|Source||Humanized (from mouse)|
|Target||Complement component 5|
|Pronunciation||rav" ue liz' ue mab|
|Other names||ALXN1210, ravulizumab-cwvz|
|Drug class||Complement inhibitor|
|Main uses||Paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome|
|Side effects||Upper respiratory tract infection, diarrhea, fever, nausea, headache|
|Chemical and physical data|
|Molar mass||144938.56 g·mol−1|
Ravulizumab, sold under the brand name Ultomiris, is a medication used to treat paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome. It is given by gradual injection into a vein.
Common side effects include upper respiratory tract infection, diarrhea, fever, nausea, and headache. Other side effect may include meningococcal infection. While there is no clear harm in pregnancy, such use has not been well studied. It is a monoclonal antibody that bind to and blocks the activity of complement component 5 (C5).
In the United States, ravulizumab is indicated for the treatment of adults and children one month of age and older with paroxysmal nocturnal hemoglobinuria and for the treatment of adults and children one month of age and older with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (TMA).
In the European Union, ravulizumab is indicated in the treatment of adults with paroxysmal nocturnal haemoglobinuria:
- in people with haemolysis with clinical symptom(s) indicative of high disease activity
- in people who are clinically stable after having been treated with eculizumab for at least the past six months.
The dose in an adult is around 2,700 mg followed by 3,300 mg every 8 weeks.
Paroxysmal nocturnal hemoglobinuria is characterized by red blood cell destruction, anemia (red blood cells unable to carry enough oxygen to tissues), blood clots, and impaired bone marrow function (not making enough blood cells). In paroxysmal nocturnal hemoglobinuria, proteins known as the 'complement system', which is part of the immune system, become overactive because of a genetic mutation and start to attack the patients' own red blood cells. Ravulizumab, is a monoclonal antibody (a type of protein) designed to attach to the C5 protein, which is part of the complement system. By attaching to the C5 protein, the medicine blocks its effect and thereby reduces the destruction of red blood cells.
Ravulizumab was approved by the US Food and Drug Administration (FDA) in December 2018. In April 2019, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) recommended the granting of a conditional marketing authorization for ravulizumab. Ravulizumab was approved for medical use in the EU in July 2019.
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