Rare Daily Staff
Share in ChemoCentryx fell more than 60 percent in early trading following a series of divided votes by the U.S. Food and Drug Administration’s Arthritis Advisory Committee on the approvability of avacopan for the treatment of Antineutrophil Cytoplasmic Autoantibody (ANCA)-associated vasculitis.
It’s been a tumultuous week for Chemocentryx shares, which traded at a high of $49.04 on May 3 and then tumbled after the U.S. Food and Drug Administration released a document ahead of the advisory committee meeting that questioned the company’s rationale for the benefit of avacopan as a treatment for anti-neutrophil cytoplasmic antibody-associated vasculitis. The shares are now trading around $10.
The committee vote split 9-9 on the question of whether the efficacy data support approval of avacopan for the treatment of adult patients with AAV (granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA).
The committee voted 10-8 on a second question as to whether 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).
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 decide regarding whether to approve the NDA for avacopan in ANCA-associated vasculitis following completion of its review process.
Anti-neutrophil cytoplasmic antibody-associated vasculitis (ANCA-associated vasculitis or ANCA vasculitis) refers to a group of rare systemic diseases in which over-activation of the complement pathway over-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. Current treatment for ANCA vasculitis consists of courses of non-specific immuno-suppressants (cyclophosphamide or rituximab), combined with high-dose long-term corticosteroid therapy that can be associated with significant clinical risk including death from infection.
Avacopan is a first-in-class, orally-administered, small molecule that employs a novel, highly targeted mode of action in the treatment of ANCA-associated vasculitis and other complement-driven autoimmune and inflammatory diseases. By precisely blocking the receptor (the C5aR) for the pro-inflammatory complement system fragment known as C5a on destructive inflammatory cells such as blood neutrophils, avacopan arrests the ability of those cells to do damage in response to C5a activation, which is known to be the driver of ANCA-associated vasculitis.
“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.”