What the FDA decided on Lirafugratinib for Cholangiocarcinoma
The FDA approved Lirafugratinib (Lyrfigtu) for cholangiocarcinoma (FGFR2 fusion).
Approved September 23, 2026 as Lyrfigtu, a few days ahead of its late September action date, for adults with previously treated unresectable, locally advanced or metastatic cholangiocarcinoma with an FGFR2 gene fusion or other rearrangement. It is the fourth FGFR drug approved for this cancer (infigratinib was later withdrawn) and the first designed to block FGFR2 more selectively than the others.
The approval rests on the 116-patient REFOCUS cohort (NCT04526106): 46% of patients responded and responses lasted a median 11.8 months. The label carries warnings for eye toxicity, high blood phosphate with soft tissue mineralization, and harm to a developing baby. The FDA's notice calls it an approval rather than an accelerated approval, the pathway used for all 3 earlier FGFR drugs in this cancer.
Who the Lirafugratinib decision matters to
Cholangiocarcinoma is a rare and aggressive cancer that forms in the bile ducts, the thin tubes that carry digestive fluid (bile) from the liver to the small intestine. It can occur inside the liver (intrahepatic), at the junction where bile ducts exit the liver (perihilar), or in the bile ducts outside the liver (distal).
It is often diagnosed at an advanced stage because symptoms appear late.
Prevalence: About 8,000 new cases per year in the U.S.; rising incidence worldwide. See our full Cholangiocarcinoma page for current treatments, recruiting trials, and community resources.
How Lirafugratinib works
Lirafugratinib: selective, irreversible (covalent) FGFR2 inhibitor designed to spare FGFR1 and FGFR4. Our full Lirafugratinib profile covers how it works, side effects in plain language, and its trial history.
Where Cholangiocarcinoma treatment stands today
Read our full Cholangiocarcinoma treatment summary
Comprehensive genomic profiling is strongly recommended for all cholangiocarcinoma patients. About 10-20% of intrahepatic CCA has FGFR2 fusions (treatable with pemigatinib, futibatinib, or lirafugratinib, approved as Lyrfigtu in September 2026), and a minority has IDH1 mutations (treatable with ivosidenib).
Clinical trials are actively evaluating new targeted therapies, immunotherapy combinations, and novel approaches.
The Cholangiocarcinoma Foundation is an excellent resource for finding trials and connecting with specialists. Many academic cancer centers have dedicated hepatobiliary programs with expertise in CCA. Getting a second opinion at a specialized center early in your diagnosis can significantly impact treatment options.
The Cholangiocarcinoma page has every treatment and trial →Meanwhile, 244 Cholangiocarcinoma studies are open or about to open
The FDA's decision does not end research on Cholangiocarcinoma. The most recently updated:
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- Trial Alerts, when a new trial opens or a trial changes
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Frequently asked questions
Has the FDA decided on Lirafugratinib?
The FDA has already decided. It approved the application.
Details: the outcome and what it means are at the top of this page.
Source: FDA approval notice
What was Lirafugratinib approved for?
The FDA approved Elevar Therapeutics' NDA for Lirafugratinib in Cholangiocarcinoma (FGFR2 fusion).
What to know: Approved September 23, 2026 as Lyrfigtu, a few days ahead of its late September action date, for adults with previously treated unresectable, locally advanced or metastatic cholangiocarcinoma with an FGFR2 gene fusion or other rearrangement. It is the fourth FGFR drug approved for this cancer (infigratinib was later withdrawn) and the first designed to block FGFR2 more selectively than the others. The approval rests on the 116-patient REFOCUS cohort (NCT04526106): 46% of patients responded and responses lasted a median 11.8 months. The label carries warnings for eye toxicity, high blood phosphate with soft tissue mineralization, and harm to a developing baby. The FDA's notice calls it an approval rather than an accelerated approval, the pathway used for all 3 earlier FGFR drugs in this cancer.
How it works: Selective, irreversible (covalent) FGFR2 inhibitor designed to spare FGFR1 and FGFR4.
More: the Lirafugratinib drug page explains it in plain language.
Is there already an FDA-approved treatment for Cholangiocarcinoma?
Yes. Trial Friend's drug list includes 7 FDA-approved drugs for Cholangiocarcinoma.
Approved: Tibsovo (ivosidenib), Pemazyre (pemigatinib), Imfinzi (durvalumab), Lytgobi (futibatinib), Keytruda (pembrolizumab), Ziihera (zanidatamab), Lyrfigtu (lirafugratinib).
Check: the Cholangiocarcinoma page also searches FDA drug labels for approved treatments.
What did the Lirafugratinib trials show?
ReFocus (NCT04526106) is a Phase 1/2 study that started September 2, 2020 and enrolled 490 patients across cholangiocarcinoma and other FGFR2-altered cancers. Its pivotal cohort enrolled 116 adults with FGFR2 fusion or rearrangement cholangiocarcinoma who had had at least 1 prior treatment but never an FGFR inhibitor.
More: the Lirafugratinib drug page has the full trial history and sources.
What are the side effects of Lirafugratinib?
In the 116-patient pivotal ReFocus cohort, the most common serious (grade 3 or higher) side effects were the on-target skin and mouth effects of blocking FGFR2: hand-foot syndrome (palmar-plantar erythrodysesthesia, painful redness and peeling on the palms and soles) in 32.8% and mouth sores (stomatitis) in 12.1%. Side effects led to a dose reduction in 75.9% of patients and a pause in 82.8%, but only 4.3% stopped the drug because of them.
More: the Lirafugratinib drug page has the label's warnings in full.
How much will Lirafugratinib cost?
Trial Friend does not list a price for Lirafugratinib. What you pay depends on your insurance and on help from the drugmaker or a charity fund.
Find help: the patient assistance finder lists programs by insurance type.
What happens after the Lirafugratinib decision?
The FDA approved Lirafugratinib for Cholangiocarcinoma (FGFR2 fusion) on September 23, 2026.
Next steps: getting the drug still takes time. Specialty pharmacy setup, insurance review and patient assistance often take weeks. Our guide covers what families can do on day 1.
Where this outcome comes from
The outcome on this page comes from FDA approval notice. This page rechecks against our calendar, which is re-verified daily.