FDA approves avacopan for ANCA-associated vasculitis
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The FDA has approved avacopan as an adjunctive treatment for adults with severe active ANCA-associated vasculitis, specifically granulomatosis with polyangiitis and microscopic polyangiitis, alongside standard therapy.
“Today is a momentous day in the history of ChemoCentryx; the culmination of decades of effort aimed at offering new hope to patients with this and other debilitating and deadly diseases,” Thomas J. Schall, PhD, president and chief executive officer of ChemoCentryx, said in a company press release. “We look forward to making Tavneos available to clinicians and patients in the next few weeks.
“We thank the agency for their collaboration and consideration, and we are also immensely grateful to the pioneering scientists, clinicians and patients who believed in the promise of Tavneos and who have worked tirelessly to make it a reality, along with my dedicated and talented colleagues at ChemoCentryx,” he added.
According to the release, avacopan (Tavneos, ChemoCentryx) is the first oral complement C5a receptor inhibitor to be approved by the FDA. The drug is approved for the treatment of microscopic polyangiitis and granulomatosis with polyangiitis, the two main forms of ANCA-associated vasculitis, in Japan. The regulatory decision in Europe following the European Medicines Agency review is expected by the end of 2021, according to the release.
The FDA’s approval for patients with ANCA-associated vasculitis follows results from the phase 3 ADVOCATE trial, in which the drug met its primary endpoints of disease remission at 26 weeks and sustained remission at 52 weeks, as assessed by the Birmingham Vasculitis Activity Score, or BVAS.
Avacopan demonstrated the drug’s superiority to a prednisone-based standard of care in terms of sustained remission at 52 weeks. The most common adverse reactions — 5% of patients and higher in the avacopan group compared with prednisone — were nausea, headache, hypertension, diarrhea, vomiting, rash, fatigue, upper abdominal pain, dizziness, blood creatinine increase and paresthesia.
The full results of the trial, a global, randomized, double-blind, active-controlled, double-dummy study of 330 patients in 20 countries, were published in February in The New England Journal of Medicine.
“I am excited that our work has helped lead to the first-in-a-decade approval of a medicine for ANCA-associated vasculitis,” Peter A. Merkel, MD, MPH, the trial’s co-primary academic investigator and chief of rheumatology at the Perelman School of Medicine at the University of Pennsylvania, said in the release. “This is an important step forward in the treatment of this disease. Patients will now have access to a new class of medication that provides beneficial effects for the treatment of ANCA-associated vasculitis.”
Joyce Kullman, executive director of the Vasculitis Foundation, was also quoted in the release, lauding the approval as a victory for patients with ANCA-associated vasculitis, which she described as a “devastating disease” with many unmet needs.
“The vasculitis community is elated that Tavneos is now approved, bringing a much-needed new treatment option to patients living with this devastating disease,” Kullman said in the release. “There is a significant unmet need in the treatment of ANCA-associated vasculitis, with current therapies often leading to serious, even fatal, side effects and a diminished quality of life. We believe new therapies like Tavneos may offer a brighter future for these patients.”
Perspective
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Adam J. Brown, MD
The running joke of rheumatologists “just using steroids” may be coming to an end with the approval of avacopan, which ushers in a new era of targeted complement therapy.
Just a little over a decade ago, rheumatologists didn’t think complement was involved in the pathogenesis of ANCA vasculitis; patients’ renal histology did not demonstrate much immune complex deposition and the serum C3 and C4 are generally in the normal range in active disease. Now, not only do we know the alternative pathway is a major driver of ANCA vasculitis, but also, we can precisely target C5a and rapidly blunt the inflammatory response in ANCA vasculitis and hopefully become much less reliant on glucocorticoids. It’s a new era in treating ANCA vasculitis.
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