Abstract
Two new orphan medicines, rilonacept [1, 2] (Regeneron®) and canakinumab [3, 4] (Ilaris®), are indicated for the treatment of cryopyrin-associated periodic syndromes (CAPS). CAPS occur in a group of rare, inherited, inflammatory diseases where patients have a mutation in the NLRP3 (nucleotide-binding domain, leucine-rich family, pyrin domain-containing-3) gene encoding for cryopyrin, which is a regulator in the acute immune reaction. Both familial cold auto-inflammatory syndrome and Muckle–Wells syndrome belong to the CAPS group. Additionally, canakinumab may be used to treat other CAPS such as neonatal-onset multisystem inflammatory disease and familial cold urticaria. The indications for both drugs might be extended in future. Currently, these two drugs are being evaluated in more common diseases including rheumatoid arthritis and systemic-onset juvenile idiopathic arthritis [5, 6].
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