Rare Daily Staff
ChemoCentryx said that following talks with the U.S. Food and Drug Administration it filed an amendment to its New Drug Application for avacopan, its experiment treatment for autoimmune disease anti-neutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis.
The amendment addresses points raised during the FDA Advisory Committee meeting on May 6, 2021. The FDA has indicated that the filing constitutes a major amendment to the NDA and will result in the setting of a new PDUFA goal date of October 7, 2021.
The NDA is primarily based on data from the phase 3 ADVOCATE trial of avacopan for the treatment of ANCA-associated vasculitis. On May 6, the FDA’s Arthritis Advisory Committee voted 9-9 on whether the efficacy data support approval of avacopan, 10-8 that the safety profile of avacopan is adequate to support approval, and 10-8 that the benefit-risk profile is adequate to support approval of avacopan at the proposed dose of 30 mg twice daily.
“We appreciate the opportunity to put additional data and information before the agency, information which we believe addresses many of the issues raised at the Advisory Committee meeting,” said Thomas Schall, president and CEO of ChemoCentryx.
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, combined with the administration of daily steroids for prolonged periods of time, which 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 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.
The Marketing Authorization Application (MAA) for avacopan in the treatment of ANCA-associated vasculitis was validated by the European Medicines Agency (EMA) in November 2020, and the Japanese New Drug Application was accepted for review by the Japanese Pharmaceuticals and Medical Device Agency in February 2021.
Photo: Thomas Schall, president and CEO of ChemoCentryx