Canakinumab: a versatile tool for treating rare diseases!

Interleukin-1B (IL-1β) plays a very important role in inflammatory processes, such as acute gouty arthritis. (canakinumab), a recombinant human anti-human IL-1β monoclonal antibody, has been approved by the US FDA for cryopyrin protein-associated periodic syndrome (CAPS). It has proven efficacy. It has been used in clinical trials to treat juvenile idiopathic arthritis (JIA) and acute attacks of gout. It has also achieved satisfactory results. Because canakinumab can treat many rare diseases, the drug is called a generalist in the treatment of rare diseases.

Canakinumab is a human anti-human IL-1β monoclonal antibody that specifically binds to human IL-1β, blocking its binding to the IL-1 receptor and neutralizing its activity. Genetic variations in the NLRP-3 position lead to overactivity of inflammatory substances. Excessive release of active IL-1β triggers inflammation and is the cause of CAPS. In addition, IL-13 is involved in mediating various inflammatory processes. For example, during an acute attack of gout, uric acid crystals will activate the NLRP-3 inflammatory response, leading to excessive release of L-1, which is the cause of acute gout attacks.

In a trial, 166 patients with CAPS received canakinumab 150 mg or 2 mg·kg-1 (±40 kg) subcutaneously, once every 8 weeks, for 2 years. 85 of 109 patients without canakinumab treatment (79 patients within 8 days, 5 patients within 10-21 days) had complete disease response. Among 141 patients who could be evaluated for recurrence, the median treatment duration was 414 days (29-687 days), and 90% did not relapse. CRP and SAA levels dropped to normal levels within 8 days and remained normal during treatment. The trial also confirmed that canakinumab is safe and effective in the treatment of CAPS. Large doses are effective in treating young patients or severe CAPS, and can achieve complete disappearance of symptoms without a significant increase in adverse reactions.

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