Lyrfigtu

Lyrfigtu (lirafugratinib) is a prescription capsule used to treat adults with a type of bile duct cancer called cholangiocarcinoma that has an FGFR2 gene fusion or other FGFR2 rearrangement.

Lyrfigtu is used when the cancer:

• is locally advanced (has grown into nearby tissue) or metastatic (has spread to other parts of the body)
• cannot be removed with surgery (unresectable)
• has already been treated with another cancer medicine

Lyrfigtu capsules are taken once daily with or without food.

Lyrfigtu is a kinase inhibitor that blocks FGFR2 (fibroblast growth factor receptor 2), a protein that can help some cancer cells grow and survive. Your doctor will test your tumor to confirm it has an FGFR2 gene fusion or rearrangement before you start treatment. An FDA-authorized test to select patients for Lyrfigtu is not currently available.

In the REFOCUS clinical trial (NCT04526106) of 116 adults with previously treated FGFR2-positive cholangiocarcinoma, 46% of people on Lyrfigtu had their tumors shrink (objective response rate). For people who responded, the response lasted a median of 11.8 months. The median time to response was 1.9 months.

Lyrfigtu received FDA approval on September 23, 2026. It is not known if Lyrfigtu is safe and effective in children.

Take Lyrfigtu by mouth once a day, with or without food, exactly as your healthcare provider tells you. You will usually keep taking it until the cancer gets worse or side effects become too severe.

• Swallow capsules whole. Do not open, break, or chew them.
• If you vomit after taking a dose, do not take another dose. Take your next dose at the usual time.
• Lyrfigtu comes in blister packs (DosePaks) sorted by daily dose, so your daily dose may be made up of more than one capsule.
• Keep all your eye exam and blood test appointments.

The recommended dose of Lyrfigtu is 70 mg taken by mouth once daily.

Your provider may pause treatment or lower your dose if you have certain side effects, including eye problems. The dose may be lowered in steps:

Lyrfigtu is stopped permanently if you cannot tolerate 20 mg once daily.

If you must take a strong CYP3A inhibitor (a type of medicine that raises Lyrfigtu levels), your provider may lower your dose to 40 mg once daily. With a moderate CYP3A inhibitor, the dose may be lowered to 50 mg once daily.

Lyrfigtu is available as 20 mg and 30 mg capsules.

Lyrfigtu can cause serious eye problems, high blood phosphate levels, and harm to an unborn baby. Lyrfigtu does not have a boxed warning.

Eye problems (ocular toxicity). Lyrfigtu can cause retinal pigment epithelial detachment, or RPED (fluid building up under the layer at the back of the eye), which may cause blurred vision. In clinical studies, RPED occurred in 31% of people, with a median time to onset of about 2 months (57 days). Lyrfigtu can also cause dry eye and inflammation of the cornea (keratitis).

• You will need a full eye exam, including a scan of the retina (optical coherence tomography, or OCT), before you start Lyrfigtu.
• Eye exams are repeated every 2 months for the first 13 months, then every 4 months.
• Tell your healthcare provider right away if you notice any change in your vision.
• Use artificial tears or lubricating eye gels to prevent or treat dry eyes.

High phosphate levels (hyperphosphatemia) and soft tissue mineralization. Lyrfigtu can raise the level of phosphate in your blood. High phosphate can lead to mineral deposits in soft tissues and blood vessels. Your provider will check your phosphate level throughout treatment. Tell your provider right away if you have muscle cramps, or numbness or tingling around your mouth.

Harm to an unborn baby (embryo-fetal toxicity). Lyrfigtu can cause harm to an unborn baby or loss of pregnancy. See the pregnancy section below for contraception advice.

Sun sensitivity (phototoxicity). Lyrfigtu may make your skin more sensitive to sunlight. Limit sun exposure and use sunscreen, protective clothing, and sunglasses.

Tell your healthcare provider about all your medical conditions, especially if you have eye or vision problems, liver problems, or are pregnant, planning a pregnancy, or breastfeeding. There are no conditions listed in the label where Lyrfigtu must never be used (no contraindications).

Tell your provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal products.

Liver problems. No dose change is needed for mild or moderate liver problems. Lyrfigtu has not been studied in people with severe liver problems.

Pregnancy. Lyrfigtu can harm an unborn baby. If you can become pregnant, your provider will check that you are not pregnant before you start treatment.

• Females who can become pregnant should use effective birth control during treatment and for 6 months after the last dose.
• Males with a female partner who can become pregnant should use effective birth control during treatment and for 3 months after the last dose.
• Tell your provider right away if you become pregnant.

