Benzinga | Feb 18, 2021 04:36PM EST

Travere Therapeutics Announces European Commission Has Granted Orphan Designation to Sparsentan for the Treatment of IgA Nephropathy

Travere Therapeutics, Inc. (NASDAQ:TVTX) today announced the European Commission (EC) has granted orphan 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 in a pivotal Phase 3 clinical study for the treatment of IgAN, as well as a pivotal Phase 3 clinical study for the treatment of focal segmental glomerulosclerosis (FSGS). The Company recently reported that the ongoing DUPLEX Study of sparsentan in FSGS achieved its pre-specified interim proteinuria endpoint and that preliminary results from the interim analysis suggest that to date in the study, sparsentan has been generally well-tolerated and has shown a comparable safety profile to irbesartan. Topline data from the interim proteinuria assessment in the ongoing PROTECT Study of sparsentan in IgAN remain on track to be reported in the third quarter of 2021.

"The limited and non-specific therapeutic strategies used for IgAN today are often associated with long-term tolerability challenges, and for many are not enough to slow the progression to ESKD," said Noah Rosenberg, M.D., chief medical officer of Travere Therapeutics. "We are pleased to receive orphan designation in Europe which further supports our goal of ultimately delivering sparsentan as a potential new treatment standard for IgAN."

Orphan designation from the EC provides incentives for companies to develop medicines intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating and where no satisfactory treatment is currently authorized. The prevalence of the condition must not exceed more than five in 10,000 people in the European Union (EU). In addition to being eligible for a 10-year period of marketing exclusivity in the EU upon product approval, orphan designation provides fee waivers, protocol assistance, and eligibility for marketing authorization under the centralized procedure granting approval in all EU countries. Travere Therapeutics was previously granted Orphan Drug designation for IgAN in the U.S., and it also holds Orphan Drug designation in the U.S. and orphan designation in Europe for sparsentan for the treatment of FSGS.