iptacopan hydrochloride monohydrate at a glance
Medicine class
Complement inhibitor targeting Factor B in the alternative complement pathway
Licensed uses
Adult PNH with haemolytic anaemia and adult C3G
How it is taken
An oral hard capsule that may be taken with or without food
Key safety point
Vaccination lowers but does not remove the risk of serious bacterial infection
What is iptacopan hydrochloride monohydrate used for?
Licensed adult indications include:
PNH with haemolytic anaemia, used alone.
C3G alongside a renin–angiotensin system inhibitor, or where that treatment is not tolerated or is contraindicated.
How does iptacopan hydrochloride monohydrate work?
Iptacopan blocks complement Factor B, reducing activity of the alternative complement pathway. In PNH this limits complement-driven destruction of red blood cells; in C3G it reduces abnormal C3 activation and deposition within the kidney’s glomeruli.
iptacopan hydrochloride monohydrate preparations and strengths
Capsule
Route: Oral
Strengths: 200 mg
How to take iptacopan hydrochloride monohydrate
Do not stop or change treatment without speaking to the specialist team.
Swallow the hard capsule with water.
It may be taken with or without food.
Keep the patient safety card available and show it to healthcare professionals involved in your care.
iptacopan hydrochloride monohydrate side effects
Common or expected effects
- Upper respiratory tract infections
- Headache
- Diarrhoea
- Dizziness, nausea or abdominal pain
- Joint pain
- Bronchitis or urinary tract infection in people treated for PNH
- Reduced platelet count, which may cause easier bleeding or bruising
Get urgent medical advice
- Serious infection caused by encapsulated bacteria, including meningococcal, pneumococcal or Haemophilus influenzae type B infection
- Pneumonia or sepsis
- After interruption or stopping in PNH: possible haemolysis causing dark or bloody urine, marked tiredness, abdominal pain, breathlessness, swallowing difficulty or thrombosis
iptacopan hydrochloride monohydrate in pregnancy
Human pregnancy data are absent or limited. Use during pregnancy or when planning pregnancy requires specialist assessment of the benefits, uncertainties and risks from uncontrolled PNH or C3G. Tell the prescriber promptly if pregnancy occurs.
iptacopan hydrochloride monohydrate while breastfeeding
It is unknown whether iptacopan passes into human milk, and a risk to the infant cannot be excluded. A specialist should assess whether breastfeeding or treatment should be stopped.
iptacopan hydrochloride monohydrate interactions
Tell the prescriber and pharmacist about all prescribed, non-prescription and herbal products. Important potential interactions include:
Rifampicin and other strong CYP2C8, UGT1A1, P-glycoprotein, BCRP or OATP1B1/3 inducers
May reduce iptacopan exposure and effectiveness; concurrent use is not recommended.
Sensitive CYP3A4 substrates, including carbamazepine, ciclosporin, ergotamine, fentanyl, pimozide, quinidine, sirolimus and tacrolimus
Iptacopan may reduce their exposure; extra caution is required, particularly with narrow-therapeutic-index medicines.
Sensitive CYP2C8 substrates, including repaglinide, dasabuvir and paclitaxel
Iptacopan may increase their exposure, so clinical review and appropriate monitoring may be needed.
Common questions about iptacopan hydrochloride monohydrate
Answers are fully visible for fast scanning and source review.
What conditions is iptacopan hydrochloride monohydrate licensed to treat?
It is licensed for adults with PNH and haemolytic anaemia, and for adults with C3G under the conditions described in the product licence.
Why are vaccines needed?
Blocking complement weakens defence against certain encapsulated bacteria, particularly meningococcal and pneumococcal bacteria. Vaccination reduces, but does not eliminate, this risk.
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.