Travere Therapeutics has said the US FDA has approved FILSPARI (sparsentan) to reduce proteinuria in adults and children aged 8 and older with focal segmental glomerulosclerosis (FSGS) without nephrotic syndrome. This marks a major expansion of its rare kidney disease franchise.
The decision makes FILSPARI the first and only FDA-approved treatment for FSGS, extending its use beyond IgA nephropathy (IgAN), where it is already the most commonly prescribed approved therapy.
The company estimates more than 30,000 people in the US with FSGS without nephrotic syndrome may be eligible for treatment.
“Today marks a historic milestone for people living with FSGS, who for the first time have an FDA-approved medicine for this rare and devastating condition,” said Eric Dube, president and chief executive officer of Travere Therapeutics.
“This approval reflects years of perseverance and our belief that those living with FSGS deserve better. It also builds on our leadership and progress in rare kidney diseases, expanding FILSPARI’s potential reach to more than 100,000 people in the U.S. with FSGS and IgAN who need better treatment options.
FILSPARI will be available for nephrologists to immediately prescribe to individuals with FSGS. We are profoundly grateful to the patients, caregivers, investigators, healthcare providers, regulators and advocates who made this moment possible.”
The FDA decision is backed by Phase 3 DUPLEX Study data, the largest interventional FSGS trial to date. In the overall population, FILSPARI reduced proteinuria by 46% from baseline to Week 108 versus 30% with maximum labeled dose irbesartan.
In patients without nephrotic syndrome, the effect was even stronger, with a 48% reduction versus 27% for irbesartan. Kidney function, measured by eGFR, was also better preserved, with a treatment difference of 1.1 mL/min/1.73 m² over 108 weeks.
“...approval of FILSPARI provides nephrologists with a new FDA-approved option for patients living with FSGS,” said Kirk Campbell, president of the National Kidney Foundation and the C. Mahlon Kline Professor and chief of the Division of Renal-Electrolyte and Hypertension in the Perelman School of Medicine at the University of Pennsylvania.
“For decades, treatment options have been limited, often relying on off-label therapies such as long-term steroids that can carry a significant burden for patients. In the DUPLEX Study, FILSPARI delivered rapid and sustained reductions in proteinuria compared to irbesartan, with particularly meaningful effects in patients without nephrotic syndrome.
"This is consistent with KDIGO guidance, which emphasize reducing proteinuria as a key strategy to slow disease progression in FSGS. For patients without active nephrotic syndrome, where optimizing foundational therapy is critical, FILSPARI represents an important new option.”
“The approval of FILSPARI as the first medication for people with FSGS is a life-changing moment for patients and families who have waited far too long,” said Josh Tarnoff, chief executive officer of NephCure.
“This milestone reflects the strength and perseverance of an extraordinary collective and broad stakeholder community who participated in research, raised awareness and never gave up hope.
"The pioneering work of the PARASOL project and the countless patients and caregivers who have shared their journeys along the way have helped make this progress possible. It stands as proof that when science and community unite with purpose, we can redefine what’s possible in rare disease care and bring light to those who have long lived in uncertainty.”
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