ravulizumab   Click here for help

GtoPdb Ligand ID: 10168

Synonyms: ALXN-1210 | ALXN1210 | ravulizumab-cwvz | Ultomiris®
Approved drug Immunopharmacology Ligand
ravulizumab is an approved drug (FDA (2018), EMA (2019))
Compound class: Antibody
Comment: Ravulizumab (ALXN1210; Ultomiris) is a humanized monoclonal antibody that binds to human complement component C5. It was developed by Alexion as a next-generation anti-C5 therapy for paroxysmal nocturnal hemoglobinuria (PNH) that was engineered from their already approved PNH agent eculizumab (Soliris), but was designed to have an inherently longer circulating half-life so as to extend dosing intervals to once/month [3].
Peptide sequence and secondary structural information for this antibody are available from its IMGT/mAb-DB record.
No information available.
Summary of Clinical Use Click here for help
Ravulizumab held EMA orphan drug designation to treat PNH patients (granted in 2016) and FDA orphan status for atypical hemolytic uremic syndrome (aHUS; granted in 2017). Therapeutic efficacy in Phase 1b/2 clinical trial was reported in 2018 [2]. FDA full approval for PNH was granted in December 2018, and EMA approval for this indication followed in July 2019. Click here to link to ClinicalTrials.gov's full list of ravulizumab (ALXN1210) trials. Direct comparison with eculizumab in C5-inhibitor-experienced adult patients with PNH has also been reported and indicates that switching from eculizumab to ravulizumab is safe and that treatment remains effective [1].

Motor neuron disease
Dysregulated complement activation is recognised as a key pathogenic driver in a wide range of immune-mediated and inflammatory diseases, including haematological and ocular pathologies, cancer and ageing-related neuroinflammatory and neurodegenerative disorders. Having already committed to testing ravulizumab as a therapy for neuromyelitis optica-driven inflammation and demyelination (in clinical trial NCT04201262), Alexion have declared their intention to evaluate ravulizumab for efficacy in slowing MND-associated neurodegeneration (in a press release Jan 14, 2020 which refers to the Phase 3 CHAMPION-ALS study).

SARS-CoV-2 and COVID-19: Ravulizumab has been entered in to clinical trials that aim to determine its ability to combat the dysregulated immune response that drives organ damage (lung and other organs) in patients with severe COVID-19.
Clinical Trials
Clinical Trial ID Title Type Source Comment References
NCT04369469 Efficacy and Safety Study of IV Ravulizumab in Patients With COVID-19 Severe Pneumonia Phase 3 Interventional Alexion Pharmaceuticals
NCT04390464 mulTi-Arm Therapeutic Study in Pre-ICu Patients Admitted With Covid-19 - Repurposed Drugs (TACTIC-R) Phase 4 Interventional Cambridge University Hospitals NHS Foundation Trust
External links Click here for help