Target complement signaling pathway Innovative therapy recognized by FDA breakthrough therapy

PNH is a acquired, life-threatening rare blood disease characterized by the destruction of red blood cells (hemolytic anemia), blood clots (thrombosis), impaired bone marrow function, and the risk of leukemiaThe disease is caused by a genetic mutation that causes the normal presence on the surface of red blood cells (RBCs), and the deletion of key CD55 and CD59 receptors that interact with the alternative pathway stoiding pathway (AP) of the complement cascade reaction, causing the tonic reaction triggered by the replacement pathway to destroy the red blood cells through intravascular hemolytic actionCurrently, the main treatment for PNH is to suppress the C5 protein by using C5 tonic inhibitorsHowever, in the course of treatment with C5 inhibitors, up to 75% of PNH patients develop anemia, and some patients will need to receive blood transfusionstonic factor D is a key protein in the alternative pathway, inhibiting the supplement factor D can control the activity of the alternative pathwayAchillion developed a complement factor D inhibitor platform and applied it to the development of danicopanDanicopan regulates the alternative pathway of complement by strong, highly specific target inhibition tonic factor D, blocking the production of C3 conversion enzymesIn addition, unlike C5 inhibitors, danicopan prevents the deposition of C3b fragments on the patient's RBC, controls red blood cell decomposition in PNH patients, and exosotic hemolysis, thereby improving the patient's treatment's mechanism of action (Photo: Achillion website)"The FDA-granted oral factor D inhibitor danicopan breakthrough therapy determination demonstrates the urgent need for new therapies for PNH patients," said Joe Truitt, President and CEO of AchillionWe look forward to continuing our partnership with the FDA in the hope of moving danicopan's clinical development to Phase III by early 2020References: https://Retrieved September 25, 2019, from https://