Ultra-Rare to Rare: Two Execs Talk About Alexion's Shift

Alexion Pharmaceuticals is a company focused on ultra-rare diseases, although it is working on a shift into rare diseases. This may sound like something of a minor shift, but it represents developing drugs for more diseases with larger patient populations. The company’s success has been built primarily on its Soliris (eculizumab), a complement inhibitor. The drug has been approved for paroxysmal nocturnal hemoglobinuria (PNH), atypical Hemolytic Uremic Syndrome (aHUS) and for generalized Myasthenia Gravis (gMG). Soliris, as well as the company’s next-generation complement inhibitor, Ultomiris, underline the company’s expertise in complement-related diseases. The complement system is part of the immune system made up of about 30 different proteins. As such it plays a role in numerous diseases at some level, and alone in several diseases, including PNH, aHUS and others.