Novartis announced that the US Food and Drug Administration (FDA) has granted three Breakthrough Therapy Designations for Ilaris® (canakinumab) to treat three rare types of Periodic Fever Syndromes, also known as Hereditary Periodic Fevers[1]. This means Novartis will work closely with the FDA to expedite the regulatory review of Ilaris for these conditions.
Periodic Fever Syndromes are a group of autoinflammatory diseases that cause disabling and recurrent fevers, which may be accompanied by joint pain and swelling, muscle pain and skin rashes, with complications that can be life-threatening[2]. Most patients present with symptoms in infancy or childhood[3]. The three conditions for which Ilaris is being reviewed are Tumor Necrosis Factor-Receptor Associated Periodic Syndrome (TRAPS) and Hyperimmunoglobulin D Syndrome (HIDS)/Mevalonate Kinase Deficiency (MKD), as well as Familial Mediterranean Fever (FMF) not adequately controlled with colchicine.
David Epstein, Division Head, Novartis Pharmaceuticals siad, This is an important day for patients, including many children, who are affected by these serious and debilitating syndromes that have no or limited treatment options. Ilaris is a promising medicine under review for these conditions, marking our commitment to making a significant difference to the lives of people with rare diseases.