Achillion reports that the 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 FDA’s decision was based on positive safety and efficacy data from the ongoing danicopan Phase 2 PNH combination trial. more