Rare Disease Drug Discovery Approach Recognized



Together, nearly 30 million Americans suffer from rare diseases. Yet, within each disease there are few patients. In the past, market share shaped drug discovery as companies sought blockbuster medicines with big payoffs. But a combination of unmet medical need, new technology and collaboration across the sciences has sent Novartis in a different direction. At Novartis Institutes for Biomedical Research (NIBR) scientists look for innovative drug targets along molecular signaling pathways that can spawn disease when out of whack. Often rare diseases provide the welcome mat to these complicated pathways. By developing medicines that improve rare conditions, science is better positioned to one day tackle more complicated diseases set off by multiple signaling disorders. Novartis was the only large pharmaceutical company recognized by the National Organization for Rare Disorders (NORD) in 2010, honored for developing Ilaris® (canakinumab,) an interleukin-1ß blocker indicated for the treatment of Cryopyrin-Associated Periodic Syndromes (CAPS), in adults and children 4 years of age and older including: Familial Cold Auto-inflammatory Syndrome (FCAS) and Muckle-Wells Syndrome (MWS).

Learn more about NORD's award.