Orphan Drug Status Granted for C3 Glomerulopathy Treatment

The Food and Drug Administration (FDA) has granted Orphan Drug designation to avacopan (ChemoCentryx) for the treatment of C3 glomerulopathy (C3G), a severe kidney disease characterized by deposition of complement protein in the glomeruli.

Patients with C3G experience progressive deterioration in renal function, ultimately leading to renal failure requiring dialysis and kidney transplant, if left untreated. Currently, there is no approved treatment, but non-specific immunosuppressants are frequently used. Kidney transplantation is the only option; however, relapse frequently occurs, with the new kidney manifesting the disease. 

Avacopan is currently under clinical development for the treatment of C3G. ChemoCentryx intends to initiate a multicenter clinical endpoint study in patients with C3G in the first half of 2017. In addition to C3G, the company is also evaluating avacopan for the treatment of anti-neutrophil cytoplasmic auto-antibody-associated vasculitis (AAV) and atypical hemolytic uremic syndrome (aHUS). The FDA has granted Orphan Drug designations to all three indications.

Avacopan (CCX168) is an oral, small molecule, selective complement C5a receptor (C5aR) inhibitor.

For more information visit ChemoCentryx.com.