlying cause of these serious kidney diseases.\n\n\nPovetacicept in IgAN\n\n\nVertex announced today that the Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to povetacicept (pove) for the treatment of IgAN. Pove is an investigational recombinant fusion protein therapeutic and dual antagonist of the BAFF (B cell activating factor) and APRIL (a proliferation inducing ligand) cytokines with best-in-class potential in IgAN and other B cell-driven diseases. BTD is for a drug that treats a serious condition, and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement on one or more clinically significant endpoints over existing treatments.\n\n\nPove is currently being studied in RAINIER, a global Phase 3 clinical trial in patients with IgAN. The study is designed to have a pre-planned interim analysis (IA) evaluating urine protein to creatinine ratio (UPCR) for the pove arm versus placebo after a specified number of patients reach 36 weeks of treatment. The IA cohort has been fully enrolled, and Vertex remains on track to file for accelerated approval in the U.S. in H1 2026 if results are supportive.\n\n\nInaxaplin in AMKD\n\n\nVertex also announced today enrollment completion of the IA cohort of AMPLITUDE, a global Phase 2/3 clinical trial d...