Fabhalta
Authorised
This medicine is authorised for use in the European Union
iptacopan
Medicine
Human
Authorised
- Application under evaluation
- CHMP opinion
- European Commission decision
Overview
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.
Fabhalta is available as capsules to be taken by mouth twice a day.
If one or more doses are missed, the medicine should be taken as soon as possible. In patients with PNH, if multiple doses are missed, patients should be monitored for signs and symptoms of haemolysis (an abnormal breakdown of red blood cells).
The medicine can only be obtained with a prescription.
For more information about using Fabhalta, see the package leaflet or contact your doctor or pharmacist.
The complement system is a group of proteins that is part of the immune system (the body’s natural defences). In patients with PNH and C3G, the complement system is over-active, damaging the patients’ own cells, particularly red blood cells in PNH and kidney cells in C3G.
The active substance in Fabhalta, iptacopan, blocks a protein of the complement system called ‘factor B’. By blocking factor B, Fabhalta prevents the complement system from damaging red blood cells in PNH and kidney cells in C3G, thereby helping to relieve the symptoms of these diseases.
PNH
Fabhalta was shown to be effective at increasing haemoglobin levels and reducing the need for blood transfusions in one main study involving 97 patients with PNH.
Patients in the study had been previously treated with ravulizumab or eculizumab (other medicines for PNH) for at least 6 months and still had anaemia. Patients took either Fabhalta or continued their treatment with ravulizumab or eculizumab. After 24 weeks of treatment, the percentage of patients who achieved an increase in haemoglobin levels of at least 2 g/dL without blood transfusions was around 82% for patients on Fabhalta, compared with 2% of patients continuing on ravulizumab or eculizumab. Around 69% of patients taking Fabhalta achieved haemoglobin levels of at least 12 g/dL without blood transfusions, compared with around 2% of the patients taking ravulizumab or eculizumab.
Data from an additional study supported the use of Fabhalta in patients with PNH who had not been previously treated.
C3G
Fabhalta was shown to be more effective than placebo at reducing kidney damage in one main study involving 74 patients with C3G. Patients in the study were already treated for C3G with other medicines such as RAS inhibitors and immunosuppressants (corticosteroids, mycophenolate mofetil or mycophenolate sodium).
The main measure of effectiveness was protein levels in the urine of patients. As healthy kidneys keep nearly all proteins in the blood, the presence of proteins in urine indicates kidney damage.
After 6 months of treatment, patients given Fabhalta had an approximately 35% reduction in their urine protein levels compared with those given placebo. This effect was maintained after 12 months of treatment.
For the full list of side effects and restrictions with Fabhalta, see the package leaflet.
In patients with PNH, the most common side effects with Fabhalta (which may affect more than 1 in 10 people) include upper respiratory tract (nose and throat) infection, headache and diarrhoea. The most common serious side effect with Fabhalta is urinary tract infection (infection of the parts of the body that collect and pass out urine), which may affect up to 1 in 10 patients.
In patients with C3G, the most common side effects with Fabhalta (which may affect more than 1 in 10 people) include upper respiratory tract infection. The most common serious side effect is infection with pneumococcal bacteria, which may affect up to 1 in 10 people.
Based on how Fabhalta works, it may increase the risk of infections. Fabhalta must not be used by patients who have an ongoing infection caused by so-called encapsulated bacteria, including Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae type B. It must also not be used by patients who are not currently vaccinated against N. meningitidis and S.pneumoniae unless the risk of delaying treatment outweighs the risk of developing an infection from these bacteria.
Fabhalta was shown to be effective at increasing haemoglobin levels and reducing the need for blood transfusions in patients with PNH. It was also shown to reduce the accumulation of proteins in the urine of patients with C3G, which suggests reduced kidney damage. The most common side effects are considered inconvenient but are not expected to pose a risk to patients. The European Medicines Agency therefore decided that Fabhalta’s benefits are greater than its risks and that it can be authorised for use in the EU.
The company that markets Fabhalta will provide doctors and patients with educational materials on the risk of infection by encapsulated bacteria and the need for patients to receive appropriate vaccination. For patients with PNH, the materials also include information on the risk of serious haemolysis when stopping treatment with Fabhalta.
Recommendations and precautions to be followed by healthcare professionals and patients for the safe and effective use of Fabhalta have also been included in the summary of product characteristics and the package leaflet.
As for all medicines, data on the use of Fabhalta are continuously monitored. Suspected side effects reported with Fabhalta are carefully evaluated and any necessary action taken to protect patients.
Fabhalta received a marketing authorisation valid throughout the EU on 17 May 2024.
Product information
Latest procedure affecting product information:II/0001
27/02/2025
This medicine’s product information is available in all official EU languages.
Select 'available languages' to access the language you need.
Product information documents contain:
- summary of product characteristics (annex I);
- manufacturing authorisation holder responsible for batch release (annex IIA);
- conditions of the marketing authorisation (annex IIB);
- labelling (annex IIIA);
- package leaflet (annex IIIB).
Product details
- Name of medicine
- Fabhalta
- Active substance
- iptacopan hydrochloride monohydrate
- International non-proprietary name (INN) or common name
- iptacopan
- Therapeutic area (MeSH)
- Hemoglobinuria, Paroxysmal
- Anatomical therapeutic chemical (ATC) code
- L04A
Pharmacotherapeutic group
ImmunosuppressantsTherapeutic 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).
News on Fabhalta
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