Ilaris, a treatment for the rare but potentially fatal auto-inflammatory disease cryopyrin-associated periodic syndrome (CAPS) has been recommended for approval in the EU.
The treatment was given the thumbs up by the Committee for Medicinal Products for Human Use (CHMP), which reviews medicines for the European Commission. If approved, the Novartis drug will be the only treatment in the EU for CAPS patients aged four years and older.
Ilaris is an advanced personalised medicine that directly targets the mutation in CAPS patients, which drives the overproduction of interleukin 1-beta (IL-1ß) causing widespread sustained inflammation and tissue damage.
Novartis Pharma head of global development Trevor Mundel said that by concentrating initially on a rare syndrome with a well-defined disease process such as CAPS, the company had been able to demonstrate a clear therapeutic advantage with Ilaris.
"Our focus now is to establish whether this could also provide a new approach to the treatment of other diseases involving a similar underlying process," Mundel said.
The EU submission was supported by data showing that Ilaris produced rapid and sustained remission of symptoms in up to 97% of CAPS patients, with most responding from the first injection.
The recommendation comes shortly after approvals in the US and Switzerland where Ilaris was granted priority review based on its potential as a significant CAPS treatment.