Futibatinib

Futibatinib is a small molecule kinase inhibitor of FGFR 1, 2, 3, and 4 with IC 50 values of less than 4 nM. Futibatinib covalently binds FGFR.

• LYTGOBI is indicated for the treatment of adult patients with previously treated, unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma harboring fibroblast growth factor receptor 2 (FGFR2) gene fusions or other rearrangements [see Dosage and Administration (2.1) ] . This indication is approved under accelerated approval based on overall response rate and duration of response [see Clinical Studies (14.1) ] .

• None.contraindicated

Confirm the presence of an FGFR2 gene fusion or other rearrangement prior to initiation of treatment with LYTGOBI. ( 2.1 ) Recommended dose is 20 mg orally (five 4 mg tablets or one 16 mg tablet and one 4 mg tablet) once daily until disease progression or unacceptable toxicity occurs. ( 2.2 ) Swallow tablet whole, with or without food. ( 2.2 ) 2.1 Patient Selection Select patients for the treatment of unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma with LYTGOBI based on the presence of an FGFR2 gene fusion or rearrangement [see Clinical Studies (14.1) ]. An FDA-approved test for detection of FGFR2 gene fusions or other rearrangements in patients with unresectable, locally advanced, or metastatic intrahepatic cholangiocarcinoma for selecting patients for treatment with LYTGOBI is not available. 2.2 Recommended Dosage The recommended dosage of LYTGOBI is 20 mg (five 4 mg tablets or one 16 mg and one 4 mg tablet) taken orally once daily until disease progression or unacceptable toxicity occurs. Take LYTGOBI with or without food at approximately the same time each day [see Clinical Pharmacology (12.3) ] . Swallow tablets whole. Do not crush, chew, split, or dissolve tablets. If the patient misses a dose of LYTGOBI for more than 12 hours or if vomiting occurs, resume dosing with the next scheduled dose. 2.3 Dosage Modification for Adverse Reactions The recommended dose reductions for adverse reactions are provided in Table 1 .

Ocular Toxicity: LYTGOBI can cause retinal pigment epithelial detachment (RPED). Perform a comprehensive ophthalmological examination including optical coherence tomography (OCT) prior to initiation of therapy, every 2 months for the first 6 months, and every 3 months thereafter and urgently at any time for visual symptoms. ( 2.3 , 5.1 ) Hyperphosphatemia and Soft Tissue Mineralization: Increases in phosphate levels can cause hyperphosphatemia leading to soft tissue mineralization, calcinosis, nonuremic calciphylaxis and vascular calcification. Monitor for hyperphosphatemia and withhold, reduce the dose, or permanently discontinue based on duration and severity of hyperphosphatemia. ( 2.3 , 5.2 ) Embryo-Fetal Toxicity: Can cause fetal harm. Advise patients of reproductive potential of the potential risk to the fetus and to use effective contraception. ( 5.3 , 8.1 , 8.3 ) 5.1 Ocular Toxicity Retinal Pigment Epithelial Detachment (RPED) LYTGOBI can cause RPED, which may cause symptoms such as blurred vision. Among 318 patients who received LYTGOBI across clinical trials [see Adverse Reactions (6.1) ] where ophthalmologic monitoring did not routinely include optical coherence tomography (OCT), RPED occurred in 9% of patients. The median time to first onset of RPED was 40 days. RPED led to dose interruption of LYTGOBI in 1.3% of patients, dose reduction in 1.6% of patients, and permanent discontinuation in 0.3% of patients.

The following adverse reactions are discussed elsewhere in the labeling: Ocular Toxicity [see Warnings and Precautions (5.1) ] Hyperphosphatemia and Soft Tissue Mineralization [see Warnings and Precautions (5.2) ] Most common (≥20%) adverse reactions were nail toxicity, musculoskeletal pain, constipation, diarrhea, fatigue, dry mouth, alopecia, stomatitis, abdominal pain, dry skin, arthralgia, dysgeusia, dry eye, nausea, decreased appetite, urinary tract infection, palmar-plantar erythrodysesthesia syndrome, and vomiting. ( 6.1 ) Most common laboratory abnormalities (≥20%) were increased phosphate, increased creatinine, decreased hemoglobin, increased glucose, increased calcium, decreased sodium, decreased phosphate, increased alanine aminotransferase, increased alkaline phosphatase, decreased lymphocytes, increased aspartate aminotransferase, decreased platelets, increased activated partial thromboplastin time, decreased leukocytes, decreased albumin, decreased neutrophils, increased creatine kinase, increased bilirubin, decreased glucose, increased prothrombin international normalized ratio, and decreased potassium. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Taiho Oncology Inc. at 1-844-878-2446 or FDA at 1-800-FDA-1088 or http://www.fda.gov/medwatch . 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice.

Lactation: Advise not to breastfeed. ( 8.2 ) 8.1 Pregnancy Risk Summary Based on findings in an animal study and its mechanism of action, LYTGOBI can cause fetal harm or loss of pregnancy when administered to a pregnant woman [see Clinical Pharmacology (12.1) ] . There are no available data on the use of LYTGOBI in pregnant women. Oral administration of futibatinib to pregnant rats during the period of organogenesis at maternal plasma exposures below the human exposure at the clinical dose of 20 mg resulted in fetal malformations, fetal growth retardation, and embryo-fetal death ( see Data ). Advise pregnant women of the potential risk to a fetus. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively. Data Animal Data Once daily oral administration of futibatinib to pregnant rats during the period of organogenesis resulted in 100% embryofetal mortality due to post-implantation loss at doses ≥10 mg/kg (approximately 5 times the recommended clinical dose of 20 mg based on body surface area, BSA).

