Ultomiris
An intravenous biological therapy administered every 8 weeks to treat rare blood and neurological disorders.
Where Ravulizumab is approved
All regulators →Of the three regulators tracked here, the first to approve Ravulizumab was the US, on 21 Dec 2018. It is approved in 3 of the 3 regulators tracked here.
From the regulators' own registers: FDA Drugs@FDA, the EMA medicines list and Health Canada's Notice of Compliance database. Dates are the first listed approval of a product containing only this substance; combination products and nationally authorised EU medicines are not counted.
What health systems spend on Ravulizumab
All medicines →From NHSBSA Prescription Cost Analysis (Open Government Licence v3.0) and CMS State Drug Utilization Data. Both are gross amounts: neither system publishes its discounts or rebates per drug. Products combining several substances are not counted here.
How Ravulizumab works
All drug targets →- Complement C5 inhibitor InhibitorTarget: C5 complement C5ChEMBL only
- IgA glomerulonephritisPhase 3
- Acute kidney injuryPhase 3
- Acute lung injuryPhase 3
- Acute respiratory distress syndromePhase 3
- Amyotrophic lateral sclerosisPhase 3
- Atypical hemolytic-uremic syndromePhase 3
- Myasthenia gravisPhase 3
- Neuromyelitis opticaPhase 3
- PneumoniaPhase 3
- Thrombotic microangiopathyPhase 3
Mechanisms and diseases from ChEMBL via the Open Targets Platform (release 26.09), ChEMBL record CHEMBL3989986, CC BY-SA 3.0. Targets checked against the IUPHAR/BPS Guide to PHARMACOLOGY (2026.3), ligand 10168, CC BY-SA 4.0. Trial phases are not approvals: approved uses are shown from the regulators' own records where we hold them.
NICE appraisals of Ravulizumab
All NICE appraisals →NICE, which decides whether the NHS in England should fund new medicines, has published 4 technology appraisals of Ravulizumab. 4 are current, and 2 of these recommend it for at least some patients.
| Appraisal | NICE recommendation | Published | Status |
|---|---|---|---|
| Ravulizumab for treating generalised myasthenia gravis (terminated appraisal) NICE TA940 | Terminated (no company submission) | 20 Dec 2023 | Current |
| Ravulizumab for treating AQP4 antibody-positive neuromyelitis optica spectrum disorder (terminated appraisal) NICE TA941 | Terminated (no company submission) | 20 Dec 2023 | Current |
| Ravulizumab for treating atypical haemolytic uraemic syndrome NICE TA710 | Recommended | 23 Jun 2021 | Current |
| Ravulizumab for treating paroxysmal nocturnal haemoglobinuria NICE TA698 | Recommended | 19 May 2021 | Current |
NICE decisions apply to the NHS in England. In Ireland the HSE decides on reimbursement, advised by the National Centre for Pharmacoeconomics. Titles, categories and dates link to the guidance on nice.org.uk. © NICE 2026 technology appraisal guidance. Available from www.nice.org.uk/guidance. All rights reserved. Subject to Notice of rights. NICE guidance is prepared for the National Health Service in England. All NICE guidance is subject to regular review and may be updated or withdrawn. NICE accepts no responsibility for the use of its content in this product/publication.
Patient leaflets for Ravulizumab
The package leaflet is the official guide for patients that comes with every medicine. These links go to the regulators that publish it.
- IEIreland: 2 products on the HPRA listEach product record links to the HPRA, which publishes the leaflet and SmPC.
- EUEuropean Union: UltomirisEMA product information: package leaflet and summary of product characteristics in every EU language.
- UKUnited Kingdom: Search MHRA ProductsPatient information leaflets and SmPCs for UK-licensed products.
- USUnited States: Search DailyMedFDA-approved labels, including patient and medication guides.
Leaflets differ between brands and countries; always read the one that comes with your own medicine. This is regulatory information, not medical advice.
What Ultomiris is used for
ULTOMIRIS is a complement inhibitor indicated for: the treatment of adult and pediatric patients one month of age and older with paroxysmal nocturnal hemoglobinuria (PNH). ( 1.1 ) the treatment of adult and pediatric patients one month of age and older with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (TMA). ( 1.2 ) Limitations of Use: ULTOMIRIS is not indicated for the treatment of patients with Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS). the treatment of adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive. ( 1.3 ) the treatment of adult patients with neuromyelitis optica spectrum disorder (NMOSD) who are anti-aquaporin-4 (AQP4) antibody-positive. ( 1.4 ) 1.1 Paroxysmal Nocturnal Hemoglobinuria ULTOMIRIS is indicated for the treatment of adult an…
How it works
12.1 Mechanism of Action Ravulizumab-cwvz is a terminal complement inhibitor that specifically binds to the complement protein C5 with high affinity, thereby inhibiting its cleavage to C5a (the proinflammatory anaphylatoxin) and C5b (the initiating subunit of the membrane attack complex [MAC or C5b-9]) thus preventing MAC formation. ULTOMIRIS inhibits terminal complement-mediated intravascular hemolysis in patients with PNH and complement-mediated thrombotic microangiopathy (TMA) in patients with aHUS. The precise mechanism by which ravulizumab-cwvz exerts its therapeutic effect in gMG patients is unknown, but is presumed to involve reduction of terminal complement complex C5b-9 deposition …
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How this page is built
The facts on this page are pulled directly from official U.S. FDA datasets — they are not written from memory. Each field below names the dataset it came from, so you can check it yourself.
- FDA Drug Label (openFDA) ↗ Indications, mechanism of action and pharmacologic class
- Drugs@FDA ↗ First FDA approval date and the company holding the application
- FDA Drug Shortage Database ↗ Current US supply / shortage status
Plain-English summaries and drug-class explainers are written and reviewed by Sreepriya Prasannan (MSc Digital Transformation of Life Sciences (Innopharma Education / Griffith College); MSc & BSc Botany). Data is retrieved automatically from the sources above and cross-checked with AI-assisted verification (Anthropic's Claude) — brand and generic names are matched against the exact FDA product record so that a combination product or a different formulation cannot be mistaken for the drug on this page. An editor reviews the result before publication. We describe this in full in our editorial standards and corrections policy. The FDA data on this page was last retrieved on 10 Oct 2026. How every register is built: methodology · fixes we have made: corrections log.