Travere Therapeutics, Inc. announced the completion of a successful pre-NDA meeting with the U.S. Food and Drug Administration (FDA) for FILSPARI® (sparsentan) in IgA nephropathy (IgAN). The Company will submit a supplemental New Drug Application (sNDA) in the first quarter of 2024 for conversion of the existing U.S. accelerated approval of FILSPARI to full approval. The Company also completed regulatory engagement on focal segmental glomerulosclerosis (FSGS) in which the FDA communicated that the Phase 3 DUPLEX Study results alone are not sufficient to support an sNDA submission for an FSGS indication for sparsentan.

As a result, the Company will be conducting additional analyses of FSGS data with plans to re-engage FDA in 2024, and is implementing a strategic reorganization in Fourth Quarter 2023 to focus near-term resources on the ongoing FILSPARI launch in IgAN and the advancement of pegtibatinase in classical homocystinuria (HCU). Following supportive FDA feedback on the Company?s plan to submit the two-year results from the Phase 3 PROTECT Study in IgAN, Travere to submit sNDA in First Quarter 2024 for full U.S. approval of FILSPARI in IgAN The Company recently completed a pre-NDA meeting with the FDA to discuss the confirmatory results from the Phase 3 PROTECT Study and next steps for submission of an sNDA for full approval. The FDA indicated support for the Company?s plan to submit an sNDA for conversion from accelerated approval to full approval and the Company is on-track to submit the sNDA in First Quarter 2024.

Following engagement with FDA on the two-year results from the Phase 3 DUPLEX Study of sparsentan in FSGS, Company conducting additional analyses of FSGS data The Company also completed its planned Type C meeting with the FDA to discuss previously reported results from the Phase 3 DUPLEX Study of sparsentan in FSGS. The FDA acknowledged the high unmet need for approved therapies as well as the challenges in studying FSGS but indicated that the two-year results from the Phase 3 DUPLEX Study alone are not sufficient to support an sNDA submission. The FDA acknowledged the work being done by the larger nephrology community to better understand proteinuria and eGFR as endpoints in clinical trials of FSGS and indicated a willingness to continue to engage with the Company on a potential path forward for sparsentan in FSGS following the Company?s consideration of additional evidence.

The Company plans to re-engage with the FDA later in 2024. Strategic Reorganization: To further align the Company?s resources on the ongoing FILSPARI launch and its planned Phase 3 program advancing the development of pegtibatinase as the first potential disease-modifying treatment for HCU, Travere is implementing an approximate 20% workforce reduction focused on non-field-based employees. These adjustments are expected to result in an estimated annualized savings of approximately $25 million beginning in 2024, and an estimated non-recurring charge of approximately $12 million to $14 million primarily recognized in the fourth quarter of 2023.

Alongside other cost reduction measures planned, the Company now expects operating expenses of less than $400 million in 2024, exclusive of cost of goods sold, depreciation and amortization and expected net milestone payments to be made of approximately $50 million. Together with $634.6 million in cash and cash equivalents as of September 30, 2023, the Company expects to have a cash runway into 2028. About IgA Nephropathy: IgA nephropathy (IgAN), also called Berger's disease, is a rare progressive kidney disease characterized by the buildup of immunoglobulin A (IgA), a protein that helps the body fight infections, in the kidneys.

The deposits of IgA cause a breakdown of the normal filtering mechanisms in the kidney, leading to blood in the urine (hematuria), protein in the urine (proteinuria) and a progressive loss of kidney function. Other symptoms of IgAN may include swelling (edema) and high blood pressure. IgAN is the most common type of primary glomerulonephritis worldwide and a leading cause of kidney failure due to glomerular disease.

IgAN is estimated to affect up to 150,000 people in the U.S. and is one of the most common glomerular diseases in Europe and Japan.