Basic Information
Ultomiris
Regulatory Information
EMEA/H/C/004954
July 2, 2019
April 26, 2019
16
November 11, 2024
Company Information
France
103-105 rue Anatole France 92300 Levallois-Perret
Alexion Europe SAS
Active Substances Detail
EMA Resources
Detailed Information
Therapeutic Indication
### Therapeutic indication Paroxysmal nocturnal haemoglobinuria (PNH) Ultomiris is indicated in the treatment of adult and paediatric patients with a body weight of 10 kg or above with PNH: \- in patients with haemolysis with clinical symptom(s) indicative of high disease activity. \- in patients who are clinically stable after having been treated with eculizumab for at least the past 6 months (see section 5.1). Atypical haemolytic uremic syndrome (aHUS) Ultomiris is indicated in the treatment of patients with a body weight of 10 kg or above with aHUS who are complement inhibitor treatment-naïve or have received eculizumab for at least 3 months and have evidence of response to eculizumab (see section 5.1). Generalized myasthenia gravis (gMG) Ultomiris is indicated as an add-on to standard therapy for the treatment of adult patients with gMG who are anti-acetylcholine receptor (AChR) antibody-positive. Neuromyelitis Optica Spectrum Disorder (NMOSD) Ultomiris is indicated in the treatment of adult patients with NMOSD who are anti-aquaporin 4 (AQP4) antibody-positive (see section 5.1). Ultomiris is indicated in the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH): \- in patients with haemolysis with clinical symptom(s) indicative of high disease activity. \- in patients who are clinically stable after having been treated with eculizumab for at least the past 6 months. Ultomiris is indicated in the treatment of adult patients with atypical haemolytic uremic syndrome (aHUS) who are complement inhibitor treatment-naïve or have received eculizumab for at least 3 months and have evidence of response to eculizumab.
Overview Summary
Ultomiris is a medicine used to treat: - adults and children weighing at least 10 kg who have paroxysmal nocturnal haemoglobinuria (PNH), a disease in which the immune system attacks and damages red blood cells, resulting in anaemia (low red blood cell counts), thrombosis (blood clots in the blood vessels), pancytopenia (low counts of blood cells) and dark urine; - adults and children weighing at least 10 kg who have atypical haemolytic uraemic syndrome (aHUS), a disease in which the immune system causes damage leading to anaemia, thrombocytopenia (a decrease in the number of platelets, components that help the blood to clot) and kidney failure; - adults with generalised myasthenia gravis (gMG), a disease in which the immune system attacks and damages receptors at the junction between nerves and muscle cells, causing muscle weakness and tiredness; - adults with neuromyelitis optica spectrum disorders (NMOSD), inflammatory disorders that affect mainly the optic nerve (which connects the eye to the brain) and the spinal cord. This leads to impaired vision, loss of sensation, loss of bladder control, weakness and paralysis of the arms and legs. In PNH, Ultomiris is used in patients who have symptoms indicating high disease activity or who have been treated with eculizumab (another medicine for PNH and aHUS) for at least the past 6 months and have evidence that the treatment is working. In aHUS, Ultomiris is used in patients who either have not received complement inhibitors (such as eculizumab) before or who have received eculizumab for at least 3 months and have evidence that the treatment is working. In gMG, Ultomiris is given with other medicines in patients whose immune system produces a specific antibody (type of protein) against a target found on muscle cells, called acetylcholine receptor. In NMOSD, Ultomiris is used in patients who have antibodies against a protein called aquaporin-4 (AQP4). Ultomiris contains the active substance ravulizumab.