Ravulizumab, sold under the brand name Ultomiris, is a humanized
monoclonal antibody complement inhibitor
medication designed for the treatment of
paroxysmal nocturnal hemoglobinuria (PNH) and
atypical hemolytic uremic syndrome. It is designed to bind to and prevent the activation of
Complement component 5 (C5).
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.
The most common side effects are upper respiratory tract infection (nose and throat infection), nasopharyngitis (inflammation of the nose and throat) and headache.
The most serious side effect is meningococcal infection, a bacterial infection caused by ''Neisseria meningitidis'' that can cause meningitis and
sepsis.
Medical uses
In the United States, ravulizumab is
indicated
In medicine, an indication is a valid reason to use a certain test, medication, procedure, or surgery. There can be multiple indications to use a procedure or medication. An indication can commonly be confused with the term diagnosis. A diagnosis ...
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.
Names
Ravulizumab is the
International Nonproprietary Name (INN).
History
Ravulizumab was developed by Alexion Pharmaceuticals, Inc. It was engineered from
eculizumab to have a longer-lasting effect.
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
The European Medicines Agency (EMA) is an agency of the European Union (EU) in charge of the evaluation and supervision of medicinal products. Prior to 2004, it was known as the European Agency for the Evaluation of Medicinal Products or Euro ...
(EMA) recommended the granting of a conditional marketing authorization for ravulizumab.
Ravulizumab was approved for medical use in the EU in July 2019.
[ Text was copied from this source which is © European Medicines Agency. Reproduction is authorized provided the source is acknowledged.]
References
Further reading
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External links
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AstraZeneca brands
Monoclonal antibodies
Orphan drugs
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