New Novartis ‘Niche Drug’ Approved for Rare Genetic Disorder

Drug maker Novartis AG has gained Food and Drug Administration approval for a new drug that treats a rare genetic disorder which afflicts just a few thousand people in the world.

The drug, Ilaris, represents a change in course of sorts for drug companies, who have begun developing more new drugs that are designed to correct one or two genetic triggers which cause rare diseases rather than cranking out medications designed to treat millions of people afflicted with more common disorders.

It may sound counter intuitive to market drugs for so-called “niche diseases” that affect fewer people than other conditions, but by focusing on specific genetic-based disorders that can be easier to unravel and develop drugs to treat, Novartis and other drug companies are gambling on the belief that such an approach will pay off in the long run, according to a report in The Wall Street Journal.

Novartis hopes the approach to developing new drugs will one day blossom into a market as big as today’s blockbuster drugs. Ilaris is Novartis’ first venture into genetic trigger drugs.

The new way of developing drugs certainly doesn’t come cheap. In 2002, Novartis spent $4 billion to build a new research center in Cambridge, Mass. and hired hundreds of scientists to work there, according to the Journal report.

Novartis won’t say how much it will cost to produce Ilaris, but industry analysts say it is likely to be among the world’s most expensive drugs.

Rare Genetic Syndrome is Treated
Ilaris is delivered via a shot and treats a rare, genetic disorder called cryopyrin-associated periodic syndrome (CAPS). The syndrome is caused by a mutation in a single gene and there are just 300 known cases in the entire United States. Worldwide, there are maybe 7,000 world-wide, medical experts say.

People with CAPS produce too much interleukin-1, a protein that is associated with the immune system, and develop inflammation, chronic rash, joint pain, and fever. In the most serious cases, CAPS can lead to kidney failure, deafness, and even death. Ilaris and its rival drug, Arcalyst, prevent the disease by stopping production of the protein.

Broader Uses Are Possible
The makers of Ilaris and Arcalyst are considering asking for FDA approval to treat gout, a more common inflammatory condition that causes chronic joint pain. Clearing the drugs for broader uses would increase profits for the drugs.

Novartis also is working on other drugs that genetically target disease. Those drugs currently in development include AIN457 for psoriasis and an inflammatory disorder of the eye that could hit pharmacy shelves as early as 2010 and AFQ056, a Parkinson’s disease drug the company plans for a 2012 launch.

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