As best I can tell, Novartis
Ilaris was approved to treat CAPS. No, not the Motley Fool investing community; this CAPS (cryopyrin-associated periodic syndrome) is a group of auto-inflammatory disorders that causes the immune system to overreact. Come to think of it, overreacting is something I seem to do when I log in to see that my (Fool) CAPS score has gone down, so maybe the two are similar.
CAPS is an ultra-orphan indication. When Regeneron Pharmaceuticals
So why are Novartis and Regeneron developing drugs that treat so few people? Because the protein that is overproduced in CAPS patients, interleukin-1 beta, has been implicated in causing symptoms of patients with other diseases including gout, systemic juvenile idiopathic arthritis and type 2 diabetes.
That last one should pique your interest. There are over 23 million diabetics in the U.S. and most of those are type 2. Merck
Getting approved for a small indication and then working up from there is a pretty common strategy -- think Onyx Pharmaceuticals'
An orphaned further Foolishness:
- The best little investment Tim Hanson knows.