An AstraZeneca drug acquired in a $930 million deal has received FDA approval as a treatment for a rare blood disorder. In this disease, paroxysmal nocturnal hemoglobinuria (PNH), a part of the immune ...

Alexion Pharmaceuticals (Nasdaq: ALXN) announced positive results today from its Phase 3 study of ALXN1210 for patients with paroxysmal nocturnal hemoglobinuria (PNH), a rare, life-threatening blood ...

Orphan drugmakers focus on developing drugs for rare diseases, but unfortunately many companies in this space never become profitable. Of those that do, few are able to succeed like Alexion ...

Alexion Pharmaceuticals is a big player in the treatment of rare diseases, but its dominance in atypical hemolytic uremic syndrome (aHUS) could be in jeopardy if a soon-to-begin clinical trial at ...