Novartis Wins Full FDA Approval for Rare Kidney Disease Drug

The U.S. Food and Drug Administration has granted full approval to Novartis’ Fabhalta for adults with primary immunoglobulin A nephropathy, a rare kidney disease that can lead to kidney failure.

The decision converts an earlier accelerated approval into traditional approval after new data showed the drug can significantly slow the loss of kidney function over time. Regulators reviewed the application under priority review, reflecting the need for more effective treatments in this condition.

IgA nephropathy is a chronic autoimmune disease in which an abnormal immune response leads to the buildup of immunoglobulin A in the kidneys. This triggers inflammation that damages the filtering units of the kidney, gradually reducing their ability to remove waste from the blood. The disease often progresses silently, but over time it can lead to worsening kidney function, dialysis or the need for a transplant. It affects about 25 people per million each year worldwide, and up to half of patients with persistent signs of disease may progress to kidney failure within 10 to 20 years.

Fabhalta is an oral therapy designed to target the alternative complement pathway, part of the immune system that plays a central role in driving inflammation in IgAN. Specifically, it inhibits a protein called Factor B, helping to reduce ongoing immune-mediated damage in the kidneys. By addressing an underlying disease mechanism rather than just symptoms, the therapy aims to preserve kidney function and delay progression. Fabhalta is the first and only approved treatment in its class for IgAN, marking a shift toward more targeted approaches in rare kidney diseases.

The approval is based on results from a large phase 3 study showing that patients taking Fabhalta experienced a 48 percent slower decline in kidney function compared with those on placebo over two years. The drug also reduced levels of protein in the urine — a key marker of disease — within weeks, with effects sustained throughout treatment. Safety findings were consistent with earlier studies, with common side effects including abdominal pain, dizziness and nausea. Because the therapy can increase the risk of certain serious infections, patients must be vaccinated and enrolled in a safety monitoring program before starting treatment.

“Today’s approval reinforces Fabhalta’s role in preserving kidney function by significantly slowing disease progression, an outcome that matters deeply to patients at risk of long-term kidney damage,” said Victor Bultó, president, U.S., Novartis. “This milestone underscores the importance of continued innovation for people living with IgAN and our commitment to addressing the underlying drivers of disease.”