Vertex gets FDA acceptance for IgA nephropathy drug application

Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) announced the U.S. Food and Drug Administration has accepted its Biologics License Application for povetacicept, an investigational treatment for adults with immunoglobulin A nephropathy.

The FDA assigned a Prescription Drug User Fee Act target action date of November 30, 2026. The application seeks accelerated approval for povetacicept, which would become Vertex's first commercialized therapy in its nephrology franchise if approved.

The submission is supported by data from a Week 36 interim analysis of the Phase 3 RAINIER trial. Patients treated with povetacicept achieved a 52.0% reduction from baseline in urine protein to creatinine ratio at Week 36, with a 49.8% reduction compared to placebo. The trial met its primary objective with statistical significance.

For secondary endpoints, patients demonstrated a 77.4% reduction from baseline in serum galactose deficient IgA1 compared to a 9.1% increase in the placebo group. Among patients with baseline hematuria, 85.1% achieved hematuria resolution in the treatment group compared to 23.4% in the placebo group.

Povetacicept was generally safe and well tolerated in the trial. The majority of adverse events were mild to moderate, with no serious adverse events related to the treatment. Anti-drug antibodies were observed but had no impact on efficacy or safety.

If approved, Vertex plans to launch povetacicept as a subcutaneous auto-injector administered once every four weeks for at-home use. The treatment is designed as a dual inhibitor of BAFF and APRIL cytokines.

IgA nephropathy is a progressive kidney disease affecting approximately 330,000 people in the United States and Europe. The RAINIER trial enrolled 605 adults with the condition and was the largest trial conducted in IgA nephropathy.

Povetacicept has received FDA Breakthrough Therapy Designation for IgA nephropathy treatment. The drug is also being studied in other conditions through ongoing trials.

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