{"entity":{"id":"ravulizumab","kind":"drug","name":"Ravulizumab","aka":["Ravulizumab-cwvz","ALXN1210"],"tldr":"Ravulizumab is an eight-weekly infusion that blocks the complement system; it treats paroxysmal nocturnal haemoglobinuria, a clonal bone marrow disorder managed by haematologists alongside aplastic anaemia and myelodysplasia.","summary":"Ravulizumab is a re-engineered, longer-acting successor to eculizumab that blocks complement C5. The United States approved it in 2018 for paroxysmal nocturnal haemoglobinuria (PNH), an acquired clonal disorder of blood stem cells in which complement destroys red cells, and then for atypical haemolytic uraemic syndrome and generalised myasthenia gravis. PNH is not a cancer but arises from the same stem-cell compartment as myelodysplastic syndromes and often follows aplastic anaemia, so it sits within the haematology and bone marrow failure workload of cancer centres. Complement blockade is also being tested for transplant-associated thrombotic microangiopathy after stem cell transplantation. Meningococcal infection risk requires vaccination before treatment.","status":"established","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Ravulizumab","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=ravulizumab"},{"label":"Drugs@FDA BLA 761108","url":"https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=761108"}],"tags":["gap-fill","supportive-care"],"related":[],"cancers":["mds"],"sections":[],"technologies":["supportive-care"],"targets":[],"drugs":[],"companies":["astrazeneca"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"notes":[],"brand":"Ultomiris","modality":"Long-acting anti-complement C5 monoclonal antibody","mechanism":"Binds complement protein C5 and prevents its cleavage into C5a and C5b, so the membrane attack complex cannot form and complement-driven destruction of blood cells stops.","approvals":[{"region":"US","year":2018,"indication":"Paroxysmal nocturnal haemoglobinuria","note":"Later extended to atypical haemolytic uraemic syndrome and generalised myasthenia gravis"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},"route":"/drugs/ravulizumab/","neighbours":{"cancer":[{"id":"mds","kind":"cancer","name":"Myelodysplastic syndromes / neoplasms (MDS)","route":"/cancers/mds/"}],"section":[{"id":"supportive-care","kind":"section","name":"Supportive Care & Survivorship","route":"/fronts/supportive-care/"}],"company":[{"id":"astrazeneca","kind":"company","name":"AstraZeneca","route":"/companies/astrazeneca/"}]}}