Orphan Drug Status Granted to Hemolytic Uremic Syndrome Treatment

The FDA has granted orphan drug designation to a treatment for a rare disease that primarily affects kidney function.

CCX168 is an orally administered medication manufactured by ChemoCentryx, Inc, for the treatment of atypical hemolytic uremic syndrome (aHUS), a rare, life-threatening, progressive disease that can lead to organ failure.

"Given the life-threatening nature of aHUS, we are very pleased with the granting of orphan drug designation for CCX168 in this disease," said ChemoCentryx President and Chief Executive Officer Thomas J. Schall, PhD, in a press release. "We are committed to supporting the community of patients with aHUS and look forward to starting our clinical studies in this disease in the coming weeks."

The disease causes chronic blood vessel damage, thrombosis, or clotting within blood vessels, hemolysis or red blood cell rupture, and sudden, progressive organ failure, including kidney failure. A genetic defect involving factors that control the activation of the complement system causes aHUS.

Current options to treat aHUS remain limited, leading to an extremely poor prognosis and quality of life for aHUS patients.

CCX168 acts as an inhibitor that targets the receptor for the complement protein C5a. Data from preclinical trials suggested C5a receptor inhibition plays an important role in reducing micro vasculature thrombosis formation in aHUS.

ChemoCentryx said it will initiate a phase 2 proof-of-concept study in patients with aHUS by the end of this year.