In short
Lirafugratinib (LYRFIGTU) is a kinase inhibitor authorised in the United States for adults with previously treated, unresectable, locally advanced or metastatic cholangiocarcinoma harbouring an FGFR2 fusion or other rearrangement. It is now being studied in a Phase 2 trial for other solid tumours with FGFR2 fusions or rearrangements. That trial is authorised and plans to enrol 30 participants.
Lirafugratinib is a kinase inhibitor. Its full name is lirafugratinib hydrochloride, and it is sold under the trade name LYRFIGTU. It was approved in the United States in 2026 for adults with previously treated, unresectable, locally advanced or metastatic cholangiocarcinoma harbouring an FGFR2 fusion or other rearrangement.
Key points
- Lirafugratinib (LYRFIGTU) is a kinase inhibitor approved in the US for FGFR2-altered cholangiocarcinoma.
- The new Phase 2 trial studies lirafugratinib in solid tumours other than cholangiocarcinoma with FGFR2 fusion or rearrangement.
- The trial is authorised and plans to enrol 30 participants.
- The main measure is objective response rate (ORR) assessed by an independent review committee using RECIST v1.1.
- Lirafugratinib is given as 70 mg orally once daily in the trial.
What is lirafugratinib?
Lirafugratinib is a kinase inhibitor, a type of medicine that blocks signals inside cancer cells. It is sold as LYRFIGTU capsules. It is an authorised medicine in the United States for a specific type of bile duct cancer called cholangiocarcinoma, when the tumour carries an FGFR2 gene fusion or other rearrangement.
How does lirafugratinib work?
In some tumours, the FGFR2 gene is altered by a fusion or rearrangement, which causes the FGFR2 protein to send constant growth signals. Lirafugratinib blocks this protein, which may slow or stop the growth of cancer cells that depend on it.
Which conditions is it used or studied for?
- Cholangiocarcinoma with FGFR2 fusion or rearrangement — lirafugratinib is approved in the US for adults with previously treated, unresectable, locally advanced or metastatic disease.
- Other solid tumours with FGFR2 fusion or rearrangement — a Phase 2 trial is studying lirafugratinib in previously treated, unresectable, locally advanced or metastatic solid tumours excluding cholangiocarcinoma.
How is lirafugratinib given?
Lirafugratinib is given by mouth as capsules. The approved dosage is 70 mg once daily with or without food. The capsules are available as 20 mg and 30 mg strengths. In the Phase 2 trial, lirafugratinib is given at 70 mg orally.
What do the clinical trials show?
One clinical trial is listed in the EU Clinical Trials Information System for lirafugratinib in solid tumours other than cholangiocarcinoma.
The trial is a Phase 2 study in previously treated, unresectable, locally advanced or metastatic solid tumours with FGFR2 fusion or rearrangement, excluding cholangiocarcinoma. It is authorised and plans to enrol 30 participants. The primary outcome is objective response rate (ORR), which means the proportion of participants whose tumours shrink or disappear. This is assessed by an independent review committee (IRC) using RECIST v1.1, a standard set of rules for measuring tumour response.
What side effects are known?
The most common adverse reactions reported with lirafugratinib, occurring in at least 20% of patients, include nail toxicity, palmar-plantar erythrodysesthesia syndrome (redness and pain on the palms and soles), stomatitis (mouth sores), alopecia (hair loss), dry eye, dry mouth, fatigue, dysgeusia (taste changes), retinal pigment epithelial detachment, constipation, dry skin, infection, rash, abdominal pain, haemorrhage, blurred vision, diarrhoea, musculoskeletal pain, nausea, and decreased appetite.
Serious side effects described in the prescribing information include ocular toxicity such as retinal pigment epithelial detachment and corneal toxicity, hyperphosphataemia (high phosphate levels in the blood) which can lead to soft tissue mineralisation, and embryo-foetal toxicity, meaning it can harm an unborn baby.