Ilaris, a new biological drug manufactured by Novartis has been approved in the EU to treat cryopyrin-associated periodic syndrome (CAPS), a rare auto-inflammatory disease.
The accelerated EU decision follows recent approvals in the US and Switzerland, where the drug was granted priority review status as an important new treatment for the disease, which has debilitating symptoms and few treatment options in children and adults.
Ilaris is a fully human monoclonal antibody injected under the skin once every two months.
Novartis Pharmaceuticals Division CEO Joe Jimenez said that Ilaris represents a significant therapeutic advance for patients with this debilitating and sometimes fatal disease.
"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 characterised by the same inflammatory process," Jimenez said.
EU approval was supported by trial data, which showed rapid and sustained remission of symptoms in up to 97% of CAPS patients, with the majority responding within hours of the first injection.
If left untreated, CAPS can have serious consequences such as deafness, bone deformities, erosive joint destruction and central nervous system damage leading to loss of vision. A quarter of patients also suffer from amyloidosis, a protein build up that can lead to renal failure.