ChemoCentryx Drug Wins EU Orphan Status In Rare Vasculitis

Clinical stage biopharmaceutical firm ChemoCentryx announced it has received Orphan Medicinal Product designation from the European Commission for CCX168 as treatment for two forms of anti-neutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis (AAV).

CCX168 is an orally administered complement C5a receptor inhibitor. The EC granted orphan status to the drug for the treatment of microscopic polyangiitis (MPA) and granulomatosis with polyangiitis (GPA), also previously known as Wegener's Granulomatosis. Both conditions are forms of ANCA-AAV.

MPA and GPA are two rare disorders that result in vasculitis or blood vessel inflammation. Vasculities in MPA and GPA patients can lead to tissue damage. MPA usually targets the kidneys, nerves, skin, joints, and lungs, while GPA typically affects the kidneys and respiratory tract. The exact cause of these rare disorders is yet to be determined by medical science.

Dr. Thomas J. Schall, President and CEO of ChemoCentryx, said that the company is delighted with the orphan designation of its lead candidate in the EU. “There exists a significant unmet medical need in these serious and life-threatening disease settings where all current treatment options such as corticosteroids, cyclophosphamide, and rituximab globally suppress the immune system and potentiate the risk of serious infections. We believe that CCX168 may have therapeutic value in treating MPA and GPA since CCX168 targets the chemo-attractant C5a receptor which we believe plays an essential role in the inflammatory process in ANCA-associated diseases.”

The EC’s orphan designation is awarded to drugs that address diseases or conditions that affect less than 5 per 10,000 people in the EU. The status makes the sponsor company eligible to a range of benefits and incentives including fee reductions, protocol assistance, and a ten-year period of marketing exclusivity in the EU upon approval.

CCX168 has also been designated an orphan drug by the U.S. Food and Drug Administration (FDA) for the treatment of atypical hemolytic uremic syndrome (aHUS). The drug has also received orphan status for ANACA-AAV granulomatosis with polyangiitis, microscopic polyangiitis, and Churg-Strauss syndrome. Once approved for its orphan indications, CCX168 will be granted a seven-year period of marketing exclusivity in the U.S.