Comment from Stewart Sandra

I am submitting this comment on the proposed withdrawal of TAVNEOS (Avacopan) as a patient living with ANCA-associated vasculitis (MPO-ANCA). A happenstance blood test in June 2025 revealed a significant impairment in my kidney function. I had virtually no symptoms. In less than a month, I received a phone call telling me that they thought I had AAV. I had a kidney biopsy within two days and started aggressive treatment with pulse steroids and Rituximab in hospital. In that one month since first blood test, my kidney function declined from 30% to 20%. When I was on the phone, I barely had time to register the facts, but afterwards I spent some time reading about the disease online. Rare disease. Fifteen cases per million. Rapid onset. Organ failure. High risk of relapse. And do you know what? I found myself hoping that the biopsy would find cancer. Why? Because it is well-known. Because there are treatments. Because I would not be alone with an orphaned disease. Thanks to my quick diagnosis (Acute Kidney Injury caused by AAV) and treatment, I did not suffer the alternative fate of end stage renal failure where I would have been looking at a lifetime of dialysis or possible organ transplant. Instead, I was enrolled in a patient support program for Avacopan and started treatment within a week of leaving hospital. This enabled me to rapidly taper prednisone from 60 mg to 0 within six weeks. During those weeks, the side effects of the prednisone felt worse than the disease ever had. I couldn’t sleep more than 3 hours at night, and I spent my days drifting off or shoveling food into my mouth. Even coming off the prednisone in six weeks, I suffered withdrawal symptoms. Because of my short-term exposure, I hope to have been spared the longer-term side effects of loss of bone density, etc. I have been on Avacopan now for 11 months, along with Rituximab every 6 months. My liver function was monitored. I suffered no liver or other ill effects. My kidney function has improved to 45%. I no longer suffer debilitating fatigue. And I have hope that didn’t exist a year ago. I will discontinue Avacopan in another month, being the standard of treatment for my disease. But I know that it helped and I would like to know that it is there as an option should my disease relapse, and for all the other patients who will receive this life-altering news in the future. Notwithstanding the issues raised regarding the trial results, real-world cases such as mine show that the drug is effective and that risks can be mitigated with proper monitoring. Taking away this option means taking away hope for the 30 people per million* who are diagnosed annually with GPA or MPA and the countless others who are already living with the disease, who have to accept a lesser quality of life just to stay alive. * Approximate combined incidence of GPA and MPA in the United States per https://www.rarediseaseadvisor.com/disease-info-pages/anca-associated-vasculitis-epidemiology/.