EC Grants Accelerated Approval to Travere and CSL Vifor’s Filspari for Rare Kidney Disease

Rare Daily Staff

The European Commission granted accelerated approval to Travere Therapeutics and CSL Vifor’s Filspari for the treatment of adults with the rare kidney disease primary IgA nephropathy.

The EC granted accelerated approval based on reduction in proteinuria. It has not been established whether Filspari slows kidney function decline in patients with primary IgA nephropathy (IgAN). Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory clinical trial.

Filspari is approved for people with IgAN with a urine protein excretion greater than or equal to 1.0 g/day (or urine protein-to-creatinine ratio greater than or equal to 0.75 g/g). The approval is granted for all member states of the European Union, as well as in Iceland, Liechtenstein, and Norway.

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 (hematuria), protein in the urine (proteinuria) and a progressive loss of kidney function. Other symptoms of IgAN may include swelling and high blood pressure. It is the most common type of primary glomerulonephritis worldwide and a leading cause of kidney failure due to glomerular disease.

Filspari is an endothelin and angiotensin II receptor antagonist indicated to reduce proteinuria in adults with primary IgAN at risk of rapid disease progression. In 2021, Travere Therapeutics granted CSL Vifor exclusive commercialization rights for Filspari in Europe, Australia and New Zealand.

“This is a significant step forward for patients in Europe living with IgAN, a rare and serious condition, and a leading cause of end-stage renal disease,” said Jürgen Floege, senior professor in the Division of Nephrology and Clinical Immunology at the University Hospital, RWTH Aachen, Germany, and steering committee member for the PROTECT Study on which the approval was based. “The approval of this innovative treatment is based on data from the only head-to-head phase 3 clinical trial in IgAN. Adult patients with IgAN who are at high risk of progressing to kidney failure will now have access to a new therapy that significantly reduces proteinuria and slows the progression of kidney disease.”

Photo: Jürgen Floege, senior professor in the Division of Nephrology and Clinical Immunology at the University Hospital, RWTH Aachen, Germany