- The FDA’s Arthritis Advisory Committee voted 10 for and 8 against the approval of ChemoCentryx Inc’s CCXI investigational drug avacopan as a treatment for adults with a rare and serious disease known as anti-neutrophil cytoplasmic autoantibody (ANCA)-vasculitis.
- The vote on whether the FDA should approve the drug was preceded by a split vote of 9 to 9 on whether the efficacy data support approval, and 10 to 8 that the safety profile of avacopan is adequate to support approval.
- In the third and final question, the Committee voted 10-8 that the benefit-risk profile is adequate to support approval of avacopan at the proposed dose of 30 mg twice daily.
- There was a contentious debate over whether the one relatively small Phase 3 trial in 330 patients comparing avacopan with prednisone was robust enough for full approval.
- The agency raised concerns about the statistical analyses of the data in the trial and what effect the use of glucocorticoids on top of cyclophosphamide or rituximab in both treatment arms had on the avacopan efficacy.
- Those voting against approval raised concerns about relying on the single-trial as evidence, insufficient safety data, and questions on whether the trial was statistically robust enough.
- Those voting in favor of approval noted that the trial could clear the bar to meet its primary endpoint.
- The agency also questioned if the other drugs used in the study may have contributed to the efficacy seen for avacopan.
- The company defended its study design, noting that it was primarily based on results from a single trial of about 200 patients when rituximab won approval in this indication. It was designed as a non-inferiority comparator trial over 26 weeks.
- The FDA is not required to follow the advice of its advisory Committee on ChemoCentryx’s avacopan, but in general, the agency usually does.
- See the FDA briefing documents here.
- Earlier this week, the FDA briefing document indicated “several areas of concerns” with avacopan. ChemoCentryx stock fell more than 48% in the last week.
- Price Action: CCXI shares closed at $27.49 on Thursday.
© 2024 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.
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