Alexion is a global biopharmaceutical company focused on developing and delivering life-transforming therapies for patients with devastating and rare diseases. Alexion was established in the U.S. in 1992 and became a public company in 1996 (NASDAQ: ALXN). The global headquarters and research operations are in New Haven, Conn. Alexion’s approximately 3,000 employees around the world serve patients in 50 countries. As continue to expand the operations to serve more patients in additional countries, Alexion has emerged as one of the world’s leading rare disease companies. Today, Alexion is advancing the most robust rare disease pipeline in the biotech industry, which, in addition to the complement and metabolic clinical programs, includes more than 30 diverse preclinical programs across a range of therapeutic modalities. Alexion developed Soliris® (Eculizumab), the world’s first approved terminal complement inhibitor, from the laboratory through commercialization.
Today, Soliris is approved in nearly 50 countries for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) and in more than 40 countries for the treatment of patients with atypical hemolytic uremic syndrome (aHUS) — two life-threatening, ultra-rare disorders caused by uncontrolled complement activation. As the global leader in complement inhibition, Alexion is strengthening and broadening its portfolio of complement inhibitors, including evaluating potential indications for Eculizumab in additional severe and ultra-rare disorders. Alexion’s metabolic franchise includes two highly innovative enzyme replacement therapies for patients with life-threatening and ultra-rare disorders: Strensiq® (Asfotase Alfa) is approved for patients with Hypophosphatasia (HPP) and Kanuma® (Sebelipase Alfa) is approved for patients with lysosomal acid lipase deficiency (LAL-D).