Ravulizumab Explained

Ravulizumab should not be confused with Rovelizumab.

Type:mab
Mab Type:mab
Source:zu/o
Target:Complement component 5
Pronounce:rav" ue liz' ue mab
Tradename:Ultomiris
Dailymedid:Ravulizumab
Pregnancy Au:B2
Routes Of Administration:Intravenous
Class:Complement inhibitor
Atc Prefix:L04
Atc Suffix:AJ02
Legal Au:S4
Legal Au Comment:[1]
Legal Ca:Rx-only
Legal Ca Comment:/ Schedule D[2] [3] [4]
Legal Uk:POM
Legal Uk Comment:[5]
Legal Us:Rx-only
Legal Eu:Rx-only
Legal Status:Rx-only
Metabolism:various proteases
Cas Number:1803171-55-2
Drugbank:DB11580
Chemspiderid:none
Unii:C3VX249T6L
Kegg:D11054
Synonyms:ALXN1210, ravulizumab-cwvz
C:6430
H:9888
N:1696
O:2028
S:48

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).[6]

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.

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).[7]

In the European Union, ravulizumab is indicated in the treatment of adults with paroxysmal nocturnal haemoglobinuria:

Adverse effects

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.

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.[12]

Society and culture

Brand names

Ravulizumab is the International Nonproprietary Name (INN).[13]

Further reading

Notes and References

  1. Web site: Ultomiris . Therapeutic Goods Administration (TGA) . 26 May 2022 . 9 March 2024.
  2. Web site: Ultomiris Product information . Health Canada . 29 May 2022.
  3. Web site: Summary Basis of Decision (SBD) for Ultomiris . . 23 October 2014 . 29 May 2022.
  4. Web site: Regulatory Decision Summary for Ultomiris . Drug and Health Products Portal . 6 January 2023 . 1 April 2024.
  5. Web site: Ultomiris 300 mg concentrate for solution for infusion - Summary of Product Characteristics (SmPC) . (emc) . 12 October 2020 . 18 October 2020 . https://web.archive.org/web/20201018031618/https://www.medicines.org.uk/emc/product/10457 . dead .
  6. Web site: Ultomiris- ravulizumab solution, concentrate . DailyMed . 20 March 2020 . 1 May 2020.
  7. Web site: FDA approves therapy for serious rare blood disease . U.S. Food and Drug Administration (FDA) . 7 June 2021 . 7 June 2021.
  8. https://searchusan.ama-assn.org/undefined/documentDownload?uri=%2Funstructured%2Fbinary%2Fusan%2Fravulizumab.pdf Statement On A Nonproprietary Name Adopted By The USAN Council - Ravulizumab
  9. Röth A, Rottinghaus ST, Hill A, Bachman ES, Kim JS, Schrezenmeier H, Terriou L, Urbano-Ispizua Á, Wells RA, Jang JH, Kulasekararaj AG, Szer J, Aguzzi R, Damokosh AI, Shafner L, Lee JW . Ravulizumab (ALXN1210) in patients with paroxysmal nocturnal hemoglobinuria: results of 2 phase 1b/2 studies . Blood Advances . 2 . 17 . 2176–2185 . September 2018 . 30171081 . 6134221 . 10.1182/bloodadvances.2018020644 .
  10. FDA approves new treatment for adult patients with rare, life-threatening blood disease . https://web.archive.org/web/20190206211918/https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm629022.htm . 6 February 2019 . U.S. Food and Drug Administration (FDA) . dead .
  11. Web site: EMA Positive Opinion - Ultomiris / ravulizumab, April 26, 2019. ema.europa.eu. 11 May 2019.
  12. Web site: Ultomiris EPAR . European Medicines Agency (EMA) . 24 April 2019 . 1 May 2020. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  13. ((World Health Organization)) . 2018 . International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 79 . WHO Drug Information . 32 . 1 . 10665/330941 . free . World Health Organization .

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