The U.S. Food & Drug Administration approved Ilaris by Novartis AG to treat children and adults. The drug is used to treat a rare but at times fatal auto-inflammatory disease which is caused by a single genetic mutation.

Novartis said the biotech drug will be the first approved treatment for young patients as young as four, who suffer from two forms of cryopyrin-associated periodic syndrome (CAPS), familial cold auto-inflammatory syndrome and Muckle-Wells syndrome.

A single gene mutation that leads to overproduction of interleukin-1 beta, which causes sustained inflammation and tissue damage results in CAPS.  The long term consequences include deafness, bone and joint deformities, central nervous system damage leading to visual loss, possible kidney failure and early death.

The drug Ilaris which blocks an immune system protein known as interleukin-1 (IL-1) had been previously tested in Novartis' lab for rheumatoid arthritis with little success and was forgotten till revived by Dr. Tim Wright, head of translational medicine at Novartis.

Wright took a radically different approach and proposed testing the drug on a rare disease called Muckle-Wells. Dr. Trevor Mundel, who is Novartis' global head of development and Wright approached Mark Fishman, Novartis' head of global discovery and other senior executives within the company. "There was some disbelief, as we were proposing studying a disease only Trevor and I had ever heard of," Wright recalls.

They tested the drug on four patients in February 2005 and within five hours of a single injection a visible improvement in the patients symptoms were seen. Within a day the symptoms receded and at the end of a week the disease was barely detectable in the blood and at the end of six months the patient remained symptom free. "The reality is, many experimental drugs fail," Wright said. "This one is basically custom-tailored for these patients."

Despite the drugs success it never came out as evidence emerged that IL-1, an inflammatory protein, could be involved in as many as 30 other indications which included more common and potentially lucrative diseases such as gout, cardiovascular disease and  type 2 diabetes. "It got completely bogged down in these interminable debates around value that have paralyzed drug companies for years," said Mundel.

In early 2007 that development of the drug was undertaken seriously for (CAPS), which include Muckle-Wells. In the ensuing delay Regeneron Pharmaceuticals REGN in Tarrytown, N.Y., was able to market their drug first, in February 2008.

Novartis' phase 3 trial of the drug, Ilaris showed over 90% of children and adults who suffered from the immune disorders experienced rapid, sustained remissions results of which were published in the June 3 the New England Journal of Medicine.

The Swiss drug maker hopes over the next three years to file for approval to use Ilaris to treat gout, juvenile arthritis, rheumatoid arthritis, and neonatal onset multisystem inflammatory disease, which causes brain inflammation in newborns. The company is also exploring its use in chronic obstructive pulmonary disease and diabetes.