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Fabhalta (iptacopan)
Australian Prescription Medicine Decision Summary
Published
Product name
Fabhalta
Active ingredient
Iptacopan
Submission type
Type A - New chemical entity
Decision
Approved
Decision date
Registration date
What this medicine was approved for
Fabhalta (iptacopan) has been approved for the treatment of adult patients with paroxysmal nocturnal haemoglobinuria.
How this medicine works
Iptacopan is a complement inhibitor. The complement system (or 'complement cascade') is a part of the immune system. It consists of inactive proteins in the blood which, upon receiving an appropriate trigger, initiate signals which result in activation of the complement cascade. This stimulates phagocytes (cells that engulf and kill microbes/microbe infected cells), to clear cellular debris, induce inflammation to attract other immune cells, and activate the 'membrane attack complex' which punches holes in the membranes of infected cells or microbes, facilitating their destruction. In paroxysmal nocturnal haemoglobinuria, red blood cell destruction within blood vessels is mediated by this membrane attack complex; other complement components induce destruction of red blood cells outside the blood vessels. Iptacopan selectively targets Factor B; Factor B activates red cell death within and outside blood vessels. Inhibition of Factor B by iptacopan, and therefore the complement pathways it activates, prevents red blood cell destruction. Complement inhibition is how iptacopan mediates its therapeutic effect of preventing red blood cell death.
Why the TGA approved or did not approve this medicine
An extensive set of quality, nonclinical pharmacology, clinical pharmacology, pharmacokinetic and toxicology studies were performed with iptacopan to demonstrate the integrity of the drug product and the effectiveness and safety of iptacopan in paroxysmal nocturnal haemoglobinuria.
The key clinical trials which demonstrated the effectiveness and safety of iptacopan were:
- A pivotal phase III study: a randomised, active-controlled study, comparing iptacopan monotherapy to anti-C5 treatment (C5 is a complement component) in paroxysmal nocturnal haemoglobinuria patients with residual anaemia despite prior anti-C5 therapy.
- A supportive Phase III study: a single arm, open-label study, evaluating iptacopan monotherapy in PNH patients who were naïve to complement inhibitor treatment.
These studies (and others) demonstrated the persistence of efficacy (and feasibility for long-term use) of iptacopan for up to 3 years.
Consumer Medicine Information (CMI)
The CMI leaflet offers guidance for consumers to support safe and effective use of the medicine. The CMI includes information on dose, how to use the medicine properly, potential side effects, safety precautions, storage instructions and more.
The Australian CMIs can be accessed through the ARTG details hyperlinks below.
Product Information (PI)
The PI document provides essential prescribing information for health professionals, including details on dosage recommendations, pregnancy category, contraindications, precautions and potential side effects.
The Australian PIs can be accessed through the ARTG details hyperlinks below.
Other resources
For health advice and information, including a symptom checker and service finder refer to the healthdirect website.
For advice on prescription medicines, over the counter medicines and other medicines (including complementary medicines) call Medicines Line.
For information on medicines subsidised by the Australian Government refer to the Pharmaceutical Benefits Scheme (PBS) website.
For data and reports on health and welfare topics in Australia refer to the Australian Institute of Health and Welfare website.