FDA clears Travere’s rare kidney disease drug, will come with REMS program

17 Feb 2023
endpts.com
Accelerated ApprovalPhase 3Drug Approval
The FDA approved Travere Therapeutics’ treatment for a rare kidney disease on Friday, notching a win for the company after the review of the drug was delayed by three months last year. The accelerated approval for sparsentan means Travere can now deliver the drug, which will be sold as Filspari, to patients with IgA nephropathy. In IgAN, or Berger’s disease, immunoglobulin builds up and disrupts the waste filtration process in the kidneys, which can lead to blood and protein in urine. The approval comes with a warning about liver and fetal toxicity, and the company will have to run a risk evaluation and mitigation strategy, or REMS, as part of the approval. It will require doctors to go through special training, and patients to be enrolled in a monitoring program, in order for the drug to be prescribed. The company plans to charge $9,900 for a 30-day supply, or about $120,000 a year. Travere estimates that there are 30,000 to 50,000 patients who could get the drug as of its launch. The FDA’s original Nov. 17 decision date was pushed back after the agency asked Travere to update its REMS, typically meant to monitor drugs that carry more significant potential for side effects. CEO Eric Dube previously said on a conference call the update to liver monitoring was “consistent with some of the other products in the endothelin receptor antagonistendothelin receptor antagonist class.” A confirmatory study of the drug is ongoing, with topline results expected in the fourth quarter to support a request for traditional approval, the company has said. Travere is scheduled to report full-year 2022 results next week. SVB Securities analysts have said that a REMS program for liver monitoring would “undoubtedly be an added burden for physicians and patients,” but that they thought it could be removed with more clinical data. They cited the example of other endothelin receptor antagonistsendothelin receptor antagonists such as Gilead’s Letairis, which is used to treat pulmonary arterial hypertension. Other biotechs are also attempting to treat IgAN. Calliditas Therapeutics won accelerated approval for the delayed-release version of an old steroid for the reduction of proteinuria (protein in the urine) in primary IgAN back in December 2021. Seattle-based Chinook Therapeutics anticipates requesting an accelerated approval for its drug in the third quarter, Novartis expects a Phase III readout in the indication this year and Vera Therapeutics is headed toward late-stage studies. Travere has tried twice to get accelerated approval of sparsentan in another indication, focal segmental glomerulosclerosis (FSGS), in which scarring forms on the kidneys. Topline results from two-year confirmatory endpoints will come out next quarter for that indication, Travere previously said , noting a supplemental NDA would be filed in the second half of this year, depending on the results. The San Diego biotech rebranded in fall 2020 to rid itself of ties to the legacy name of Retrophin, the company founded by “Pharma Bro” Martin Shkreli in 2011. Travere’s moniker stems from Latin roots of the words “path” (tractus) and “truth” (ver). Editor’s note: This story has been updated to include the cost of the drug.
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