ChemoCentryx (NASDAQ: CCXI) develops drugs for orphan diseases, meaning those with fewer than 200,000 patients in the U.S. Currently, ChemoCentryx has four drug candidates in development with the most advanced being avacopan. This quarter, ChemoCentryx will announce pivotal trial results with avacopan in a make-or-break binary event for investors.
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Avacopan's pivotal phase 3 clinical trial seeks to help patients with anti-neutrophil cytoplasmic autoantibody (ANCA) vasculitis. Simply put, the patient's immune system activates a specific type of white blood cells, called neutrophils, that then attack the small blood vessels, causing inflammation and swelling. The irreversible damage can lead to kidney failure. According to ChemoCentryx, ANCA vasculitis afflicts between 40,000 to 75,000 patients in the U.S. and 50,000 to 100,000 patients in Europe.