Swiss drug-maker Novartis AG (NVS) said Wednesday that its new biological drug Ilaris has been approved in the European Union to treat adults and children up to four years with cryopyrin-associated periodic syndrome, a rare auto-inflammatory disease.
Ilaris is already approved in the US and Switzerland, where it was granted priority review in view of the significant unmet medical need.
According to Novartis, studies proved that Ilaris produced rapid and sustained remission of symptoms in up to 97% of cryopyrin-associated periodic syndrome, or CAPS, patients, with most of them responding within hours of the first injection.
CAPS is a rare life-long auto-inflammatory disease with debilitating symptoms and few treatment options. Ilaris targets and normalizes the production of a protein within the immune system called interleukin-1 beta.
Ilaris is the only medicine approved in the EU for CAPS patients as young as four years old, and for patients with the most debilitating form of CAPS, neonatal-onset multisystem inflammatory disease. Ilaris is a fully human monoclonal antibody given by injection under the skin once every two months - the longest dosing interval of any available treatment.
The EU approval was granted under a common practice with orphan drugs, which reflects a need for additional data due to factors such as the rarity of the disease or lack of scientific knowledge. The situation is reviewed every year until the European Medicines Agency is able to grant a normal approval.
In addition to its orphan drug status for CAPS, Ilaris has been designated as an orphan drug for treating SJIA, the most severe form of arthritis in children, in the US, EU and Switzerland, and has fast-track status for SJIA in the US.
Ilaris was approved in Switzerland in July 2009 to treat all three forms of CAPS in adults and children over four years old, and in the US in June 2009 to treat two forms of CAPS, namely FCAS and MWS, while a study in NOMID patients is under way. Novartis said priority reviews are ongoing in other countries including Australia, Brazil and Canada.
Joe Jimenez, chief executive officer of the Novartis Pharmaceuticals Division, said, "Initially we studied Ilaris in a very rare disease with a well-understood genetic profile, and now that its efficacy has been proven, we are able to move ahead rapidly with development in other diseases characterized by the same inflammatory process."
NVS closed Tuesday's regular trading at $52.01 per share on the NYSE.
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