U.S. Food and Drug Administration
White Oak Campus
10903 New Hampshire Ave
Silver Spring, MD  20993

 Attention: 

• Kyle Diamantas, J.D., Acting Commissioner of Food and Drugs, U.S. Food and Drug Administration
• Michael Davis, MD, PhD, Director of the U.S. Food and Drug Administration’s Center for Drug Evaluation and Research 
• Lowell M. Zeta, J.D., Acting Chief of Staff and Deputy Commissioner for Strategic Initiatives at the U.S. Food and Drug Administration 
• Nikolay P. Nikolov, MD, Director, Office of Immunology and Inflammation, Office of New Drugs, U.S. Food and Drug Administration
• Mary Tran Thanh Hai, MD, Director, Office of New Drugs at U.S. Food and Drug Administration

RE: Docket No. FDA-2026-N-1321 — Request for a Public Hearing Regarding the Proposed Withdrawal of NDA 214487 for TAVNEOS® (avacopan)

Dear Commissioner Diamantas, Dr. Davis, Mr. Zeta, Dr. Nikolov, and Dr. Hai:

The Vasculitis Foundation (VF), writing on behalf of our organization, the thousands of individuals living with antineutrophil cytoplasmic antibody-associated vasculitis (AAV), and the broader community that supports them, respectfully requests that the U.S. Food and Drug Administration (FDA) convene a public hearing regarding the proposed withdrawal of NDA 214487 for TAVNEOS® (avacopan) before the Agency reaches a final determination. 

A public hearing is necessary because the issues raised by the proposed withdrawal are sufficiently consequential to patients and clinicians, that they warrant a transparent opportunity for meaningful public discussion to:

1. Fully examine the benefit-risk profile of avacopan in the context of the risks, toxicities, and limitations of available alternative treatments;

2. Provide a meaningful opportunity for patients and caregivers living with Granulomatosis with Polyangiitis (GPA) and Microscopic Polyangiitis (MPA) to present their perspectives, real-world experiences, and risk tolerances directly to the Agency; 

3. Hear directly from clinicians and vasculitis specialists regarding real-world management of severe GPA and MPA in the United States, clinical practice dilemmas during this period of uncertainty, and the clinical consequences of losing an available therapeutic option in the United States;

4. Consider the full body of relevant clinical, safety, and postmarketing evidence concerning avacopan alongside different clinical monitoring practices and emerging international regulatory assessments;

5. Evaluate whether alternative risk-management approaches, including updated labeling, enhanced liver monitoring protocols, or restricted prescribing conditions, could address identified safety concerns while preserving access for appropriate patients; and

6. Ensure that the Agency’s final decision and underlying benefit-risk determination are communicated clearly and transparently to patients, caregivers, clinicians, and the public.

 As a matter of background, we believe it is important to stand true to the patient during this time and would like to highlight the real, patient-facing issues that are a part of this conversation. 

• The Consequences of Withdrawal are significant for patients – AAV is a chronic, potentially fatal autoimmune disease that causes severe inflammation of blood vessels, leading to irreversible damage to vital organs including the kidneys, lungs, upper respiratory tract, and nervous system. Renal involvement can progress rapidly to end-stage kidney disease, contributing substantially to morbidity and mortality.
  
  The existing standard of care relies heavily on nonspecific immunosuppressive therapies, including cyclophosphamide or rituximab, frequently combined with prolonged courses of glucocorticoids. While these therapies can be highly effective and remain essential to the care of many patients, they also carry serious and well-established risks, including severe infections, metabolic complications, osteoporosis, weight gain, adrenal suppression, and other substantial effects on health and quality of life.

  For patients with severe GPA and MPA, treatment decisions often require balancing the need to control a potentially life‑threatening disease against the substantial toxicity of immunosuppressive therapy. Because individuals respond differently, have distinct risk factors and comorbidities, and may experience adverse effects that limit suitability of specific agents, maintaining multiple treatment options is critically important for people living with a rare and serious disease.

  Accordingly, a determination concerning avacopan cannot be considered solely in terms of the risks associated with that individual product. The relevant question for patients is also what harms or risks they will face if that treatment option is no longer available. 

• Patient Perspectives Should Be Heard Directly – The FDA has recognized the importance of incorporating patient experience and patient preferences into regulatory decision-making, particularly when evaluating treatments for serious and rare diseases where patients face significant disease-related and treatment-related burdens.

 Individuals living with AAV and their caregivers can provide important information about the total patient experience including the practical consequences of treatment decisions that may not be fully captured by clinical trial endpoints alone. 

Patients can speak directly to questions such as:

• What does disease control mean in their daily lives?
• What treatment burdens are they willing to accept to reduce the risk of organ damage or relapse?
• What adverse effects have had the greatest impact on their lives?
• How do they weigh the risks of available treatments against the risks of uncontrolled disease?
• How important is it to have multiple therapeutic options when existing treatments carry substantial toxicity?

These perspectives should not replace scientific and clinical evidence. Rather, they should be an integral part of the complementary evidence for consideration. 

