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Successful treatment with avacopan (CCX168) in a pediatric patient with C3 glomerulonephritis

Federica Zotta1, Francesca Diomedi-Camassei2, Antonio Gargiulo3

  • 1Division of Nephrology and Dialysis, Department of Pediatric Subspecialties, Bambino Gesù Pediatric Hospital IRCCS, Piazza S. Onofrio 4, 00165, Rome, Italy. federica.zotta@opbg.net.

Pediatric Nephrology (Berlin, Germany)
|June 12, 2023
PubMed

Insights

Avacopan, a C5a receptor antagonist, demonstrated safety and tolerability in a pediatric patient with C3 glomerulonephritis (C3GN). The treatment allowed for discontinuation of mycophenolate mofetil while maintaining disease remission.

Area of Science:

  • Nephrology
  • Complement System Biology
  • Pharmacology

Background:

  • C3 glomerulonephritis (C3GN) is a rare kidney disease driven by complement alternative pathway dysregulation, lacking approved treatments.
  • Current therapies, including immunosuppressants and biologics, offer limited success for C3 glomerulopathy (C3G).
  • Avacopan is an oral C5a receptor antagonist targeting a key inflammatory mediator in complement-mediated diseases.

Observation:

  • A pediatric patient with biopsy-proven C3GN was treated with avacopan within the ACCOLADE study framework.
  • The patient initially received a placebo, followed by open-label avacopan, and later continued treatment via an expanded access program.
  • Treatment involved oral avacopan administration twice daily.

Findings:

  • Avacopan treatment was found to be safe and well-tolerated in this pediatric C3GN case.
  • The patient achieved and maintained remission of C3GN while on avacopan therapy.
  • Concomitant mycophenolate mofetil (MMF) was successfully discontinued during avacopan treatment.

Implications:

  • Avacopan shows promise as a safe and effective therapeutic option for pediatric C3GN.
  • Targeting the C5a receptor with avacopan may enable reduction of other immunosuppressive agents.
  • Further research into complement inhibitors like avacopan is crucial for advancing C3G treatment.
Abstract

Keywords:
Alternative pathway of complementAvacopanC3 glomerulopathyC5a complement receptor inhibitorChildMembranoproliferative glomerulonephritis

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