News: Orphan drug Avacopan, approved by FDA to treat ANCA-related diseases

Introduction Anti-neutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis is a group of autoimmune diseases involving small and medium blood vessels with positive anti-neutrophil cytoplasmic antibodies
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The incidence of ANCA-related vasculitis in China is about 20 cases per million population, and kidney involvement is the most common condition
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At present, worldwide, there are limited studies on renal treatment options for ANCA-related vasculitis, and the mortality of patients is relatively high
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 On October 8, 2021, the U.
S.
Food and Drug Administration (FDA) approved Avacopan to treat ANCA-related diseases (ANCA vasculitis and nephritis)
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This is the first complement 5a receptor antagonist approved by the FDA, which can specifically treat ANCA vasculitis and nephritis
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The ADVOCATE trial proved that after 52 weeks of treatment, the patient's condition had sustained remission
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The study showed that compared with the glucocorticoid treatment group, it can increase the estimated glomerular filtration rate (eGFR) of patients
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The specific details of the trial and expert comments can be seen in the ERA-EDTA conference report: the new drug is effective against rare nephropathy, and patients may be able to get rid of the side effects of glucocorticoids｜2021EDTA
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In general, the experts praised Avacopan
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Professor of Rheumatology at Columbia University, Peter A.
Merkel believes that this is a very important clinical application that can provide more treatment options for patients with ANCA vasculitis and nephritis
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Joyce Kullman, executive director of the Vasculitis Foundation, believes that Avacopan can reduce the mortality of ANCA patients and contribute to the prognosis of patients
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According to Avacopan’s production plant, ChemoCentryx’s public statement, the use of Avacopan should pay attention to the following: 1.
Avacopan does not exclude the use of glucocorticoids, which can be combined with glucocorticoids for treatment; 2.
Before and during treatment with Avacopan, Liver tests are performed every 4 weeks
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Avacopan is not suitable for patients with active, untreated and/or uncontrolled chronic liver disease (such as HBV, uncontrolled autoimmune liver disease) and cirrhosis
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Avacopan should be used with caution in patients with liver disease, and the risk/benefit ratio should be investigated; 3.
Avacopan is not suitable for patients with active and severe infections (including local infections)
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For patients with chronic infections or people at increased risk of infection (such as visiting high-risk areas), be cautious before using Avacopan
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The most common infections of Avacopan are pneumonia and urinary tract infection; 4.
Common adverse reactions: nausea, headache, high blood pressure, diarrhea, vomiting, rash, fatigue, upper abdominal pain, dizziness, increased blood creatinine and abnormal perception
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5.
It is necessary to avoid the combination of Avacopan and strong and moderate CYP3A4 hepatic enzyme inducers
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When combined with CYP3A4 hepatic enzyme inhibitors, the dose of Avacopan should be reduced to 30 mg/d
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References: 1.
ChemoCentryx.
ChemoCentryx Announces FDA Approval of TAVNEOS™ (avacopan) in ANCA-AssociatedVasculitis.
ChemoCentryx.
October 08, 2021.