Posted by Michael Wonder on 25 Sep 2019
Achillion receives breakthrough therapy designation from FDA for danicopan for treatment of paroxysmal nocturnal hemoglobinuria
25 September 2019 - Achillion Pharmaceuticals today announced that the U.S. FDA has granted breakthrough therapy designation for danicopan (ACH-4471) for treatment in combination with a C5 monoclonal antibody for patients with paroxysmal nocturnal haemoglobinuria who are sub-optimal responders to a C5 inhibitor alone.
The FDA’s 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 Paroxysmal Nocturnal Hemoglobinuria Meeting in May 2019.
The top line data from this combination trial is expected in the fourth quarter of 2019.
