Travere Therapeutics' FILSPARI® Approved by FDA for IgA Nephropathy

JUPITER, Fla.--Ligand Pharmaceuticals Incorporated announced that its collaborator, Travere Therapeutics, Inc., has secured full approval from the U.S. Food and Drug Administration (FDA) for FILSPARI® (sparsentan). This medication aims to decelerate the decline in kidney function in adults suffering from primary IgA nephropathy (IgAN) who are at an elevated risk of disease progression. Ligand stands to benefit from milestone payments and a 9% royalty on the global net sales of FILSPARI.

Initially granted accelerated approval in February 2023 based on proteinuria as a surrogate marker, FILSPARI now has full FDA approval. This approval comes on the back of long-term confirmatory results from the PROTECT Study, which demonstrated FILSPARI's superior efficacy over irbesartan in slowing kidney function decline over two years.

"This marks a significant achievement for Travere and presents a promising development for patients battling this rare kidney condition," stated Todd Davis, CEO of Ligand. "We are optimistic about Travere’s efforts to promote FILSPARI in the U.S. and are eagerly anticipating its European launch soon. As a key component of Ligand’s commercial-stage royalty portfolio, FILSPARI is expected to drive considerable revenue growth in the coming years."

FILSPARI distinguishes itself as the only oral, once-daily, non-immunosuppressive medication that targets glomerular injury in the kidney by inhibiting two critical pathways involved in IgAN disease progression: endothelin-1 and angiotensin II.

The FDA-approved label includes two-year efficacy data, analyzed through a modified intention-to-treat (ITT) approach, evaluating all patients irrespective of treatment discontinuation. In a final analysis of 404 randomized participants, FILSPARI showed a significant reduction in the rate of kidney function decline from baseline to Week 110 compared to irbesartan. Specifically, the mean estimated glomerular filtration rate (eGFR) slope from baseline to Week 110 was -3.0 mL/min/1.73 m^2/year for FILSPARI, versus -4.2 mL/min/1.73 m^2/year for irbesartan, indicating a statistically significant treatment effect of 1.2 mL/min/1.73 m^2/year (p=0.0168). The treatment benefits on proteinuria observed at Week 36 persisted through the two-year measurement period, resulting in a 3.8 mL/min/1.73 m^2 difference in the mean change from baseline between FILSPARI and irbesartan.

The PROTECT Study also highlighted FILSPARI's well-defined safety profile, consistent across all conducted clinical trials. Following discussions with the FDA, the company plans to submit a supplemental New Drug Application (sNDA) for potential modifications to the liver-monitoring requirements of the Risk Evaluation and Mitigation Strategy (REMS).

IgA nephropathy, also known as Berger's disease, is a rare and progressive kidney disorder characterized by the accumulation of immunoglobulin A (IgA) in the kidneys. This condition disrupts the kidneys' filtering mechanisms, leading to hematuria, proteinuria, and progressive kidney function decline. Symptoms may also include edema and high blood pressure. IgAN is the most prevalent type of primary glomerulonephritis worldwide and a leading cause of kidney failure due to glomerular disease, affecting up to 150,000 people in the U.S.

The PROTECT Study is one of the most extensive interventional studies in IgAN and the only Phase 3 head-to-head trial in this rare kidney disease. It is a global, randomized, multicenter, double-blind, parallel-arm, active-controlled clinical trial evaluating the safety and efficacy of 400 mg of FILSPARI compared to 300 mg of irbesartan in 404 patients with IgAN and persistent proteinuria.

The primary efficacy endpoint for the interim analysis was the change from baseline in urine protein/creatinine ratio at Week 36. For the final analysis, the key secondary endpoint was the rate of change in eGFR over 110 weeks. Results showed that after 36 weeks, patients on FILSPARI experienced a mean reduction in proteinuria of 49.8% from baseline, compared to a 15.1% reduction for those treated with irbesartan (p<0.0001).

Patients who completed the PROTECT study's double-blind portion were eligible to join the open-label extension of the trial.

Ligand Pharmaceuticals is a biopharmaceutical firm that fosters scientific advancement by supporting the clinical development of high-value medicines. Its business model aims to generate value for stockholders through a diversified portfolio of biopharmaceutical revenue streams, supported by an efficient corporate structure. Ligand partners with other pharmaceutical companies to leverage their expertise in late-stage development, regulatory management, and commercialization.

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