Vifor Fresenius Medical Care Renal Pharma (VFMCRP ) and ChemoCentryx have reported positive data from the Phase III clinical trial of avacopan to treat anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis.
ANCA vasculitis is a systemic disease characterised by inflammation and small blood vessel damage. The condition causes organ damage and failure and can lead to death if left untreated.
Avacopan is a selective complement 5a receptor inhibitor intended to prevent damage from destructive inflammatory cells in response to C5a activation.
Named ADVOCATE, the global, double-blind, double-dummied Phase III trial enrolled 331 patients across 20 countries.
According to top-line results, the trial met its primary endpoints of disease remission and sustained remission at 26 and 52 weeks, respectively.
Remission was defined as a zero Birmingham Vasculitis Activity Score (BVAS) and discontinuation of glucocorticoid treatment for the disease for at least the previous four weeks.
Compared to 70.1% of patients treated with the glucocorticoid standard of care, 72.3% on avacopan experienced BVAS remission at week 26. The remission was sustained at 52 weeks in 65.7% versus 54.9% of participants.
Avacopan also demonstrated a significant decrease in glucocorticoid-related toxicity and kidney function improvement in patients who had renal disease at baseline. The drug also led to improvements in health-related quality of life measures.
Safety analysis showed an acceptable profile of the drug. Patients experiencing serious adverse events were fewer in the avacopan arm than the glucocorticoid standard of care arm.
ChemoCentryx president and CEO Thomas Schall said: “Until now, ANCA vasculitis patients have had to endure regimens that contain chronic high doses of steroids and all their noxious effects, but with today’s data it is clear that the time of making patients sick with steroid therapy in an attempt to make their acute vasculitis better may, at last, be over.
“Working with our partner VFMCRP, we plan to make regulatory submissions for full marketing approval to both the European Medicines Agency (EMA) and the Food and Drug Administration (FDA) in 2020.”
VFMCRP holds exclusive rights to commercialise the drug in all markets outside the US. The company sub-licensed the rights in Japan to Kissei Pharmaceutical.