Travere Therapeutics’ Drug Approved for One Rare Kidney Disease Fails in Study of Another

Travere Therapeutics is a startup that mainly focuses on rare forms of diseases. They made a revelation that a Phase 3 study of an investigational therapy for focal segmental glomerulosclerosis, a rare and fatal kidney condition, did not enhance kidney function.

Half of the 371 participants in the research were randomly allocated to receive sparsentan, while the other half got irbesartan, a blood pressure medicine used as a control. For a period of two years, the groups were under observation, and it was found that kidney functionality was better in the sparsentan group compared to the placebo group.

The urine protein levels were lower in those taking the drug, indicating that the therapy was safe. The company also informed its investors of the same.

The higher management of the company informed investors of their potential meeting with regulators from the US and Europe to find a way for treatment. Until then, they stated that it is too early to tell if the corporation would conduct another experiment.

The findings are a significant setback for Travere, who will use the positive side of the trial data for approval application of the drug for use in the US and Europe. The news came after the FDA authorized sparsentan for IgA nephropathy, a rare and severe autoimmune condition that destroys the kidneys.

FSGS, or focal segmental glomerulosclerosis, is caused by the slow development of scar tissue in different sections of the kidney that filter blood. Within 10 to 15 years, the disease can eventually lead to kidney failure.

Approximately 40,000 people in the United States have FSGS, and it is believed that 40% of patients may have a relapse after kidney transplant. Current medications aim to decrease blood pressure and protein accumulation in the urine, but there is no authorized treatment for FSGS.

Travere was known as Retrophin at the time, and it was founded by Martin Shkreli, who was criticized for boosting the prices of pharmaceuticals bought by his firms by as much as 2000% to 5000%. He was eventually imprisoned for cheating investors and ousted by Retrophin’s board of directors in 2014, igniting a court battle that didn’t end until 2019. Retrophin was renamed Travere in 2020.

The company’s founders emphasized that they do not feel the FSGS trial results are negative for Sparsentan’s chances as a therapy for IgA nephropathy. The FDA awarded the medicine fast clearance for IgA nephropathy based on interim data from a separate Phase 3 study, PROTECT, which indicated a statistically significant drop in urine protein levels among individuals on the treatment.

By the end of this year, the business will have two years’ worth of PROTECT data to determine if the decreases in protein levels reported by IgA nephropathy patients translate into better kidney function.

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