FDA approves expanded indication making FILSPARI available to patients with IgA nephropathy (IgAN) at risk of progression; updated label includes data showing long-term durable benefit on proteinuria and kidney function preservation that accrued over two years

Conversion to full approval based on results from the PROTECT Study, where FILSPARI delivered superior long-term kidney function preservation compared to the active comparator irbesartan in the only Phase 3 head-to-head trial conducted in IgAN

As an oral, non-immunosuppressive and dual acting, once-daily medicine with superior long-term results vs. irbesartan, FILSPARI has the potential to become foundational care in IgAN

Travere Therapeutics, Inc., announced that the U.S. Food and Drug Administration (FDA) has granted full approval to FILSPARI^® (sparsentan) to slow kidney function decline in adults with primary IgAN who are at risk of disease progression. FILSPARI was granted accelerated approval in February 2023 based on the surrogate marker of proteinuria. Full approval is based on positive long-term confirmatory results from the PROTECT Study demonstrating that FILSPARI significantly slowed kidney function decline over two years compared to irbesartan.

“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. 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,” said Eric Dube, Ph.D., president and chief executive officer of Travere Therapeutics. “With KDIGO’s recent draft guidelines recommending FILSPARI as a foundational kidney-targeted therapy and lowering the targeted proteinuria level for all IgAN patients to under 0.5 g/day or 0.3 g/day – FILSPARI is well positioned to become foundational care for IgAN as the treatment landscape evolves. We are grateful to the patients, caregivers, clinical trial investigators, healthcare providers, and advocates who have worked alongside our team at Travere for so many years to help raise the bar on protecting and preserving kidney health for those living with rare kidney disease.”

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 (endothelin-1 and angiotensin II).

The two-year efficacy data contained in the FDA-approved label is a modified intention to treat (ITT) analysis, and as preferred by the FDA, 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. In the ITT analysis included in the label, the mean eGFR slope from baseline to Week 110 was -3.0 mL/min/1.73 m²/year for FILSPARI and -4.2 mL/min/1.73 m²/year for irbesartan, corresponding to a statistically significant treatment effect of 1.2 mL/ min/1.73 m²/year (p=0.0168). 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. Additional results from the PROTECT Study demonstrated the benefit of FILSPARI on absolute eGFR accrued over time and by Week 110 resulted in a 3.8 mL/min/1.73 m² difference in the mean change from baseline between FILSPARI and irbesartan.

Results from the PROTECT Study showed that FILSPARI was well tolerated with a clearly defined safety profile that has been consistent across all clinical trials conducted to date. Following engagement with the FDA, the Company expects to submit an sNDA for a potential modification to the liver-monitoring REMS.

“As a physician who has dedicated my career to treating patients with glomerular diseases, I believe the full approval of FILSPARI for IgAN provides us with a critically important tool for patient management,” said Brad Rovin, M.D., medical director at The Ohio State University Center for Clinical Research Management, director, Division of Nephrology, and steering committee member for the PROTECT Study. “This approval should facilitate patient access to a medication that targets injury directly in the kidney, reduces proteinuria, even to the point of complete remission in some patients, and is more effective than current standard-of-care treatment in preserving kidney function over time. This is a very exciting milestone in the evolution of treating IgAN.”

“Today’s full approval of FILSPARI brings new hope to the IgAN community, and I’m grateful for the progress that has been made in giving patients a new treatment option that can help protect their kidneys,” said Bonnie Schneider, executive director and co-founder of the IgA Nephropathy Foundation.

“The expanded indication and full approval of FILSPARI is welcome news for the rare kidney disease community,” said Josh Tarnoff, chief executive officer of NephCure. “We have waited a long time for a medicine to slow the irreversible kidney damage from IgAN and appreciate Travere’s leadership in championing new endpoints for IgAN that have spurred significant innovation for this rare kidney disease.”

Travere Therapeutics has a comprehensive patient support program, Travere TotalCare^®, to enable a smooth experience for patients, their caregivers, and healthcare providers. This program provides services, assistance, and resources that can help patients understand IgAN, manage the insurance process, fill their prescriptions and initiate treatment.