Alexion Pharmaceuticals, Inc. (ALXN, Financial) is a biopharmaceutical company that pioneered the “orphan disease” space by focusing on delivering life-transforming treatments for patients with devastating (life threatening) and ultra-rare (or orphan) diseases. Alexion is a leader in disease education and diagnostic initiatives to ensure that patients for its drugs are rapidly and accurately diagnosed. We believe Alexion’s first commercial drug, known as Soliris, which is used to treat paroxysmal nocturnal hemoglobinuria (called PNH) and atypical hemolytic uremic syndrome (referred to as aHUS), continues to possess a large global growth opportunity. Alexion has a pipeline of additional rare-disease treatments, and recently acquired Synageva, a company with two additional drugs for treating rare and devastating metabolic diseases, as well as a pipeline of other compounds, giving the company what we believe to be the most robust rare disease pipeline in the biotech industry.
From Baron Funds’ second quarter 2015 commentary.