Further, we believe that a thorough and timely hearing is imperative to stabilize the current treatment ecosystem. The current level of ambiguity is affecting this community in very real ways. 

• Patients currently managed on avacopan are deeply anxious about the prospect of losing access to a treatment that has stabilized their disease, while newly diagnosed patients are left confused and distressed about whether avacopan remains a viable treatment option for induction or maintenance. 

• Healthcare providers are facing overwhelming uncertainty regarding how best to advise both current patients and candidates for therapy. In the absence of definitive clarity, some providers have preemptively stopped avacopan, leaving patients uncertain how to manage their disease safely and heightening the risk of disease flares or unnecessary exposure to high-dose glucocorticoids.

• Global regulatory bodies are issuing divergent assessments of the same clinical evidence, creating a fragmented international landscape that further amplifies confusion and erodes confidence among U.S. patients, caregivers, and clinicians.

The vasculitis community has a profound interest in ensuring that decisions affecting the treatment options available to people with severe GPA and MPA are made on the basis of a complete, transparent, and fully informed record. The proposed withdrawal of an approved therapy raises questions that extend well beyond the safety profile of a single drug and directly implicate the treatment landscape for a serious and potentially fatal disease.

For these reasons, we believe that a public hearing would meaningfully serve the interests of patients, clinicians, researchers, and the Agency. Such a forum would allow the FDA to hear directly from individuals living with or touched by AAV, experts, and other stakeholders who can speak to the patient experience, clinical realities, unmet needs, and potential approaches for managing identified risks while preserving access where appropriate. This process would help ensure that the Agency’s final determination reflects the full range of relevant evidence, perspectives, and lived experience. 

We welcome the opportunity to assist the FDA in identifying and facilitating participation by stakeholders of the AAV community who can provide relevant perspectives – most importantly, patients and their caregivers.

Thank you for your consideration of this request and for your continued attention to the needs of people living with vasculitis. We respectfully request a written response by September 30, 2026 so that we may appropriately inform the community and plan next steps. 

Respectfully submitted, 
Joyce A. Kullman
Executive Director, Vasculitis Foundation

Related submission:
June 9, 2026 — Comment
Tracking Number: mq6-sxee-pvb9 

About the Vasculitis Foundation

The VF is the leading patient-focused nonprofit organization dedicated to improving the lives of people affected by vasculitis. Established in 1986, VF works to advance research, improve access to care, and support the more than 500,000 Americans living with vasculitis, including people living with antineutrophil cytoplasmic antibody-associated vasculitis (AAV), specifically granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA).

July 31, 2026
The Vasculitis Foundation continues to track updates from the U.S. Food and Drug Administration (FDA) regarding Tavneos (avacopan), including safety communications and ongoing regulatory discussions with Amgen.

On July 23, 2026, Amgen submitted their request for a public hearing to the U.S. Food and Drug Administration (FDA) in support of TAVNEOS, with the goal of preserving access to the medicine in the United States.

Amgen’s FDA full submission is available here and includes data from clinical trials, post-marketing real-world evidence, and post-marketing safety data. 

• Duke Clinical Research Institute Independent Re-Adjudication
   In 2026, Amgen sponsored an independent, fully blinded re-adjudication, conducted by Duke Clinical Research Institute, evaluating the primary outcomes of ADVOCATE to address the FDA-cited concerns around the process followed by ChemoCentryx in 2019, prior to our acquisition of the company. 
• Real-World Effectiveness and Safety of Avacopan in ANCA-Associated Vasculitis: A Systematic Literature Review and Meta-analysis
  In this systematic literature review and meta-analysis of 71 real-world studies, TAVNEOS was associated with high rates of disease control, including 87% remission at 6 months (23 studies; n=807), 93% remission at 12 months (12 studies; n=362), 86% sustained remission at 12 months (8 studies; n=283), and a 7% relapse rate at 12 months (12 studies; n=353). 
• Effectiveness and Safety of TAVNEOS in ANCA-Associated Vasculitis: 
  Real-world evidence is important for evaluating the effectiveness of medicines when used in day-to-day practice.
• A Post-Marketing Evaluation of TAVNEOS Safety with a Focus on Hepatic Adverse Events: 
  In this retrospective, descriptive analysis of the Amgen Global Safety Database, serious hepatic adverse events were reported at a rate of approximately 31 events per 1,000 patient-years.

“We recognize that the Tavneos issue continues to be of great concern to our patients, families, and healthcare providers,” said Joyce Kullman, Executive Director of the  Vasculitis Foundation. “Our priority is to ensure the community has access to accurate, timely information while encouraging patients to make informed decisions in partnership with their healthcare team.”

The Vasculitis Foundation does not endorse any specific medications and does not provide medical advice. Treatment decisions are highly individualized, and patients should not  discontinue  or change their treatment without first consulting their healthcare provider.

For the most current information, patients and clinicians are encouraged to review official updates from the FDA and Amgen: