Aspaveli

Aspaveli is a medicine used to treat: - adults with paroxysmal nocturnal haemoglobinuria (PNH), an acquired disease in which there is excessive breakdown of red blood cells (haemolysis), leading to large amounts of haemoglobin (the protein in red blood cells that carries oxygen around the body) being released into the urine. Aspaveli is used in patients with PNH who have anaemia (low levels of red blood cells) due to haemolysis; - adults and adolescents aged 12 to 17 years with complement 3 glomerulopathy (C3G) or primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN), either in combination with a RAS inhibitor (a medicine that acts on the renin-angiotensin system) or alone in patients who cannot take a RAS inhibitor. C3G and IC-MPGN are diseases in which the immune system mistakenly damages the kidney’s filtering units; this prevents the kidneys from filtering the blood correctly, leading to toxin build-up, reduced urine production and swelling. PNH, C3G and IC-MPGN are rare, and Aspaveli was designated an ‘orphan medicine’ (a medicine used in rare diseases). Further information on the orphan designations can be found on the EMA website (PNH: [22 May 2017](https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1873); C3G with or without immune complexes: [10 November 2022](https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-22-2716)). Aspaveli contains the active substance pegcetacoplan.