Chapter 8: Discovery and Development of Ilaris® for the Treatment of Cryopyrin-Associated Periodic Syndromes Check Access

Canakinumab is a human anti-human IL-1β monoclonal antibody which is therapeutically applied to neutralise the bioactivity of IL-1β produced in acute and chronic inflammatory conditions. IL-1β is part of the innate immune response to bacterial pathogens, but has a prominent role in a number of biological responses and processes in mammals, such as the fever response, insulin metabolism, pain sensitisation, endothelial activation and orchestration of the neutrophil response, reproduction, tissue remodelling, and the induction of cytokines and chemokines. Canakinumab is marketed under the trade name ILARIS® and is indicated for the treatment of cryopyrin associated periodic syndrome (CAPS). CAPS is an extremely rare monogenetic disease with an incidence of about 1 per million and is caused by a functional defect in the NLRP3 protein, a component of the inflammasome, which controls the release of mature and active IL-1β from phagocytes. Chronic overproduction of IL-1β in these patients leads to high spiking fever attacks, urticaria, fatigue, arthralgia, and in the long term to amyloidosis and sensoneuronal hearing loss. Canakinumab is particularly well suited for the treatment of CAPS, as it specifically targets the critical effector molecule, IL-1β, responsible for the clinical pathology of this disease. Treatment with canakinumab provides a rapid and sustained remission of clinical symptoms and biochemical markers of inflammation in CAPS patients, while it is generally well tolerated.