Metabolism

The pharmacokinetics of futibatinib were observed at steady state in patients with advanced solid tumors unless otherwise specified at the approved recommended dosage and are presented as mean (%CV) unless otherwise specified. Futibatinib maximum concentration (C max ) is 144 mg/mL (50%) and systemic exposure (AUC) is 790 ng∙hr/mL (45%).

• Sep 30, 2022NDANDA214801Taiho Oncology

1. 1Diarrhoea3318%
2. 2Death3116%
3. 3Disease Progression3016%
4. 4Hyperphosphataemia2513%
5. 5Fatigue2413%
6. 6Nausea2312%
7. 7Dry Mouth2111%
8. 8Off Label Use1910%
9. 9Dry Eye168.5%
10. 10Vomiting168.5%
11. 11Constipation147.4%
12. 12Neuropathy Peripheral147.4%
13. 13Asthenia136.9%
14. 14Pain In Extremity136.9%
15. 15Blood Phosphorus Increased126.4%

The 10 most recently updated of 23 ClinicalTrials.gov registrations naming Futibatinib as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• Preoperative Durvalumab, Cisplatin, Gemcitabine With or Without Futibatinib Targeted Therapy for the Treatment of Resectable Intrahepatic Cholangiocarcinoma, OPTIC Trial
  Not yet recruiting · Phase 2 · Interventional · 44 enrolled · Northwestern University
  NCT07639528updated 2026-06-10
• Study of the CHK1 Inhibitor BBI-355, an ecDNA-directed Therapy (ecDTx), and the RNR Inhibitor BBI-825, in Subjects With Tumors With Oncogene Amplifications
  Terminated · Phase 1 · Interventional · 85 enrolled · Boundless Bio, Inc.
  NCT05827614updated 2026-06-08
• TAPUR: Testing the Use of Food and Drug Administration (FDA) Approved Drugs That Target a Specific Abnormality in a Tumor Gene in People With Advanced Stage Cancer
  Recruiting · Phase 2 · Interventional · 4,200 enrolled · American Society of Clinical Oncology
  NCT02693535updated 2026-05-29
• Study of Futibatinib in Patients With Advanced Cholangiocarcinoma With FGFR2 Fusion or Rearrangement
  Recruiting · Phase 2 · Interventional · 120 enrolled · Taiho Oncology, Inc.
  NCT05727176updated 2026-05-28
• Futibatinib in Patients Previously Enrolled in an Antecedent Futibatinib as Monotherapy or Combination Therapy.
  Enrolling by invitation · Phase 2 · Phase 3 · Interventional · 15 enrolled · Taiho Oncology, Inc.
  NCT06506955updated 2026-05-28
• Personalized Medicine for Advanced Biliary Cancer Patients
  Recruiting · Phase 3 · Interventional · 800 enrolled · UNICANCER
  NCT05615818updated 2026-05-19
• Futibatinib With Paclitaxel and Ramucirumab for the Treatment of Locally Advanced or Unresectable Gastric, Gastroesophageal Junction, or Esophageal Adenocarcinoma
  Not yet recruiting · Phase 1 · Interventional · 18 enrolled · Northwestern University
  NCT07594548updated 2026-05-18
• Molecular Profiling of Advanced Soft-tissue Sarcomas
  Completed · Phase 3 · Interventional · 603 enrolled · Institut National de la Santé Et de la Recherche Médicale, France
  NCT03784014updated 2026-04-29
• Phase 2 Futibatinib in Combination With PD-1 Antibody Based Standard of Care in Solid Tumors
  Active not recruiting · Phase 2 · Interventional · 53 enrolled · Taiho Oncology, Inc.
  NCT05945823updated 2026-04-09
• A Phase II Study of Futibatinib and Pembrolizumab in Metastatic Microsatellite Stable Endometrial Carcinoma
  Terminated · Phase 2 · Interventional · 9 enrolled · M.D. Anderson Cancer Center
  NCT05036681updated 2026-03-27

Frequently asked questions

How does Futibatinib work?

Futibatinib is a small molecule kinase inhibitor of FGFR 1, 2, 3, and 4 with IC 50 values of less than 4 nM. Futibatinib covalently binds FGFR.

What is Futibatinib used for?

According to FDA labeling, Futibatinib carries indications including: LYTGOBI is indicated for the treatment of adult patients with previously treated, unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma harboring fibroblast growth factor receptor 2 (FGFR2) gene fusions or other rearrangements [see Dosage and Administration (2.1) ] . This indication is approved under accelerated approval based on overall response rate and duration of response [see Clinical Studies (14.1) ] .. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Futibatinib?

Futibatinib is classified as Fibroblast growth factor receptor (FGFR) tyrosine kinase inhibitors, Kinase Inhibitor, Breast Cancer Resistance Protein Inhibitors, Fibroblast Growth Factor Receptor Inhibitors, Kinase Inhibitors, P-Glycoprotein Inhibitors, Cellular Communication Alteration, Cellular Proliferation Alteration.

What are the brand names for Futibatinib?

Futibatinib is marketed under brand names including Lytgobi.

What are the contraindications for Futibatinib?

Futibatinib labeling lists contraindications including: None.. Always consult the full prescribing information and a clinician.