Basic Information
Fabhalta
Regulatory Information
EMEA/H/C/005764
May 17, 2024
March 21, 2024
4
April 11, 2025
Company Information
Ireland
Vista Building Elm Park Merrion Road Dublin 4 D04 A9N6
NOVARTIS EUROPHARM LIMITED
Drug Classification
Active Substances Detail
EMA Resources
Detailed Information
Therapeutic Indication
### Therapeutic indication Paroxysmal nocturnal haemoglobinuria Fabhalta is indicated as monotherapy in the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who have haemolytic anaemia. Complement 3 glomerulopathy Fabhalta is indicated for the treatment of adult patients with complement 3 glomerulopathy (C3G) in combination with a renin-angiotensin system (RAS) inhibitor, or in patients who are RAS-inhibitor intolerant, or for whom a RAS inhibitor is contraindicated (see section 5.1).
Overview Summary
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.