Drugs

Travere’s Filspari Approved for Rare Kidney Disease FSGS

FDA has granted full approval for Travere Therapeutics’ Filspari (sparsentan), a therapy for treating patients with Focal Segmental Glomerulosclerosis (FSGS), marking the first approved medicine specifically for the rare kidney disorder. Sparsentan is described as a first-in-class medication used to slow kidney function decline in adults with primary immunoglobulin A nephropathy who are at risk of rapid disease progression. It was granted full FDA approval 9/2024 after previously receiving accelerated approval 2/2023.

The new approval allows Filspari to be used to reduce proteinuria in adults and children aged eight years and older with FSGS who do not have nephrotic syndrome. The decision significantly expands the drug’s use beyond its existing indication in IgA nephropathy, positioning it as a treatment option for a broader population of patients with rare kidney diseases.

FSGS is a progressive condition characterized by scarring of the kidney’s filtering units, often leading to kidney failure. Until now, treatment options have largely relied on off-label therapies, including steroids, with limited efficacy and significant side effects.

Travere estimates that more than 30,000 patients in the U.S. with FSGS — specifically those without nephrotic syndrome — could be eligible for treatment under the new indication. Combined with patients treated for IgA nephropathy, the total addressable population for Filspari exceeds 100,000 individuals, it says.

The approval decision was supported by results from the Phase 3 DUPLEX study, which showed that patients treated with Filspari experienced a 46% reduction in proteinuria over 108 weeks, compared with a 30% reduction for those receiving irbesartan.

Filspari works through a dual mechanism targeting endothelin A and angiotensin II receptors, pathways associated with inflammation and scarring in the kidney, according to the company.

The drug carries a Boxed Warning for liver toxicity and embryo-fetal toxicity and is available only through a Risk Evaluation and Mitigation Strategy program requiring monitoring and enrollment by prescribers and patients.

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