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.
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.
Previous treatments for these rare conditions consisted of oral anti-inflammatory drugs, such as corticosteroids, which were used only to help manage the symptoms. While other medicines, such as non-steroidal anti-inflammatory drugs, have also been used to help reduce symptoms, they do not prevent or change the overall course of a flare.
Ilaris is a selective, high-affinity, human monoclonal antibody that inhibits Interleukin-1 (IL-1) beta, which is an important part of the body's immune system defences. Excessive production of IL-1 beta plays a prominent role in certain inflammatory diseases. Ilaris works by blocking the action of IL-1 beta for a sustained period of time, therefore inhibiting inflammation that is caused by its over-production. Novartis has reformulated Ilaris from a powder that needed to be reconstituted into a solution prior to use to a solution for injection (ready to use) which will ease administration of the product.
Ilaris is currently approved and marketed for the treatment of Systemic Juvenile Idiopathic Arthritis (SJIA) in the US and EU and for the treatment of AOSD and the symptomatic treatment of refractory acute gouty arthritis in the EU. Ilaris is also approved in more than 70 countries, including in the EU, Switzerland, US, Canada, and Japan for the treatment of Cryopyrin-Associated Periodic Syndromes (CAPS): rare, lifelong, genetic disorders with debilitating symptoms. In the EU, Ilaris is approved for following subtypes of CAPS: Muckle-Wells syndrome (MWS), neonatal-onset multisystem inflammatory disease (NOMID)/chronic infantile neurological, cutaneous, articular syndrome (CINCA), severe forms of familial cold autoinflammatory syndrome (FCAS)/familial cold urticaria (FCU) presenting with signs and symptoms beyond cold-induced urticarial skin rash. The approved indications may vary depending upon the individual country.