A targeted tablet for bile-duct cancer
Pemigatinib
A targeted tablet used to treat bile-duct cancer driven by a specific FGFR gene change.
What is Pemigatinib?
Pemigatinib is a specialist targeted cancer medicine, taken as a tablet, used to treat bile-duct cancer (cholangiocarcinoma) that carries a particular change in a gene called FGFR2 and has come back or spread. It blocks faulty FGFR signals that drive the cancer's growth. A characteristic effect is a rise in blood phosphate (hyperphosphataemia), which is managed with diet, medicines and dose changes. The most important safety issue is eye problems, including a type of retinal detachment that can blur vision, so eye checks are needed. It can also cause nail and skin changes and mouth soreness, and it can harm an unborn baby, so reliable contraception is essential.
Education and reference only. This is a plain-language guide to Pemigatinib — it deliberately contains no doses. Doses depend on the person, the brand and the reason for treatment, and belong with your prescriber. Always check the BNF, the product labelling (SmPC) and follow medical advice.
What it is
Pemigatinib is a targeted treatment for bile-duct cancer, known medically as cholangiocarcinoma, in people whose cancer carries a specific change (a fusion or rearrangement) in a gene called FGFR2. It is one of a group of medicines called FGFR inhibitors, which block faulty growth signals inside cancer cells. It is taken by mouth as a tablet, on a repeating schedule, and is used when the cancer has come back or spread after earlier treatment and has the relevant gene change. Because of its particular side effects, it is prescribed and monitored by a cancer specialist team.
How it works
In this type of bile-duct cancer, a change in the FGFR2 gene produces a faulty protein that keeps sending growth signals, telling the cancer cells to multiply. Pemigatinib blocks the FGFR proteins, switching off these abnormal signals so the cancer's growth slows. Because these signals are a key driver of cancers carrying the gene change, targeting them can hold back the disease. The same pathway also helps control phosphate in the body, which is why blocking it tends to raise blood phosphate, an effect the team manages and uses to confirm the medicine is working as expected.
Company & origin
Originated / developed by: Specialist manufacturer.
A specialist targeted cancer medicine used to treat bile-duct cancer (cholangiocarcinoma) with a specific gene change.
Practical use
How to take Pemigatinib
General, dose-free guidance — always follow your prescriber's and the leaflet's specific instructions.
- Take the tablets by mouth as prescribed, on the repeating schedule your team gives you, swallowed whole.
- Attend your blood tests for phosphate, and follow any low-phosphate diet or phosphate-lowering medicine advised.
- Attend your eye checks and report any blurring, distortion or change in vision straight away.
- Tell your team about nail or skin problems and mouth soreness, which can be managed with their help.
- Use reliable contraception during treatment and for as long as advised, as it can harm an unborn baby.
Weighing it up
Advantages & disadvantages of Pemigatinib
Advantages
- A targeted tablet for bile-duct cancer carrying a specific FGFR2 gene change.
- Taken by mouth rather than as a drip in hospital.
- Blocks a key growth signal that drives this particular type of cancer.
Disadvantages
- Commonly raises blood phosphate, needing diet, medicines and monitoring.
- Can cause eye problems, including a type of retinal detachment that blurs vision.
- Can cause nail and skin changes and mouth soreness, and can harm an unborn baby.
Practical use
Good to know
A characteristic effect of pemigatinib is a rise in blood phosphate (hyperphosphataemia), which is expected from how it works; it is managed with a low-phosphate diet, phosphate-lowering medicines and, if needed, dose changes, and is checked with regular blood tests. The most important safety concern is the eyes: it can cause a type of fluid build-up under the retina (a form of retinal detachment) that blurs or distorts vision, so you have eye checks before and during treatment and must report any change in sight promptly. It commonly causes nail changes (such as brittle, separating or inflamed nails), dry skin and hair changes, and soreness of the mouth, which the team helps you manage. It can harm an unborn baby, so reliable contraception is essential during and after treatment, and it is taken only under specialist cancer care.
Who should not take it / use with caution
- People who have had a serious allergic reaction to pemigatinib should not take it.
- It must not be used in pregnancy, as it can seriously harm an unborn baby; reliable contraception is needed.
- It is used with particular care, and extra eye monitoring, in people with existing eye problems, under specialist supervision.
Monitoring
- Regular blood tests for phosphate, with diet and medicines to manage a rise.
- Eye checks before and during treatment, with prompt review of any change in vision.
- Watching nail, skin and mouth side effects, and reviewing how the cancer responds over time.
Side effects
- A rise in blood phosphate (hyperphosphataemia), managed with diet and medicines.
- Eye problems, including a form of retinal detachment that blurs or distorts vision, and dry eyes.
- Nail changes, dry skin, hair changes, soreness of the mouth and changes in taste.
- Tiredness, diarrhoea, reduced appetite and changes in blood tests.
Key interactions
- Some medicines can change pemigatinib levels in the body, so tell your team what you take.
- Medicines and supplements that affect phosphate balance may need reviewing.
- Give a full list of all your medicines, including anything bought over the counter, to your specialist team.
Available as: Tablets taken by mouth.
Answers
Pemigatinib: frequently asked questions
What is pemigatinib used for?
It is used to treat bile-duct cancer (cholangiocarcinoma) carrying a specific change in the FGFR2 gene, blocking the faulty growth signals that drive the cancer.
Why does my blood phosphate go up?
The pathway pemigatinib blocks also helps control phosphate, so a rise in blood phosphate is expected; it is managed with diet, medicines and, if needed, dose changes, and checked with blood tests.
Why do I need eye checks?
It can cause a type of fluid build-up under the retina that blurs or distorts vision, so eye checks are done before and during treatment and any change in sight should be reported promptly.
What side effects affect the nails and skin?
It commonly causes nail changes such as brittle, separating or inflamed nails, along with dry skin, hair changes and soreness of the mouth, which your team can help manage.
Is it safe in pregnancy?
No. It can seriously harm an unborn baby, so it must not be used in pregnancy and reliable contraception is essential during and after treatment.
Authoritative sources
- BNF
- NICE CKS
Building a medicines information resource?
We create evidence-led, dose-free drug and formulary references for teams.