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NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) Recommendations for Cholangiocarcinoma

Futibatinib (LYTGOBI) is recommended as a National Comprehensive Cancer Network® (NCCN®) subsequent-line systemic therapy option for unresectable or metastatic intrahepatic or extrahepatic cholangiocarcinoma with FGFR2 fusions or rearrangements if disease progression5*†

Sample‡ treatment algorithm for subsequent-line use for CCA with FGFR2 fusions or rearrangements:

Primary Therapy for Unresectable
and Metastatic Disease

Subsequent-Line Therapy
if Disease Progression

Durvalumab +
gemcitabine + cisplatin

Futibatinib (LYTGOBI)

*NCCN Category 2A recommendation: based upon lower-level evidence, there is uniform NCCN consensus (≥85% support of the Panel) that the intervention is appropriate.
†Treatment selection depends on clinical factors, including previous treatment regimen/agent, somatic molecular testing results, and extent of liver dysfunction.
‡These treatment algorithms are examples only; other treatment options are recommended in the NCCN Guidelines.

LYTGOBI demonstrated an overall response rate (ORR) of 42% in patients with previously treated locally advanced or metastatic iCCA1

ORR: 42%

LYTGOBI demonstrated 42% overall response rate in patients with previously treated locally advanced or metastatic cholangiocarcinoma

2.5 months median time to response
(range: 0.7-7.4)

The ORR for LYTGOBI was

42%

(95% CI: 32%, 52%)

  • PR: 42%

Patients experienced a median duration of response (mDoR) of nearly 10 months with LYTGOBI1

Median DoR

9.7

Months

(95% CI: 7.6-17.1)

72%
of responders (n=31)
had responses that
lasted ≥6 months

14%
of responders (n=6)
had responses that
lasted ≥1 year

FOENIX-CCA2: Additional endpoints

LYTGOBI received accelerated approval from the FDA based on ORR and DoR in a single-arm study1

  • For this reason, a confirmatory study in cholangiocarcinoma is underway1
  • Progression-free survival, overall survival, and disease control rate were prespecified secondary endpoints that were studied in FOENIX-CCA2 and that are not reflected in the full Prescribing Information2
  • Due to potential variability in the natural history of the disease, a single-arm study may not adequately characterize these time-to-event endpoints and the results may not be interpretable
  • This data presentation is neither intended to draw conclusions regarding the efficacy of LYTGOBI nor to imply that there is a treatment effect of LYTGOBI on these time-to-event endpoints and the results should be interpreted with caution

Progression-free survival (PFS)2,3

Kaplan-Meier estimate of PFS (N=103)

Graph showing estimate of progression-free survival with LYTGOBI

Median, 9.0 mo (95% CI: 6.9, 13.1)

  • Median follow-up at time of data cutoff was 17.1 months

Overall survival (OS)2,3

Kaplan-Meier estimate of OS (N=103)

Graph showing estimate of overall survival with LYTGOBI

Median, 21.7 mo (95% CI: 14.5, Not Reached)

  • At the time of data cutoff: Median follow-up was 17.1 months; the OS data were not mature; during the study, 40 patients (39%) died following treatment discontinuation with the majority (90%) dying from disease progression.2,3

Disease control rate (DCR) (n=103)2,3a

LYTGOBI's disease control rate was 85%
  • FOENIX-CCA2 was a single-arm study2
    • In this setting, the DCR results may reflect the natural history of cholangiocarcinoma in an individual patient, rather than the direct effect of treatment
  • *DCR is the sum of complete response, partial response, and stable disease.

Supplementary results

Efficacy results at extended follow-up

At a nonprespecified follow-up analysis conducted 8 months after the primary analysis (data cutoff, May 29, 2021; median follow-up, 25.0 months), efficacy in the overall study population was maintained with2,4:

  • ORR of 41.7%
  • DCR of 82.5%
  • median DoR of 9.5 months
  • median PFS of 8.9 months
  • median OS of 20.0 months

The extended follow-up data were collected after the primary analysis and are descriptive in nature, and results should be interpreted with caution.

phase-trial

See how LYTGOBI was studied in a clinical trial
LYTGOBI® (futibatinib) safety profile icon

View the safety profile of LYTGOBI
pill

Learn about LYTGOBI dosing

CI=confidence interval; DoR=duration of response; iCCA=intrahepatic cholangiocarcinoma; mo=months; PR=partial response.

References:

1. 

LYTGOBI [package insert]. Princeton, NJ: Taiho Oncology, Inc.; 2024.

