Ultomiris ULTOMIRIS® (ravulizumab)
PNH is a rare, chronic, life-threatening disease affecting the complement cascade, leading to uncontrolled terminal complement activation.3,5-9

Uncontrolled terminal complement activation leads to life-threatening consequences such as IVH, thromboembolic events, renal impairment and pulmonary hypertension.10-12 If PNH is left untreated, it can lead to early mortality.5,13
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ULTOMIRIS® targets uncontrolled terminal complement activity and delivers immediate, complete and sustained C5 inhibition.1,2,4 For more details, please visit our dedicated Mechanism of Action page below.
Mechanism of Action of ULTOMIRIS®
ULTOMIRIS® is contraindicated in patients who have not been vaccinated against Neisseria meningitidis. If a patient initiates ULTOMIRIS® treatment less than 2 weeks after receiving a meningococcal vaccine, appropriate prophylactic antibiotics should be administered until two weeks after the vaccination.1

ULTOMIRIS® is also contraindicated in patients with hypersensitivity to the active substance or any excipients within the formulation.1

For further information, please visit our dedicated Dosing and Administration page.. 
Dosing and Administration
The efficacy and safety of ULTOMIRIS® were evaluated in adults and paediatrics in three Phase 3 trials.2,4,14 More information on how ULTOMIRIS® was studied can be found below.
ULTOMIRIS® Study Design
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Detailed information on the efficacy of ULTOMIRIS® is available below.
Efficacy of ULTOMIRIS®
With ULTOMIRIS®, treatment can be kept on track with a well-established safety profile.1,4 Further details about the safety of ULTOMIRIS® can be accessed on the page beneath.
Safety of ULTOMIRIS®
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*ULTOMIRIS® indications: PNH, atypical haemolytic uraemic syndrome, generalised myasthenia gravis and neuromyelitis optica spectrum disorder.1
Please consult the Summary of Product Characteristics prior to prescribing.
Please report any adverse events via your national reporting system. Adverse events can also be reported to Alexion, AstraZeneca Rare Disease by contacting: https://contactazmedical.astrazeneca.com/
IVH, intravascular haemolysis; MAVE, major adverse vascular events; PNH, paroxysmal nocturnal haemoglobinuria.                                                                              
ULTOMIRIS® EU Summary of Product Characteristics available at: https://www.ema.europa.eu/en/search?f%5B0%5D=ema_search_entity_is_document%3ADocument&search_api_fulltext=Ultomiris%20%3A%20EPAR%20-%20Product%20information. Last accessed January 2026. Kulasekararaj AG, et al. Ravulizumab (ALXN1210) vs eculizumab in C5-inhibitor–experienced adult patients with PNH: the 302 study. Blood. 2019;133(6):540–549. Kulasekararaj AG, et al. Long-term safety and efficacy of ravulizumab in patients with paroxysmal nocturnal hemoglobinuria: 2-year results from two pivotal phase 3 studies. Eur J Haematol. 2022;3(109):205–214. Lee JW, et al. Ravulizumab (ALXN1210) vs eculizumab in adult patients with PNH naive to complement inhibitors: the 301 study. Blood. 2019;133(6):530–539. Brodsky RA.  Blood. 2014;124(18):2804–2811. Hill A, et al. Paroxysmal nocturnal hemoglobinuria. Blood. 2013;25(121):4985–4996. Risitano AM, et al. The complement alternative pathway in paroxysmal nocturnal hemoglobinuria: From a pathogenic mechanism to a therapeutic target. Immunological Reviews. 2023; 262-278. Kulasekararaj AG, et al. Ravulizumab demonstrates long-term efficacy, safety and favorable patient survival in patients with paroxysmal nocturnal hemoglobinuria Annals of Hematology. 2025;104(1):81–94. Kulasekararaj AG, et al. The Importance of Terminal Complement Inhibition in Paroxysmal Nocturnal Hemoglobinuria. Therapeutic Advances in Hematology. 2022. Risitano AM, Rotoli B. Paroxysmal nocturnal hemoglobinuria: pathophysiology, natural history and treatment options in the era of biological agents. Biologics. 2008;2(2):205–222. Szlendak U, et al. Paroxysmal Nocturnal Hemoglobinuria Advances in the Understanding of Pathophysiology, Diagnosis, and Treatment. Polish Archives of Internal Medicine 2022. Van Bijnen, et al. Mechanisms and clinical implications of thrombosis in paroxysmal nocturnal hemoglobinuria. Journal of Thrombosis and Haemostasis. 2012. Sharma VR. Paroxysmal nocturnal hemoglobinuria: pathogenesis, testing, and diagnosis. Clinical Advances in Hematol & Oncology. 2013;9(11 Suppl 13):2–8. Chonat C, et al. Pharmacokinetics, pharmacodynamics, efficacy, and safety of ravulizumab in pediatric paroxysmal nocturnal hemoglobinuria. Blood Advances. 2024;8(11):2813–2824. Alexion Pharmaceuticals LP. Data on File. DE market share. May 2024. Alexion Pharmaceuticals LP. Data on File. Periodic benefit–risk evaluation report: Ravulizumab (ULTOMIRIS). February 2025.