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).[8][7]
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).[9] 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.[8] Ravulizumab, is a monoclonal antibody (a type of protein) designed to attach to the C5 protein, which is part of the complement system.[8] By attaching to the C5 protein, the medicine blocks its effect and thereby reduces the destruction of red blood cells.[8]
^"FDA-sourced list of all drugs with black box warnings (Use Download Full Results and View Query links.)". nctr-crs.fda.gov. FDA. Retrieved 22 October 2023.
^"Ultomiris". Therapeutic Goods Administration (TGA). 26 May 2022. Retrieved 9 March 2024.
^"Ultomiris Product information". Health Canada. Retrieved 29 May 2022.
^"Summary Basis of Decision (SBD) for Ultomiris". Health Canada. 23 October 2014. Retrieved 29 May 2022.
^"Regulatory Decision Summary for Ultomiris". Drug and Health Products Portal. 6 January 2023. Retrieved 1 April 2024.
^"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.
^ ab"Ultomiris- ravulizumab solution, concentrate". DailyMed. 20 March 2020. Retrieved 1 May 2020.
^ abcdeCite error: The named reference Ultomiris EPAR was invoked but never defined (see the help page).
^Cite error: The named reference FDA PR 20210607 was invoked but never defined (see the help page).
Ravulizumab, sold under the brand name Ultomiris, is a humanized monoclonal antibody complement inhibitor medication designed for the treatment of paroxysmal...
United States in March 2024. Danicopan is indicated as add-on therapy to ravulizumab or eculizumab for the treatment of extravascular hemolysis in adults...
to treat rare diseases. Its products include eculizumab (Soliris) and ravulizumab (Ultomiris), both used to treat the rare disorders of atypical hemolytic...
antibodies.: 1933 Prior to availability of eculizumab (Soliris) and ravulizumab (Ultomiris), an estimated 33–40% of patients died or developed end-stage...
phase 3 trials comparing iptacopan to anti-C5 therapy (eculizumab or ravulizumab), iptacopan treatment was superior to anti-C5 treatments in patients...
Global Information