The US Food and Drug Administration has given the thumbs up to Novartis’ auto-inflammatory disorder drug Ilaris (canakinumab).

Specifically the drug will treat children and adults with cryopyrin-associated periodic syndrome, which includes a number of rare but life-long auto-inflammatory disorders.

CAPS is caused by a single gene mutation that leads to overproduction of interleukin-1 beta, causing sustained inflammation. Ilaris, a fully human monoclonal antibody, selectively blocks interleukin-1 beta.

The approval is a positive move for CAPS sufferers – of which there are about 300 known cases in the USA – as current treatments are limited to traditional inflammatory-disease medications that suppress the entire immune system or treatments that require frequent injections. Ilaris has a dosing schedule of one injection every eight weeks.

The drug’s approval follows the announcement of positive late-stage data earlier this month. A Phase III study, involving 35 CAPS patients aged nine to 74, found that more than 90% of CAPS patients treated with Ilaris remained in remission at the end of the final four-month phase of the study.

Ilaris has orphan drug designation in the USA and European Union. Other studies are ongoing evaluating the potential of Ilaris to treat people with the currently untreated neonatal-onset multisystem inflammatory disease, systemic juvenile idiopathic arthritis, forms of gout, chronic obstructive pulmonary disorder, type 2 diabetes and rheumatoid arthritis.