Date: 27-Sep-2019

Achillion Receives Breakthrough Therapy Designation from FDA for Danicopan for Treatment of Paroxysmal Nocturnal Hemoglobinur

Achillion Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company dedicated to transforming the lives of patients and families affected by complement-mediated diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for danicopan (ACH-4471) for treatment in combination with a C5 monoclonal antibody for patients with paroxysmal nocturnal hemoglobinuria (PNH) who are sub-optimal responders to a C5 inhibitor alone. The FDAs decision was based on positive safety and efficacy data from the ongoing danicopan Phase 2 PNH combination trial. Interim data was reported at the New Era of Aplastic Anemia and PNH Meeting in May 2019. The top line data from this combination trial is expected in the fourth quarter of 2019.