Comment from Anna Castella

Thank you for the opportunity to provide feedback on the issue of TAVNEOS (avacopan) medication approval to the FDA’s Center for Drug Evaluation and Research. I have lived with what is now called GPA for more than 30 years. Most of my adult life, since age 23, has been spent trying new medications to ward off the worst symptoms of this disease and to keep it from being active as well as from moving into new areas of my body. In all of these years there has been no drug treatment that is specifically for GPA. Instead, GPA patients have had to resort to general vasculitis or auto-immune therapies, alone or in various combinations. Different patients have had success with different medications, so it's always been trial and error. Sometimes the doctors have tried throwing almost anything our way to stop the disease. We have to treat both the damage that the disease has done to parts of our body, as well as the GPA itself. Because it’s a type of vasculitis, any kind of organ damage is up for grabs to this disease at any time. I've had years of both medicated and non-medicated remissions as well as years of difficult flare-ups. I have always counted myself among the lucky ones, however, because for me this disease has mostly stayed confined to my upper respiratory area and has responded to medication, even if it takes a while to figure out what treatment is best and after organ damage has already been done and is unable to be reversed. A few years ago, my situation changed and GPA moved into new areas of my body and no longer responded to the same medications it had in the past. I was able to get partial and temporary relief from more toxic treatments that I took both orally and via infusions. I’m now at a point where even that is not enough to stop my GPA and I’m about to start TAVNEOS (avacopan) along with existing drug therapies. I wish I could properly express how excited I was to get approval for this medication and to even have an option available to me that is specifically for my named disease. TAVNEOS (avacopan) isn’t a generalized medication or one for just my symptoms or just for the umbrella of vasculitis. It is specific to exactly what I have, and that has never existed before now. I understand concerns about liver and bile duct problems with this medication. As I see it, I have already been on such toxic medications and for so long that I’ve already accepted many risks and experienced a variety of side effects. There may even be things that happen down the road to me due to my past medication use that no one can even predict yet. I have always understood and accepted this. After all, the option if I didn’t could mean more organ damage, in both number of organs affected and in how much they are affected, or this disease could be fatal at any time. Anyone living with this chronic illness knows this. Since patients like me are already accepting risks, some we may not even know about, the risks associated with TAVNEOS (avacopan) really seem no different to me. Patients need to have the option of this drug’s benefits available to us despite the known risks. As I have reviewed comments across various platforms from the GPA patient community, I see nothing but positivity, and in fact excitement. For both having a drug that is for our exact disease, and for the positive benefits that it has been giving patients who have been on it so far. I always do a lot of research myself about GPA and any therapies I consider with my doctors, because I believe that within the healthcare system we have, that is essential for all patients of any kind. So while I can’t offer direct experience with TAVNEOS (avacopan) yet, I hope my perspective as a long-term GPA patient will be considered. I am ready and willing to start taking this drug despite its risks. I’ve taken risks with other medications in the past and the benefits still came. I strongly encourage the FDA to let doctors and patients make up their own minds about whether they want to and will take this drug, and to keep TAVNEOS (avacopan) available.