Shares of ChemoCentryx, Inc., (NASDAQ:CCXI) plummeted by 61% after announcing the outcome of the U.S. Food and Drug Administration (“FDA”) Arthritis Advisory Committee (“Committee”) on avacopan for the treatment of Antineutrophil Cytoplasmic Autoantibody (ANCA)-associated vasculitis (or AAV). In the final part of the public meeting, the Committee voted on three questions presented by the FDA:
- The Committee vote split 9-9 on the first question, whether the efficacy data support approval of avacopan for the treatment of adult patients with AAV (granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA)).
- On the second question, the Committee voted 10-8 that the safety profile of avacopan is adequate to support approval of avacopan for the treatment of adult patients with AAV (GPA and MPA).
- In the third and final question, the Committee voted 10-8 that the benefit-risk profile is adequate to support approval of avacopan at the proposed dose of 30 mg twice daily for the treatment of adult patients with AAV (GPA and MPA).
“We are grateful to the Committee for their careful deliberations and look forward to working with the FDA as its review of our application continues,” said Thomas J. Schall, Ph.D., President and Chief Executive Officer of ChemoCentryx. “Discussion from patients and clinicians during the public forum portion of the meeting underscored the need for new treatment options.”
“We hope that the FDA will take into account the dire situation faced by patients living with this debilitating disease,” said Joyce Kullman, Executive Director, Vasculitis Foundation. “ANCA vasculitis flares can lead to kidney failure and death; current therapy all too often causes serious, even fatal, side effects; and patients suffer a lower quality of life due to the disease and to the way it is treated.”
FDA Advisory Committees provide the FDA with independent opinions and non-binding recommendations from outside medical experts. While the FDA will consider the opinions expressed and recommendations made by the Advisory Committee, the FDA will make a decision regarding whether to approve the NDA for avacopan in ANCA-associated vasculitis following completion of its review process.
About ADVOCATE and ANCA-Associated Vasculitis
The ADVOCATE trial of avacopan was a global, randomized, double-blind, active-controlled, double-dummy Phase III trial of 331 patients with ANCA-associated vasculitis in 20 countries. Eligible study subjects were randomized to receive avacopan plus either rituximab or cyclophosphamide (followed by azathioprine/mycophenolate) or prednisone plus either rituximab or cyclophosphamide (followed by azathioprine/mycophenolate).
ANCA-associated vasculitis is a systemic disease in which over-activation of the complement pathway further activates neutrophils, leading to inflammation and destruction of small blood vessels. This results in organ damage and failure, with the kidney as the major target, and is fatal if not treated. Currently, treatment for ANCA-associated vasculitis consists of courses of non-specific immuno-suppressants (cyclophosphamide or rituximab), combined with the administration of daily glucocorticoids (steroids) for prolonged periods of time, which can be associated with significant clinical risk including death from infection.
