Rare Daily Staff
The U.S. Food and Drug Administration granted Travere Therapeutics full approval and expanded use of Filspari to slow kidney function decline in adults with the rare kidney disease primary IgA nephropathy who are at risk of disease progression.
The agency granted full approval based on positive long-term confirmatory results from the PROTECT study, which demonstrated that Filspari significantly slowed kidney function decline over two years compared to irbesartan, a high blood pressure medicine that is often used to treat the condition.
The FDA granted Filspari accelerated approval in February 2023 based on the surrogate marker of protein levels in urine.
IgA nephropathy (IgAN), also called Berger’s disease, is a rare progressive kidney disease characterized by the buildup of immunoglobulin A (IgA), a protein that helps the body fight infections, in the kidneys. The deposits of IgA cause a breakdown of the normal filtering mechanisms in the kidney, leading to blood in the urine, protein in the urine, and a progressive loss of kidney function. Other symptoms of IgAN may include swelling and high blood pressure.
Filspari is the only oral, once-daily, non-immunosuppressive medication that directly targets glomerular injury in the kidney by blocking two critical pathways of IgAN disease progression.
“We know that most people living with IgAN are at risk of disease progression and are seeking a safe, effective and convenient treatment option that can help preserve their kidney function,” said Eric Dube, president and CEO of Travere Therapeutics. “Full approval now enables physicians to confidently prescribe Filspari more broadly as a once-daily, oral, non-immunosuppressive treatment, that can provide superior preservation of kidney function and replace current standard of care.”
The two-year efficacy data contained in the FDA-approved label is a modified intention to treat analysis evaluates data from all patients regardless of treatment discontinuation. In the final analysis of the 404 randomized patients, Filspari significantly reduced the rate of decline in kidney function from baseline to Week 110 compared to irbesartan.
The positive treatment effects on proteinuria compared to the active control irbesartan that were observed at Week 36 were durable out to the two-year measurement period.
Filspari was well tolerated with a clearly defined safety profile that has been consistent across all clinical trials conducted to date.
“The expanded indication and full approval of FILSPARI is welcome news for the rare kidney disease community,” said Josh Tarnoff, CEO of NephCure. “We have waited a long time for a medicine to slow the irreversible kidney damage from IgAN.”
Photo: Eric Dube, president and CEO of Travere Therapeutics
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