The U.S. Food and Drug Administration (FDA) has accepted for review ChemoCentryx‘s application requesting the approval of avacopan to treat ANCA-associated vasculitis (AAV), the company announced in a press release.
The agency expects to announce its decision regarding approval on or before July 7, 2021. In its acceptance letter, the FDA also indicated it may hold an advisory committee meeting to discuss the company’s new drug application (NDA) per guidelines for new molecular entities.
Data from the ADVOCATE Phase 3 trial (NCT02994927) supported the company’s application, submitted in July. This trial assessed the safety and efficacy of avacopan, compared with the corticosteroid prednisone, at inducing and maintaining clinical remission in adults with AAV.
It enrolled 331 patients, who were randomly assigned to either avacopan or prednisone, both given alongside standard of care therapies (rituximab or cyclophosphamide, followed by azathioprine) for 52 weeks (about one year).
Top-line data from ADVOCATE showed avacopan was superior to prednisone at inducing clinical remission within the first 26 weeks of treatment, and also at ensuring patients remained symptom free for one year.
Avacopan’s use also led to greater improvements in patients’ health-related quality of life and kidney function. It was seen to have lower glucocorticoid toxicity, and to cause fewer serious side effects than prednisone.
Based on these data, ChemoCentryx’s partner Vifor Pharma is planning to file a similar request — in the form of a marketing authorization application — with the European Medicines Agency before the end of this year.
Avacopan (previously known as CCX168) is a first-in-class, oral small molecule inhibitor of the C5a protein, one of the most potent pro-inflammatory proteins of the complement system. By doing so, the experimental therapy is expected to ease the inflammation and blood vessel damage caused by AAV.
The investigational therapy was previously designated an orphan drug for the treatment of AAV by the FDA and the European Commission. The European Medicines Agency (EMA) also added avacopan to its priority medicines, or PRIME, regulatory initiative in 2016. PRIME aims to support the development and hasten the review of new therapies addressing unmet medical needs.
ChemoCentryx currently holds avacopan’s development and commercial rights in the U.S., while Vifor Pharma owns and retains the therapy’s marketing rights in nearly all other countries. In Japan, avacopan’s development and commercial rights have been sub-licensed to Kissei Pharmaceutical.