Regulators in Europe have given a green light to Novartis’ auto-inflammatory disorder drug Ilaris.

Ilaris (canakinumab) has been approved in the European Union to treat adults and children as young as four years old with cryopyrin-associated periodic syndrome. CAPS is caused by a single gene mutation that leads to overproduction of interleukin-1 beta, causing sustained inflammation and symptoms include debilitating fatigue, fever, joint pain and conjunctivitis.

Novartis noted that Ilaris is the only approved treatment in the EU for patients who suffer from two forms of CAPS, notably the most debilitating form of the disease, neonatal-onset multisystem inflammatory disease. The decision follows approvals in the USA and Switzerland earlier this year and the company added that long-term consequences of CAPS include deafness, bone and joint deformities, plus central nervous system damage leading to visual loss. Around 25% of patients develop amyloidosis, whereby the build-up of proteins can cause vital organs to fail, resulting in renal failure and requiring kidney transplantation.

Analysts have valued the CAPS market at about $50 million, but Novartis believes that the compound has a lot of potential in a number of other indications. Joe Jimenez, chief executive of Novartis Pharma, said that “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”.

Studies with the compound are ongoing in other diseases in which IL-1 beta plays an important role, such as chronic obstructive pulmonary disease, type 2 diabetes, systemic juvenile idiopathic arthritis and gout. Last week, Novartis presented new Phase II results which show that canakinumab “is significantly more effective” than the injectable corticosteroid triamcinolone in treating gout.