FDA Likely to Extend PDUFA Date for Travere’s Sparsentan for IgA Nephropathy by Three Months

Travere Therapeutics said that following late-cycle review interactions with the U.S. Food and Drug Administration, the company expects the previously assigned Prescription Drug User Fee Act (PDUFA) target action date of November 17, 2022, for its New Drug Application for accelerated approval of sparsentan for the treatment of IgA nephropathy to be extended by three months.

Photo: Eric Dube, president and CEO of Travere Therapeutics

As part of its late-cycle review, the FDA has requested that the company update its proposed Risk Evaluation Mitigation Strategy (REMS) to include liver monitoring for sparsentan consistent with certain other approved products in the endothelin receptor antagonist class. Travere expects to submit an updated REMS plan in the coming days. Based upon feedback from the FDA, the updated submission is likely to be considered a major amendment to the NDA and is expected to result in a three-month extension of the PDUFA target action date to allow sufficient time to review the information. No additional clinical data or studies have been requested as part of the application review process.

“While this request for additional monitoring within the REMS came unexpectedly, the strength of the clinical data supporting the profile of sparsentan and our confidence in the potential for sparsentan to be approved as a new therapy for IgA nephropathy remain unchanged,” said Eric Dube, president and CEO of Travere Therapeutics. “Many people living with IgA nephropathy continue to face a progression of disease with no currently approved non-immunosuppressive treatments available. We will use the additional time to work collaboratively with the FDA as we continue the labeling process, and further prepare for launch with the goal of enabling sparsentan to ultimately become a new treatment standard, if approved.”

IgA nephropathy (IgAN), also known as Berger’s disease, is a rare chronic kidney disorder in which an estimated 20 to 40 percent of patients progress to end-stage renal disease within 10 to 20 years of diagnosis. IgAN is often defined by progressive proteinuria, hematuria, and acute onset of nephrotic syndrome. The disorder is estimated to affect more than 100,000 people in the U.S. and is one of the leading causes of acute nephritis in Europe and Japan.

Sparsentan, a dual endothelin angiotensin receptor antagonist, is a novel investigational product candidate selectively targeting the endothelin A receptor and the angiotensin II subtype 1 receptor. Pre-clinical data have shown that blockade of both endothelin type A and angiotensin II type 1 pathways in forms of rare chronic kidney disease, reduces proteinuria, protects podocytes, and prevents glomerulosclerosis and mesangial cell proliferation. Sparsentan has been granted Orphan Drug designation for the treatment of IgAN in the U.S. and Europe.

Author: Rare Daily Staff