Travere Announces Orphan Drug Designation for IgA Nephropathy Treatment

Travere Therapeutics announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to sparsentan for the treatment of IgA nephropathy (IgAN), a rare kidney disorder and a leading cause of end-stage kidney disease (ESKD). Sparsentan is an investigational product candidate currently being evaluated for the treatment of IgAN, as well as focal segmental glomerulosclerosis (FSGS) in pivotal Phase 3 clinical trials that are expected to report topline data from interim proteinuria assessments in 2021.

“People living with IgAN face a significant unmet need with limited treatment options available,” said Noah Rosenberg, M.D., chief medical officer of Travere Therapeutics. “Obtaining Orphan Drug Designation is another milestone in our development program as we continue to advance towards the goal of delivering sparsentan as a potential new treatment standard for people living with IgAN. We continue to look forward to topline data from the interim proteinuria assessment in the ongoing Phase 3 PROTECT Study in IgAN during the third quarter of this year.”

The Orphan Drug Designation program is intended to encourage the development of therapeutics for diseases that affect fewer than 200,000 individuals in the United States. Prior to FDA approval, Orphan Drug Designation qualifies sponsors for certain incentives, such as tax credits toward the cost of clinical trials and prescription drug user fee waivers. Orphan Drug Designation may also convey seven years of marketing exclusivity for sparsentan if approved by the FDA for the treatment of IgAN. Travere Therapeutics previously received a positive opinion from the European Medicines Agency Committee for Orphan Medicinal Products on the company’s application for Orphan Drug Designation for IgAN in Europe, and it holds Orphan Drug Designation for sparsentan for the treatment of FSGS in the U.S. and Europe.