Ravulizumab

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).[5][4]

Not to be confused with Rovelizumab.
Ravulizumab
Monoclonal antibody
TypeWhole antibody
SourceHumanized (from mouse)
TargetComplement component 5
Clinical data
Pronunciationrav" ue liz' ue mab
Trade namesUltomiris
Other namesALXN1210, ravulizumab-cwvz
AHFS/Drugs.comMonograph
MedlinePlusa619014
License data
Pregnancy
category
  • AU: B2
Routes of
administration		Intravenous
ATC code
Legal status
Legal status
Pharmacokinetic data
Metabolismvarious proteases
Identifiers
CAS Number
DrugBank
ChemSpider
  • none
UNII
KEGG
Chemical and physical data
FormulaC6430H9888N1696O2028S48
Molar mass144938.56 g·mol−1

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).[6] 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.[5] Ravulizumab, is a monoclonal antibody (a type of protein) designed to attach to the C5 protein, which is part of the complement system.[5] By attaching to the C5 protein, the medicine blocks its effect and thereby reduces the destruction of red blood cells.[5]

The most common side effects are upper respiratory tract infection (nose and throat infection), nasopharyngitis (inflammation of the nose and throat) and headache.[5][4] The most serious side effect is meningococcal infection, a bacterial infection caused by Neisseria meningitidis that can cause meningitis and sepsis.[5][4]

Medical uses

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).[4][6]

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[5]
  • in people who are clinically stable after having been treated with eculizumab for at least the past six months.[5]

Names

Ravulizumab is the International Nonproprietary Name (INN).[7]

History

Ravulizumab was developed by Alexion Pharmaceuticals, Inc.[8] It was engineered from eculizumab to have a longer-lasting effect.[9]

Ravulizumab was approved by the US Food and Drug Administration (FDA) in December 2018.[10] 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.[11] Ravulizumab was approved for medical use in the EU in July 2019.[5]

References
  1. "Ultomiris Product information". Health Canada. Retrieved 29 May 2022.
  2. "Summary Basis of Decision (SBD) for Ultomiris". Health Canada. 23 October 2014. Retrieved 29 May 2022.
  3. "Ultomiris 300 mg concentrate for solution for infusion - Summary of Product Characteristics (SmPC)". (emc). Archived from the original on 18 October 2020. Retrieved 12 October 2020.
  4. "Ultomiris- ravulizumab solution, concentrate". DailyMed. 20 March 2020. Retrieved 1 May 2020.
  5. "Ultomiris EPAR". European Medicines Agency (EMA). 24 April 2019. Retrieved 1 May 2020. Text was copied from this source which is © European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  6. "FDA approves therapy for serious rare blood disease". U.S. Food and Drug Administration (FDA). 7 June 2021. Retrieved 7 June 2021. Public Domain This article incorporates text from this source, which is in the public domain.
  7. World Health Organization (2017). "International Nonproprietary Names for Pharmaceutical Substances (INN). Proposed INN: List 117" (PDF). WHO Drug Information. 31 (2).
  8. Statement On A Nonproprietary Name Adopted By The USAN Council - Ravulizumab, American Medical Association.
  9. Röth A, Rottinghaus ST, Hill A, Bachman ES, Kim JS, Schrezenmeier H, et al. (September 2018). "Ravulizumab (ALXN1210) in patients with paroxysmal nocturnal hemoglobinuria: results of 2 phase 1b/2 studies". Blood Advances. 2 (17): 2176–2185. doi:10.1182/bloodadvances.2018020644. PMC 6134221. PMID 30171081.
  10. "FDA approves new treatment for adult patients with rare, life-threatening blood disease" (Press release). U.S. Food and Drug Administration (FDA). Archived from the original on 6 February 2019.
  11. "EMA Positive Opinion - Ultomiris / ravulizumab, April 26, 2019" (PDF). ema.europa.eu. Retrieved 11 May 2019.

Further reading
  • "Ravulizumab". Drug Information Portal. U.S. National Library of Medicine.


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