Fabhalta

Fabhalta is a medicine used to treat: - haemolytic anaemia in adults with paroxysmal nocturnal haemoglobinuria (PNH). PNH is a disease in which the excessive breakdown of blood cells results in anaemia (low levels of haemoglobin, the protein in red blood cells that carries oxygen around the body), thrombosis (blood clots in blood vessels), pancytopenia (low levels of blood cells) and dark urine (due to large amounts of haemoglobin being released into the urine). - complement 3 glomerulopathy (C3G) in adults, 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 is a disease in which progressive damage to the kidneys prevent them from filtering the blood correctly, leading to toxin buildup, reduced urine production and swelling. PNH and C3G are rare, and Fabhalta 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: 4 June 2020; C3G: 4 December 2018). Fabhalta contains the active substance iptacopan.