- In March 2025, Novartis initiated a Phase III clinical trial for Ilaris (canakinumab) targeting Cryopyrin-Associated Periodic Syndromes (CAPS), Familial Mediterranean Fever (FMF), Tumor Necrosis Factor Receptor-Associated Periodic Syndrome (TRAPS), and Hyperimmunoglobulinemia D Syndrome (HIDS). This non-interventional study aims to evaluate the safety and effectiveness of Ilaris in both adult and pediatric patients across multiple centers in Korea
- In February 2025, The U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to Goflikicept (RPH-104), a novel heterodimeric hybrid protein capable of binding with high affinity to human IL-1β and IL-1α, for the treatment of CAPS. This designation is expected to facilitate the drug's development and expedite its review process
- In January 2025, The European Medicines Agency (EMA) included discussions on anti-TNF therapies for TRAPS in its Committee for Medicinal Products for Human Use (CHMP) meeting agenda. The focus was on evaluating the efficacy and safety of these therapies, reflecting the EMA's commitment to addressing rare autoinflammatory conditions
- In December 2024, A study published in the Journal of Rheumatology reported that tocilizumab effectively reduces flares of Hyperimmunoglobulinemia D Syndrome (HIDS) in pediatric patients. The study highlighted the drug's potential in managing this rare autoinflammatory disease, offering a promising treatment option for affected children
- In November 2024, A global patient registry for Periodic Fever Syndromes was established to collect comprehensive data on disease prevalence, genetic markers, and treatment outcomes. This initiative aims to enhance understanding of these rare conditions and support the development of targeted therapies
