FDA grants Achillion's danicopan breakthrough therapy
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Last Update: 2020-06-08
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Source: Internet
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Author: User
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a sleeping hemoglobinuria (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 leukemiaToday, Http://, a company developed by Http:// announcedthat the company's danicopan was approved by the U.SFDA(http://, and is used in conjunction with C5 complement inhibitors to treat patients with periphery ouseremia (PNH) who are not responding well to The Treatment of C5 inhibitorsThe finding sits based on positive data from Danicopan's(http://in a Phase 2trial, the results of which will be published at the end of this yearIn 2017, the FDA has granted the danicopan orphan drug a licenseTonic factor D is a key protein in the alternative pathway, and inhibiting tonic 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 enzymesUnlike C5 inhibitors, danicopan prevents the deposition of C3b fragments on the patient's RBC, controls the breakdown of red blood cells in PNH patients, and exosotic hemolysis, thereby improving the patient's treatment
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