ChemoCentryx, Inc has submitted a New Drug Application (NDA) to the Food and Drug Administration (FDA) for avacopan in the treatment of antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis.
ANCA-associated vasculitis is a systemic disease characterized by the inflammation and destruction of small blood vessels resulting in organ damage and failure. Avacopan is a first-in-class, orally-administered, small molecule that works by selectively blocking the complement 5a receptor (C5aR) on destructive inflammatory cells such as blood neutrophils.
The NDA is supported by data from the global, double-blind, active-controlled phase 3 ADVOCATE trial that evaluated the efficacy and safety of avacopan in 331 adult patients with ANCA-associated vasculitis. Patients were randomized to receive avacopan or prednisone, in addition to either rituximab or cyclophosphamide (followed by azathioprine/mycophenolate). The co-primary end points were the proportion of patients achieving disease remission as assessed by Birmingham Vasculitis Activity Score (BVAS) at week 26 and sustained disease remission as assessed by BVAS at week 52.
Results showed that at week 26, BVAS remission was achieved by 72.3% of avacopan-treated patients and 70.1% of prednisone-treated patients, establishing noninferiority of the investigational treatment (P <.0001). At week 52, sustained remission was observed in 65.7% of patients treated with avacopan vs 54.9% of those in the prednisone group (P =.0066). Additionally, treatment with avacopan was associated with significant reduction in glucocorticoid toxicity, as well as improvements in renal function and health-related quality of life measures (all secondary end points).
The safety profile of avacopan was found to be favorable with fewer serious adverse events reported compared with prednisone.
“There is an urgent need for a nonimmunosuppressive, targeted therapy that can achieve and sustain remission in this organ- and life-threatening disease, while reducing the toxicities associated with daily steroid use,” said Thomas J. Schall, PhD, President and CEO of ChemoCentryx. “Submission of our NDA is a critical step toward addressing this unmet need, as we seek to improve patients’ lives.”
The FDA previously granted Orphan Drug designation to avacopan for this indication.
For more information visit chemocentryx.com.