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Canakinumab investigated for treating familial Mediterranean fever

Ruby Haviv1,2, Philip J Hashkes1,3

  • 1a Pediatric Rheumatology Unit , Shaare Zedek Medical Center , Jerusalem , Israel.

Expert Opinion on Biological Therapy
|September 8, 2016
PubMed
Abstract

Insights

Canakinumab offers an effective treatment for Familial Mediterranean Fever (FMF) resistant to colchicine. This interleukin-1 beta (IL-1β) inhibitor shows a favorable safety profile in clinical trials.

Area of Science:

  • Rheumatology
  • Immunology
  • Genetics

Background:

  • Familial Mediterranean Fever (FMF) is the most prevalent hereditary autoinflammatory disease.
  • Colchicine is the standard treatment, but approximately 40-50% of patients exhibit partial or non-response.
  • Understanding pyrin's role in IL-1β regulation has paved the way for anti-IL-1 therapies.

Purpose of the Study:

  • To review the efficacy and safety of anti-interleukin-1 (IL-1) agents, specifically canakinumab, for colchicine-resistant Familial Mediterranean Fever (FMF).

Main Methods:

  • A comprehensive literature search was conducted on PubMed/Medline/Scopus (since 2001) and rheumatologic conference proceedings (since 2011).
  • The search focused on anti-IL-1 treatments for FMF, with a particular emphasis on canakinumab, a humanized IL-1β antibody.
  • Inclusion of unpublished studies from conference proceedings aimed to provide a complete overview.

Main Results:

  • Numerous reports since 2007 demonstrated successful outcomes with anti-IL-1 agents.
  • Canakinumab treatment showed significant success in case reports (2011) and Phase II trials (2014-2015).
  • A Phase III randomized, placebo-controlled trial confirmed significantly superior efficacy of canakinumab over placebo in colchicine-resistant FMF patients.

Conclusions:

  • Canakinumab demonstrated a favorable safety profile comparable to its use in other indications.
  • The drug proved highly effective in achieving primary and secondary outcome measures in a rigorous trial.
  • Canakinumab represents a promising and effective alternative for most patients with colchicine-resistant FMF.
Keywords:
Amyloidosisanakinracanakinumabcolchicinefamilial Mediterranean feverinterleukin-1pyrinrilonacepttreatment

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