Achillion Receives Breakthrough Therapy Designation from FDA for Danicopan for Treatment of PNH
Achillion Pharmaceuticals 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 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 PNH Meeting in May 2019. The top line data from this combination trial is expected in the fourth quarter of 2019.
“The FDA’s granting of Breakthrough Therapy designation for our lead oral factor D inhibitor, danicopan, underscores the urgent need for new treatment options for patients living with PNH,” said Joe Truitt, President and Chief Executive Officer at Achillion. “Danicopan, with its demonstrated ability to limit both intravascular and extravascular hemolysis with oral administration, has the potential to benefit a significant number of patients with PNH that continue to have an unmet medical need on standard of care. We appreciate the review and decision by the FDA and plan to work closely with the Agency in advancing the development of danicopan into Phase 3 in early 2020.”
FDA Breakthrough Therapy designation is designed to expedite the development and review of medicines for serious or life-threatening conditions. Receiving Breakthrough Therapy designation from the FDA indicates preliminary clinical evidence has demonstrated the drug may provide substantial improvement on at least one clinically significant endpoint compared with currently available therapy. The benefits of this Breakthrough Therapy designation include more intensive guidance from FDA on an efficient drug development program, access to a scientific liaison to help accelerate review time and eligibility for Accelerated Approval and Priority Review if relevant criteria are met. Danicopan (ACH-4471) has previously received orphan drug designation for the treatment of PNH in 2017.