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Monoclonal antibody
TypeWhole antibody
SourceHumanized (from mouse)
TargetComplement component 5
Pronunciationrav" ue liz' ue mab
Trade namesUltomiris
Other namesALXN1210, ravulizumab-cwvz
Clinical data
Drug classComplement inhibitor[1]
Main usesParoxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome[2]
Side effectsUpper respiratory tract infection, diarrhea, fever, nausea, headache[3][2]
  • AU: B2
Routes of
External links
License data
Legal status
  • AU: S4 (Prescription only)
  • UK: POM (Prescription only) [4]
  • US: ℞-only [5]
  • EU: Rx-only [3]
  • In general: ℞ (Prescription only)
Metabolismvarious proteases
Chemical and physical data
Molar mass144938.56 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.[2] It is given by gradual injection into a vein.[1]

Common side effects include upper respiratory tract infection, diarrhea, fever, nausea, and headache.[3][2] Other side effect may include meningococcal infection.[3] While there is no clear harm in pregnancy, such use has not been well studied.[6] It is a monoclonal antibody that bind to and blocks the activity of complement component 5 (C5).[3]

Ravulizumab was approved for medical use in the United States in 2018 and Europe in 2019.[1][3] In the United States it costs about 460,000 USD per year as of 2019.[7]

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).[5][8]

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[3]
  • in people who are clinically stable after having been treated with eculizumab for at least the past six months.[3]


The dose in an adult is around 2,700 mg followed by 3,300 mg every 8 weeks.[2]


Pathogenesis of hemolysis in paroxysmal nocturnal hemoglobinuria[9]

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).[8]

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.[3]

Ravulizumab, is a monoclonal antibody (a type of protein) designed to attach to the C5 protein, which is part of the complement system.[3]

By attaching to the C5 protein, the medicine blocks its effect and thereby reduces the destruction of red blood cells.[3]


Ravulizumab was developed by Alexion Pharmaceuticals, Inc.[10] It was engineered from eculizumab to have a longer-lasting effect.[11]

Ravulizumab was approved by the US Food and Drug Administration (FDA) in December 2018.[12] 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.[13] Ravulizumab was approved for medical use in the EU in July 2019.[3]


Ravulizumab is the International Nonproprietary Name (INN).[14]


  1. 1.0 1.1 1.2 "Ravulizumab-cwvz Monograph for Professionals". Drugs.com. Retrieved 16 October 2021.
  2. 2.0 2.1 2.2 2.3 2.4 "DailyMed - ULTOMIRIS- ravulizumab solution, concentrate". dailymed.nlm.nih.gov. Archived from the original on 23 September 2020. Retrieved 16 October 2021.
  3. 3.00 3.01 3.02 3.03 3.04 3.05 3.06 3.07 3.08 3.09 3.10 3.11 "Ultomiris EPAR". European Medicines Agency (EMA). 24 April 2019. Archived from the original on 8 May 2020. Retrieved 1 May 2020. Text was copied from this source which is © European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  4. "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.
  5. 5.0 5.1 "Ultomiris- ravulizumab solution, concentrate". DailyMed. 20 March 2020. Archived from the original on 23 September 2020. Retrieved 1 May 2020.
  6. "Ravulizumab (Ultomiris) Use During Pregnancy". Drugs.com. Archived from the original on 29 October 2020. Retrieved 16 October 2021.
  7. Stern, RM; Connell, NT (2019). "Ravulizumab: a novel C5 inhibitor for the treatment of paroxysmal nocturnal hemoglobinuria". Therapeutic advances in hematology. 10: 2040620719874728. doi:10.1177/2040620719874728. PMID 31534662.
  8. 8.0 8.1 "FDA approves therapy for serious rare blood disease". U.S. Food and Drug Administration (FDA). 7 June 2021. Archived from the original on 7 June 2021. Retrieved 7 June 2021. Public Domain This article incorporates text from this source, which is in the public domain.
  9. Gurnari, Carmelo; Nautiyal, Ishani; Pagliuca, Simona (2021). "Current Opinions on the Clinical Utility of Ravulizumab for the Treatment of Paroxysmal Nocturnal Hemoglobinuria". Therapeutics and Clinical Risk Management. 17: 1343–1351. doi:10.2147/TCRM.S273360. ISSN 1176-6336.
  10. Statement On A Nonproprietary Name Adopted By The USAN Council - Ravulizumab Archived 2018-02-27 at the Wayback Machine, American Medical Association.
  11. 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.
  12. "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.
  13. "EMA Positive Opinion - Ultomiris / ravulizumab, April 26, 2019" (PDF). ema.europa.eu. Archived (PDF) from the original on 5 May 2019. Retrieved 11 May 2019.
  14. World Health Organization (2017). "International Nonproprietary Names for Pharmaceutical Substances (INN). Proposed INN: List 117" (PDF). WHO Drug Information. 31 (2). Archived (PDF) from the original on 2018-02-05. Retrieved 2021-06-08.

External links

External sites: