BOSTON — Vertex Pharmaceuticals said the U.S. Food and Drug Administration has accepted its biologics license application seeking accelerated approval of povetacicept for adults with immunoglobulin A nephropathy.
The FDA assigned a Prescription Drug User Fee Act target action date of Nov. 30, 2026. If approved, povetacicept would become the first commercialized therapy in Vertex’s emerging nephrology franchise.
Povetacicept is an investigational engineered fusion protein and dual inhibitor of the BAFF and APRIL cytokines. Vertex said the therapy is designed to provide B cell control by inhibiting BAFF and APRIL activity, which can drive the pathogenesis of multiple autoimmune diseases.
“The Phase 3 RAINIER trial is the largest conducted in IgAN and achieved full enrollment faster than any contemporary IgAN trial, reflecting the significant unmet need in IgAN and our urgency to bring povetacicept to patients with this serious disease,” said Nia Tatsis, Ph.D., executive vice president and chief regulatory and quality officer at Vertex. “With today’s FDA acceptance of the BLA, we are one step closer to our goal of transforming the care of patients living with IgAN given povetacicept’s potential best-in-class clinical profile, including every 4-week dosing delivered in a low-volume autoinjector.”
The application is supported by data from a prespecified Week 36 interim analysis of the ongoing Phase 3 RAINIER trial, which Vertex said showed a statistically significant and clinically meaningful reduction in proteinuria, a key marker of kidney disease progression, compared with placebo.
The trial met its primary objective, with patients treated with povetacicept achieving a 52% reduction from baseline in urine protein-to-creatinine ratio at Week 36. Vertex said the result represented a statistically significant and clinically meaningful 49.8% reduction compared with placebo. The reduction in proteinuria was consistent across all prespecified subgroups.
The study also met secondary objectives. Patients treated with povetacicept showed a 77.4% reduction from baseline in serum galactose-deficient IgA1, compared with a 9.1% increase in the placebo group. Among patients with baseline hematuria, 85.1% of those receiving povetacicept achieved hematuria resolution, compared with 23.4% of patients receiving placebo.
Vertex said povetacicept was generally safe and well tolerated, with most adverse events classified as mild to moderate. The company said no serious adverse events were related to povetacicept. Anti-drug antibodies were observed, as expected, but Vertex said they had no effect on efficacy or the therapy’s risk profile.
If approved, Vertex said it plans to launch povetacicept as a low-volume subcutaneous autoinjector administered once every four weeks at home.
Povetacicept has received FDA Breakthrough Therapy Designation for IgA nephropathy. Vertex said the therapy is the only dual BAFF and APRIL inhibitor in pivotal trials for multiple kidney diseases, including the ongoing Phase 2/3 OLYMPUS trial in primary membranous nephropathy. The company is also studying povetacicept in generalized myasthenia gravis through the Phase 2 ETNA trial.
Povetacicept remains investigational and has not been approved by health authorities.
