FDA approves new drug, Ravulizumab, to be marketed for PNH

PNH is a rare blood disease characterized by complement-mediated red blood cell destruction (hemolytic) that can cause a wide range of debilitating symptoms and complications, such as hemolytic, venous thrombosis and regenerative anemiaThe disease can occur throughout the body and can lead to organ damage and premature deathrecently,fda(http://approved thenew drug(http://, Ululizumab, for the treatment of haemoglobinuria (PNH)The drug is the first long-acting C5 supplement inhibitorUltomirisUlulizumab is the first and currently the only long-acting C5 complement inhibitor that works by suppressing the C5 protein in the end-to-end complement cascade reactionC5 protein is part of the body's immune system, when it is not controlled and activated, it can cause PNH, hemolytic uremic syndrome (aHUS), anti-acetylcholine receptor (AchR) and antibody-positive severe muscle weakness and other serious rare diseasesin addition to approved PNH indications, Ultomiris is currently conducting phase III clinicaltrial
synod (http://for hemolytic uremia syndrome (aHUS) In addition, clinical trials for the treatment of severe muscle weakness and the feasibility of subcutaneous injections are also planned to begin the approval is based on two comprehensive studies published in the journal Blood 441 patients participated in the trial, some of the subjects had never used tonic inhibitors, and others had been treated with Soliris for a long time and were stable in stable condition comparison found that Ultomiris, which was given every eight weeks, had the same effect as Soliris, which was given every 2 weeks, and was similar in safety