Comment from Anonymous

I am commenting from the perspective of a person with Osteoporosis who was recently treated for severe active ANCA associated vasculitis utilizing Rituximab, prednisone, and Tavneos as an adjunct. In the decision document the agency states that “there is no demonstrated benefit to balance the risks associated with Tavneos”. That statement seems to overlook significant benefits related to a reduction in prednisone dosing when Tavneos is used, especially for patients with osteoporosis. Patients with osteoporosis or diabetes benefit in both long term health and life expectancy from lower total and shorter-term prednisone dosing that occurs when Tavneos is used as an adjunct. To give you an idea of the reduction potential I quantified the difference in prednisone dosing based on my own treatment experiences in the paragraph below. My vasculitis diagnosis is Granulomatosis with Polyangiitis (GPA). In 2009, prior to Tavneos approval, I was successfully treated under an older protocol utilizing only Cyclophosphamide and prednisone. My recent 2024 treatment utilizing Rituximab, Tavneos, and prednisone used 71% less prednisone taken for only 4.6 months (2255 mg total), compared to prednisone taken for 35.6 months (7717 mg total) during my 2009 treatment prior to Tavneos approval. Both treatment methods were addressing severe active disease. Both treatment methods seemed to achieve medically induced remission in about the same time. In my recent treatment which included Tavneos, high dose prednisone was limited and quickly tapered during first month, the remaining 3.6 months dosing was low at 5 mg/day. In 2009 (without Tavneos) during induction and maintenance, prednisone dosing was >5 mg/day for 420 days. I am a 67 year old male and my osteoporosis is presumably related to past prednisone use in treating my ANCA associated Vasculitis autoimmune disease. The femoral neck in my hips show the highest bone density loss. Hip fractures are correlated with a significant reduction in life expectancy, particularly for older adults. I balance the risks of Tavneos liver toxicity, which can and is monitored monthly, against a hip fracture or development of diabetes from prednisone use. Like most vasculitis patients who have had flare-ups, its likely I will flare again and need treatment again in the future. When that time comes I will need options like Tavneos that limit prednisone, while still controlling inflammation damage, until achieving and maintaining remission. I recommend that the agency allow Avacopan/Tavneos as an adjunct in treatment of ANCA Associated Vasculitis under an emergency use or other special authorization for patients with osteoporosis, diabetes, and other serious pre-existing medical conditions exacerbated by prednisone use. Regarding the safety of Tavneos from a liver toxicity perspective I was pretested prior to authorization and tested monthly during treatment for liver function indicators ALT (SGPT), Alkaline Phosphatase, AST (SGOT), and Bilirubin. The manufacturer of Tavneos provides action levels for each and what actions should be taken if results are outside of normal ranges. I feel comfortable with this approach. I recommend that the frequency and types of required tests for liver health be re-evaluated to ensure they are adequate warning systems during use of Tavneos.