Periodic Fever Syndromes are a group of diseases that cause serious recurrent fever and pathogenic inflammation through non-infectious activation of the immune system. Most patients present with symptoms in infancy or childhood, but in some patients the condition only becomes apparent or diagnosed in adulthood
Novartis announced that the Committee for Medicinal Products for Human Use (CHMP) has recommended approval of Ilaris® (canakinumab) in Europe to treat three rare and distinct Periodic Fever Syndromes.
If approved, Ilaris will be the first and only approved biologic treatment in Europe for Tumor Necrosis Factor-Receptor Associated Periodic Syndrome (TRAPS), Hyperimmunoglobulin D Syndrome (HIDS)/Mevalonate Kinase Deficiency (MKD) and Familial Mediterranean Fever (FMF).
“Few treatments exist for people with these potentially life-threatening conditions. Today’s positive recommendation is a great stride forward for patients who urgently need new treatment options,” said Paul Hudson, CEO, Novartis Pharmaceuticals. “Novartis is committed to paving the way forward for rare diseases especially through treatments like Ilaris which has the potential to raise the standard of care.”
The CHMP positive opinion, which also recommends the use of a new formulation which is a 150 mg/ml solution for injection, is based on results from the pivotal Phase III CLUSTER study. Results showed rapid (at Day 15) and sustained disease control with Ilaris compared to placebo through 16 weeks, in patients with either TRAPS, HIDS/MKD or FMF. Data show Ilaris to be a well-tolerated and effective treatment for these three rare conditions.
All three conditions are part of a group of rare autoinflammatory diseases called Periodic Fever Syndromes, which are also referred to as Hereditary Periodic Fevers (HPF). These can cause disabling and persistent fevers which may be accompanied by joint pain, swelling, muscle pain and skin rashes with complications that can be life-threatening. The most common syndrome is FMF, which mainly affects people of Eastern Mediterranean ancestry. It affects 1 in 250 to 1 in 1,000 individuals in these populations, many of whom are children.
In August 2016, the European Commission approved Ilaris for a license extension to treat patients with Adult-Onset Still’s Disease (AOSD), a rare type of inflammatory arthritis. The US Food and Drug Administration (FDA) previously granted three simultaneous approvals of Ilaris for the treatment of TRAPS, HIDS/MKD and FMF in September 2016.