Study Shows Avacopan Effective for Most Serious Vasculitis Cases

WASHINGTON, Nov. 18, 2024 /PRNewswire/ -- New research to be presented by an investigator from Hospital for Special Surgery (HSS) shows avacopan is effective in achieving disease control with less steroid exposure in patients with ear, nose and throat (ENT) manifestations of ANCA associated vasculitis -

Robert F. Spiera, MD, Director of the Scleroderma, Vasculitis, and Myositis Center at Hospital for Special Surgery (HSS), helped lead this latest analysis which was presented at the ACR Convergence 2024, the annual meeting of the American College of Rheumatology. The study built on the pivotal ADVOCATE trial, focusing on patients with the most severe manifestations of vasculitis in the ENT region, which has historically been harder to treat.

Avacopan (Tavneos, Amgen) was approved by the US Food and Drug Administration in 2021 for the treatment of severe active anti-neutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis, a rare autoimmune disorder that can cause potentially life-threatening inflammation by restricting blood flow to vital organs. 

Once considered a fatal condition, ANCA associated vasculitis is now treatable, especially if identified early and treated with appropriate immunsosuppressive medications. Avacopan is a targeted therapy that offers a safer and more effective alternative to prednisone which had traditionally been a mainstay of remission induction therapy in these disorders but carries with it high risks such as infections, weight gain, diabetes, and bone thinning.

While the original ADVOCATE trial established the avacopan as an improved treatment option, the new analysis zeroes in on a group of patients whose disease has historically been more difficult to treat – those with ENT involvement. These patients often experience debilitating symptoms such as pain, hearing loss, and chronic inflammation, which severely impact quality of life.

"We know patients with this disease often achieve remission in their kidneys and lungs, but ENT disease frequently becomes a significant factor impacting their quality of life," Dr. Spiera said. "This analysis provides crucial insights, showing that avacopan not only brings faster relief but sustains improvement over time in patients with those manifestations."

The new analysis focused on a subgroup of 144 patients from the ADVOCATE trial, all of whom had severe ENT symptoms. Findings revealed that patients treated with avacopan showed significant improvements as early as week 4, with results continuing to favor avacopan over prednisone at week 52. Remarkably, 71% of these patients reached remission at 26 weeks, closely mirroring the remission rates in the broader trial.

Beyond clinical improvement, patients taking avacopan reported higher quality of life scores compared to the group taking prednisone. "Avacopan offers these patients a real opportunity to live with fewer limitations," noted Dr. Spiera.

Equally important, the study showed that avacopan's safety profile was comparable to that of prednisone, with similar rates of adverse events such as infections.

"These encouraging results underscore the importance of considering avacopan, for patients with microscopic polyangiitis and granulomatosis with polyangiitis with active ENT symptoms," Dr. Spiera said.

About HSS

HSS is the world's leading academic medical center focused on musculoskeletal health. At its core is Hospital for Special Surgery, nationally ranked No. 1 in orthopedics (for the 15th consecutive year), No. 3 in rheumatology by U.S. News & World Report (2024-2025), and the best pediatric orthopedic hospital in NY, NJ and CT by U.S. News & World Report "Best Children's Hospitals" list (2024-2025). In a survey of medical professionals in more than 20 countries by Newsweek, HSS is ranked world #1 in orthopedics for a fifth consecutive year (2025). Founded in 1863, the Hospital has the lowest readmission rates in the nation for orthopedics, and among the lowest infection and complication rates. HSS was the first in New York State to receive Magnet Recognition for Excellence in Nursing Service from the American Nurses Credentialing Center five consecutive times. An affiliate of Weill Cornell Medical College, HSS has a main campus in New York City and facilities in New Jersey, Connecticut and in the Long Island and Westchester County regions of New York State, as well as in Florida. In addition to patient care, HSS leads the field in research, innovation and education. The HSS Research Institute comprises 20 laboratories and 300 staff members focused on leading the advancement of musculoskeletal health through prevention of degeneration, tissue repair and tissue regeneration. In addition, more than 200 HSS clinical investigators are working to improve patient outcomes through better ways to prevent, diagnose, and treat orthopedic, rheumatic and musculoskeletal diseases. The HSS Innovation Institute works to realize the potential of new drugs, therapeutics and devices. The HSS Education Institute is a trusted leader in advancing musculoskeletal knowledge and research for physicians, nurses, allied health professionals, academic trainees, and consumers in more than 165 countries. The institution is collaborating with medical centers and other organizations to advance the quality and value of musculoskeletal care and to make world-class HSS care more widely accessible nationally and internationally. www.hss.edu.

SOURCE Hospital for Special Surgery