Travere Therapeutics Announces Orphan Drug Designation for Sparsentan for the Treatment of IgA Nephropathy
January 13, 2021 at 03:00 am IST
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Travere Therapeutics, Inc. announced that 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. 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.
Travere Therapeutics, Inc. is a biopharmaceutical company. The Company is focused on identifying, developing, and delivering life-changing therapies to people living with rare kidney and metabolic diseases. Its product, FILSPARI (sparsentan), is indicated to reduce proteinuria in adults with primary IgAN at risk of rapid disease progression. Sparsentan is also in late-stage development for focal segmental glomerulosclerosis (FSGS). The Companyâs Pegtibatinase is a novel investigational human enzyme replacement candidate being evaluated for the treatment of classical homocystinuria (HCU), which is a rare metabolic disorder. Its commercial products, Thiola and Thiola EC are for the treatment of cystinuria, a rare genetic cystine transport disorder that causes high cystine levels in the urine and the formation of recurring kidney stones. It is also engaged in the identification of potential small molecule therapeutics for Alagille syndrome (ALGS).