pharmafile | April 27, 2016 | News story | Business Services, Research and Development | Breakthrough Therapy Designation, Novartis, US FDA, periodic fever syndromes
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Novartis (VTX: NOVN) on Wednesday said its drug to treat three rare types of periodic fever syndromes has been granted breakthrough therapy designation by the US Food and Drug Administration (FDA).
The drug Ilaris (canakinumab) is targeted at treating the disease also known as Hereditary Periodic Fevers.
David Epstein, division head, Novartis Pharmaceuticals, said, these serious and debilitating syndromes have no or limited treatment options, adding that, Ilaris is a “promising medicine under review for these conditions.”
The FDA considers a treatment a breakthrough therapy if it is intended to treat a serious or life-threatening condition and preliminary evidence indicates it may be better than existing treatments.
The breakthrough therapy designations were granted based on the pivotal Phase III Cluster trial.
Ilaris was approved by the FDA in 2009 to treat two subtypes of a rare autoinflammatory disease called Cryopyrin-Associated Periodic Syndromes (CAPS): Muckle-Wells syndrome (MWS) and Familial Cold Autoinflammatory Syndrome (FCAS). In 2013, the FDA approved Ilaris for a rare, autoinflammatory form of juvenile idiopathic arthritis called Systemic Juvenile Idiopathic Arthritis (SJIA) in patients aged two and older.
Periodic fever syndromes are a group of autoinflammatory diseases that cause serious recurrent fevers that do not have an infectious cause. Most patients present with symptoms in infancy or childhood, but in some patients the condition only becomes apparent or diagnosed in adulthood.
There are no approved medicines available to treat Tumor Necrosis Factor-Receptor Associated Periodic Syndrome (TRAPS) and Hyperimmunoglobulin D Syndrome (HIDS)/Mevalonate Kinase Deficiency (MKD), and very limited options for patients with Familial Mediterranean Fever (FMF).
Anjali Shukla