Novartis announced that the Food and Drug Administration (FDA) have granted Ilaris (canakinumab) three Breakthrough Therapy Designations, to treat three types of rare Periodic Fever Syndromes (also known as Hereditary Periodic Fevers).
The three conditions the Breakthrough Designation applies to are, Tumor Necrosis Factor-Receptor Associated Periodic Syndrome (TRAPS), Hyperimmunoglobulin D Syndrome (HIDS)/Mevalonate Kinase Deficiency (MKD), and Familial Mediterranean Fever (FMF) in patients who are not adequately controlled with colchicine.
The designation is supported by the Phase 3 trial: Canakinumab Pivotal Umbrella Study in Three Hereditary Periodic Fevers. There are currently no approved medicines available to treat TRAPS or HIDS/MKD, and just one treatment option exists for FMF patients: colchicine.
Ilaris, a selective, high-affinity and human monoclonal antibody that inhibits Interleukin-1 (IL-1) beta, is approved for the treatment of Cryopyrin-Associated Periodic Syndromes (CAPS), in adults and children ≥4yrs of age including: Familial Cold Autoinflammatory Syndrome (FCAS); Muckle-Wells Syndrome (MWS), as well as systemic juvenile idiopathic arthritis (SJIA) in patients ≥2yrs of age.
The FDA will now work closely with Novartis to expedite the review of Ilaris for these new indications.