Breastfeeding. Do not breastfeed during treatment and for 1 week after your last dose. It is not known if Lyrfigtu passes into breast milk.

Fertility. Based on animal studies, Lyrfigtu may affect fertility in males.

Older adults. No overall differences in safety or effectiveness were seen between people 65 years and older and younger adults.

Common Lyrfigtu side effects

The most common Lyrfigtu side effects included nail problems (89%), hand-foot syndrome (82%), mouth sores (80%), hair loss (66%), and dry eye (53%) in the REFOCUS clinical trial.

Other Common side effects reported in 20% or more of people taking Lyrfigtu included:

• dry mouth (50%)
• tiredness (43%)
• change in taste (39%)
• retinal pigment epithelial detachment (38%)
• constipation and dry skin (37% each)
• infection and rash (34% each)
• stomach pain (28%)
• bleeding, blurred vision, diarrhea, and muscle or bone pain (22% each)
• nausea (21%)
• decreased appetite (20%)

Serious side effects

Get emergency medical help if you have signs of an allergic reaction, such as hives, difficulty breathing, or swelling of your face, lips, tongue, or throat. A serious allergic reaction (anaphylaxis) was reported in the clinical trial.

Serious side effects occurred in 32% of people in the REFOCUS clinical trial. The most common serious side effects were infection, pneumonia, tiredness, and bleeding. Call your provider right away if you have:

• any change in vision, such as blurred vision
• muscle cramps, or numbness or tingling around your mouth
• signs of infection, such as fever, chills, or cough
• unusual bleeding
• painful redness, swelling, or blistering of the palms or soles
• severe mouth sores that make it hard to eat or drink

Blood test changes

Lyrfigtu commonly changed blood test results, including increased phosphate, liver enzymes, creatinine (a kidney marker), bilirubin, and glucose, and decreased sodium, red blood cells, white blood cells, and platelets. Your provider will monitor your blood during treatment.

This is not a complete list of side effects. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088 or to Elevar Therapeutics at 1-866-4ELEVAR.

If you miss a dose by less than 4 hours, take it as soon as you remember. If 4 hours or more have passed, skip the missed dose and take your next dose at the usual time. Do not take 2 doses at the same time.

Seek emergency medical attention or call the Poison Help line at 1-800-222-1222 if you think you or someone else has taken too much Lyrfigtu. The prescribing information does not describe specific overdose symptoms.

Some medicines can raise or lower the amount of Lyrfigtu in your blood, and Lyrfigtu can raise the levels of some other medicines. Tell your provider about all medicines you take, including over-the-counter medicines and herbal products.

Medicines that raise Lyrfigtu levels (strong or moderate CYP3A inhibitors). These can increase the risk of side effects. Avoid taking them with Lyrfigtu. If they cannot be avoided, your dose of Lyrfigtu will be lowered. Examples studied include itraconazole and erythromycin. Once the other medicine has been stopped for long enough, your provider may return you to your previous dose.

Medicines that lower Lyrfigtu levels (strong or moderate CYP3A inducers). These can make Lyrfigtu less effective. Avoid taking them with Lyrfigtu. Examples studied include rifampin (rifampicin) and efavirenz.

Medicines whose levels Lyrfigtu may raise. Lyrfigtu may increase levels of medicines that are moved through the body by certain transporter proteins (P-gp, BCRP, OATP1B1, or OATP1B3). Your provider may monitor you more closely for side effects of these medicines.

No important interactions were found with esomeprazole or omeprazole (stomach acid reducers). This is not a complete list of interactions.

• Store at room temperature, 68°F to 77°F (20°C to 25°C). Short periods between 59°F and 86°F (15°C and 30°C) are allowed.
• Keep capsules in the child-resistant DosePak blister packs and out of the reach of children.

Active ingredient: lirafugratinib (as lirafugratinib hydrochloride).

Inactive ingredients: colloidal silicon dioxide, croscarmellose sodium, magnesium stearate, microcrystalline cellulose, and pregelatinized starch. The capsule shell contains gelatin and titanium dioxide; the 30 mg shell also contains black iron oxide and yellow iron oxide. The printing ink contains black iron oxide, butyl alcohol, dehydrated alcohol, isopropyl alcohol, propylene glycol, potassium hydroxide, purified water, shellac, and strong ammonia solution.

What it looks like:

Distributed by Elevar Therapeutics, Inc., Fort Lee, NJ. Manufactured by Quotient Sciences, Philadelphia, LLC.