The Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for avacopan (ChemoCentryx) for 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 designed to selectively block the complement 5a receptor (C5aR) on destructive inflammatory cells such as blood neutrophils. 

The NDA includes data from the double-blind, active-controlled phase 3 ADVOCATE trial, which evaluated the efficacy and safety of avacopan in 331 adult patients with ANCA-associated vasculitis. Findings from the study showed that 72.3% of avacopan-treated patients achieved disease remission (as assessed by Birmingham Vasculitis Activity Score) at week 26 compared with 70.1% of prednisone-treated patients, establishing noninferiority of the investigational treatment (P <.0001). At week 52, avacopan demonstrated statistical superiority in sustained remission compared with prednisone (65.7% vs 54.9%, respectively; P =.0066). 

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The safety profile of avacopan was found to be favorable with fewer serious adverse events reported compared with prednisone.

A Prescription Drug User Fee Act (PDUFA) target date of July 7, 2021 has been set for this application.

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ChemoCentryx announces FDA acceptance of the avacopan New Drug Application (NDA) for the treatment of ANCA-associated vasculitis. Accessed September 17, 2020. 

This article originally appeared on MPR