The Food and Drug Administration (FDA) has granted Orphan Drug designation to sparsentan (Travere Therapeutics) for the treatment of immunoglobulin A (IgA) nephropathy.
IgA nephropathy (IgAN) is a rare disease characterized by the accumulation of IgA in the kidneys, which causes cellular changes within the glomeruli and damages the glomerular filtration barrier. Sparsentan is an investigational small molecule candidate designed to selectively block the receptors of endothelin type A and angiotensin II type I, which are associated with kidney disease progression.
The Company is currently evaluating the efficacy and safety of sparsentan in the pivotal phase 3 PROTECT study in patients with IgAN. Patients were randomized to receive either sparsentan or irbesartan (active control). The primary end point is the change from baseline in urine protein-to-creatinine ratio at week 36. Topline study data are expected in the third quarter of 2021.
“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,” said Noah Rosenberg, MD, chief medical officer of Travere Therapeutics. “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.”
Sparsentan is also being investigated for the treatment of focal segmental glomerulosclerosis in the phase 3 DUPLEX study.
For more information visit travere.com.
Travere Therapeutics announces Orphan Drug designation for sparsentan for the treatment of IgA nephropathy. [press release]. San Diego, CA: Travere Therapeutics, Inc.; January 12, 2021.