Reata Pharmaceuticals $RETA said the European Commission (EC) granted orphan drug designation to the company’s investigational drug bardoxolone methyl (bardoxolone) for the treatment of Alport syndrome.
Alport syndrome is a rare, genetic form of chronic kidney disease (CKD) caused by mutations in genes encoding type IV collagen, which is a major structural component of the glomerular basement membrane in the kidney. Many Alport syndrome patients develop CKD and progress to end-stage renal disease.
Bardaxolone has completed the phase II portion of a planned phase II/III study; the phase II results demonstrated a statistically significant glomerular filtration rate (eGFR) after 36 weeks for the treatment group.
Reata CEO Warren Huff noted there are no currently approved treatments for Alport syndrome.
Orphan designation in Europe to drugs, which provides specific regulatory and financial incentives, is awarded to treatments aimed at life-threatening or chronically debilitating diseases that affect no more than five in 10,000 people in the European Union.