FDA approves a new indication for Alexion's Lourtomiris (ravulizumab-cwvz)

recently, Alexion(http:// announcedthat the U.SFood andDrug(http://Administration (http://
http://has approved a new indication of Ululizumab-cwvz for the treatment of atypical hemolytic uremic syndrome (aHUS) adults and pediatrics (aHUS) adults and pediatrics (?1 month) patients to suppress tonic lythrocytic thrombosis (TMA)Ultomiris is the first and only long-acting C5 tonic inhibitor approved by regulation, and this approval is also Ultomiris's first paediatric indication approved, with Ultomiris intravenously infusion every 8 weeks or every 4 weeks after a load dose (dose) of less than 20 kg in pediatric patientsUltomiris' application for a new indications for aHUS is under review by EU and Japanese regulatorsThe approval is based on data from two global, one-arm, open label studies (one in adult aHUS and one in children's aHUS)At present, paediatric research is under way, and 14 out of 16 child patients are included in the medium-termanalysis(http://The efficacy assessment of full TMA remission was evaluated using hematological normalization parameters (platelet count and lactic acid dehydrogenase) and improved renal function (measured by an increase of 25% in serum creatinine over baseline levels) 　　The results showed that 54% of adults and 71% of children (interim data) showed complete TMA remission during the first 26 weeks of treatment Ultomiris treatment reduced thrombocytopenia (low platelet count) in 84% of adults and 93% of children, hemolytic (red blood cell destruction) in 77% of adults and 86% of children, improved kidney function in 59% of adults and 79% of children (medium-term data) 　　The most common adverse reactions are upper respiratory tract infections, diarrhea, nausea, vomiting, headache, high blood pressure and fever Severe meningococcal infections occur in patients treated with Ultomiris To minimize patient risk, a dedicated risk mitigation program (including REMS) has been developed for Ultomiris