Travere Reorganizes to Focus on Full Approval of Filspari in IgAN, Cuts 20 Percent of Workforce
December 5, 2023
Rare Daily Staff
Travere Therapeutics said it would be reducing its workforce as it works with the U.S. Food and Drug Administration to obtain full approval of Filspari in IgA nephropathy and re-engage with the agency over the drug’s use in focal segmental glomerulosclerosis.
IgA nephropathy (IgAN) 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, protein in the urine, and a progressive loss of kidney function. Other symptoms of IgAN may include swelling (edema) and high blood pressure. 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.
Focal segmental glomerulosclerosis (FSGS) is a rare proteinuric kidney disorder in both children and adults that is estimated to affect more than 40,000 patients in the United States with similar prevalence in Europe. The disorder is defined by progressive scarring of the kidney and often leads to kidney failure. FSGS is characterized by proteinuria, where protein leaks into the urine due to a breakdown of the normal filtration mechanism in the kidney. Once in the urine, protein is considered to be toxic to other parts of the kidney, especially the tubules, and is believed to contribute to further disease progression. There is currently no approved therapeutic indicated for the treatment of FSGS.
After completing a pre-NDA meeting with FDA, Travere plans to submit a supplemental New Drug Application to convert the accelerated approval of Filspari in IgAN in the first quarter of 2024. The company also completed regulatory engagement on FSGS in which the FDA communicated that the phase 3 DUPLEX study results alone are not sufficient to support a supplemental NDA submission for an FSGS indication for Filspari. As a result, Travere said it will be conducting additional analyses of FSGS data with plans to re-engage FDA in 2024.
In order to focus near-term resources on the ongoing Filspari launch in IgAN and the advancement of pegtibatinase in classical homocystinuria (HCU), Travere is implementing a strategic reorganization in Q4 2023 that involves an approximate 20 percent workforce reduction focused on non-field-based employees, which is expected to extend its cash runway into 2028.
“Following a successful pre-NDA meeting, we are pleased to be moving forward with our planned sNDA submission for full approval of Filspari in IgA nephropathy,” said Eric Dube, president and CEO of Travere Therapeutics. “Unfortunately, there is uncertainty around a regulatory path forward for FSGS. While we intend to continue to engage with FDA on a way forward for the more than 40,000 people living with FSGS in the U.S., we must at the same time prioritize our operating expenses. As a result, we have made the difficult decision to reduce our workforce to further focus our resources on the Filspari launch and clinical development of pegtibatinase; this action is expected to extend our cash runway into 2028.”
In September, Travere reported two-year confirmatory secondary endpoint results from the pivotal head-to-head phase 3 PROTECT study of Filspari in IgA nephropathy (IgAN) versus irbesartan. While Filspari demonstrated long-term kidney function preservation and achieved a clinically meaningful difference in estimated glomerular filtration rate (eGFR) total and chronic slope versus irbesartan, it narrowly missing statistical significance in eGFR total slope while achieving statistical significance in eGFR chronic slope for purposes of regulatory review in the EU. After discussing the confirmatory two-year results, Travere plans to submit the sNDA in the first quarter of 2024 for full U.S. approval of Filspari in IgAN.
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. Travere plans to re-engage with the FDA later in 2024.
Photo: Eric Dube, president and CEO of Travere Therapeutics
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