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First FDA-Approved Treatment for Patients with Focal Segmental Glomerulosclerosis—A Rare Kidney Condition

The U.S. Food and Drug Administration on Monday approved a new indication for Filspari (sparsentan) tablets for reducing proteinuria (abnormal levels of protein in the urine) in patients aged eight years and older with focal segmental glomerulosclerosis (FSGS) without nephrotic syndrome. This approval marks an important milestone because it provides the first FDA-approved treatment for patients living with this rare kidney condition.

Filspari is a dual endothelin and angiotensin II receptor antagonist. It was previously approved to slow kidney function decline in adults with primary immunoglobulin A nephropathy who are at risk for disease progression. FSGS is a rare kidney condition where small portions of the kidney's filters become scarred and damaged. This damage causes protein to leak into the urine and may eventually cause the kidneys to stop working properly.

"For the first time, children and adults with a common form of the rare, progressive kidney condition of FSGS have an FDA approved treatment," said Center for Drug Evaluation and Research (CDER) Acting Director Tracy Beth Høeg, M.D., Ph.D. "Full approval was granted based on a randomized trial demonstrating significant improvement in proteinuria in patients without nephrotic syndrome compared with standard of care. Based on the available data, our FDA reviewers concluded that Filspari’s effect on proteinuria in this FSGS subpopulation had a high likelihood of translating into clinically meaningful benefit of slowing the loss of kidney function and progression to kidney failure. The collaborative process between the division and the sponsor reflect our agency’s commitment to identifying and approving effective treatments for rare diseases and conditions.”

Because of the risk of hepatotoxicity, Filspari is available only through a restricted drug distribution program, under a risk evaluation and mitigation strategy (REMS).The REMS requires health care professionals who prescribe Filspari, and pharmacies that dispense Filspari, to be specially certified in the REMS, and that patients be enrolled in the REMS. Healthcare providers must obtain liver tests prior to initiation of treatment and every three months during treatment.

The most common side effects of Filspari in patients with FSGS (≥5%) are peripheral edema (swelling in the extremities), hypotension (blood pressure that is too low, including orthostatic hypotension), hyperkalemia (elevated blood potassium levels), dizziness, and anemia (low red blood cell counts).

The FDA granted approval of Filspari to Travere Therapeutics, Inc. This product was also granted orphan-drug designation in 2015.

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