2. 

Goyal L, Meric-Bernstam F, Hollebecque A, et al. Futibatinib for FGFR2-Rearranged Intrahepatic Cholangiocarcinoma. N Engl J Med. 2023;388(3):228-239.

3. 

Goyal L, Meric-Bernstam F, Hollebecque A, et al. Primary results of phase 2 FOENIX-CCA2: the irreversible FGFR1–4 inhibitor futibatinib in intrahepatic cholangiocarcinoma with FGFR2 fusions/rearrangements. Abstract presented at: American Association for Cancer Research Annual Meeting; April 10-15, 2021, and May 17-21, 2021. Abstract CT010.

4. 

Goyal L, Meric-Bernstam F, Hollebecque A, et al. Updated results of the FOENIX-CCA2 trial: Efficacy and safety of futibatinib in intrahepatic cholangiocarcinoma (iCCA) harboring FGFR2 fusions/rearrangements. Abstract presented at ASCO Annual Meeting 2022. Abstract 4009. J Clin Oncol. 2022;40(16 suppl).

5. 

Referenced with permission from the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Biliary Tract Cancers. V.4.2024. © National Comprehensive Cancer Network, Inc. 2024. All rights reserved. Accessed September 10, 2024. To view the most recent and complete version of the guideline, go online to NCCN.org. NCCN makes no warranties of any kind whatsoever regarding their content, use or application and disclaims any responsibility for their application or use in any way.

INDICATIONS AND USAGE

INDICATION AND USAGE

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.

This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

IMPORTANT SAFETY INFORMATION

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS
  • Ocular Toxicity: LYTGOBI can cause Retinal Pigment Epithelial Detachment (RPED), which may cause symptoms such as blurred vision. RPED occurred in 9% of 318 patients who received LYTGOBI across clinical trials. 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. Perform a comprehensive ophthalmological examination, including optical coherence tomography (OCT) of the macula, prior to initiation of therapy, every 2 months for the first 6 months, and every 3 months thereafter. For onset of visual symptoms, refer patients for ophthalmologic evaluation urgently, with follow-up every 3 weeks until resolution or discontinuation of LYTGOBI. Withhold or reduce the dose of LYTGOBI as recommended. Dry Eye/Corneal Keratitis: Among 318 patients who received LYTGOBI across clinical trials, dry eye occurred in 15% of patients. Treat patients with ocular demulcents as needed.
  • Hyperphosphatemia and Soft Tissue Mineralization: LYTGOBI can cause hyperphosphatemia leading to soft tissue mineralization, calcinosis, nonuremic calciphylaxis, and vascular calcification. Hyperphosphatemia was reported in 88% of 318 patients treated with LYTGOBI across clinical trials with a median time of onset of 5 days (range 3‑117). Phosphate binders were received by 77% of patients who received LYTGOBI. Monitor for hyperphosphatemia throughout treatment. Initiate a low-phosphate diet and phosphate-lowering therapy when serum phosphate level is ≥5.5 mg/dL; initiate or intensify phosphate-lowering therapy when >7 mg/dL; reduce dose, withhold, or permanently discontinue LYTGOBI based on duration and severity of hyperphosphatemia.
  • Embryo-fetal Toxicity: Based on findings in an animal study and its mechanism of action, LYTGOBI can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to the fetus. Advise female patients of reproductive potential, and males with female partners of reproductive potential, to use effective contraception during treatment with LYTGOBI and for 1 week after the last dose.

ADVERSE REACTIONS
  • Serious adverse reactions occurred in 39% of patients receiving LYTGOBI, and in ≥2% of patients included pyrexia (3.9%), gastrointestinal hemorrhage (3.9%), ascites (2.9%), musculoskeletal pain (2.9%), and bile duct obstruction (2.9%).
  • The most common adverse reactions (≥20%) were nail toxicity (47%), musculoskeletal pain (43%), constipation (39%), diarrhea (39%), fatigue (37%), dry mouth (35%), alopecia (34%), stomatitis (30%), abdominal pain (30%), dry skin (29%), arthralgia (25%), dysgeusia (25%), dry eye (25%), nausea (24%), decreased appetite (23%), urinary tract infection (23%), palmar-plantar erythrodysesthesia syndrome (21%), and vomiting (20%).
  • The most common laboratory abnormalities (≥20%) were increased phosphate (97%), increased creatinine (58%), decreased hemoglobin (52%), increased glucose (52%), increased calcium (51%), decreased sodium (51%), decreased phosphate (50%), increased alanine aminotransferase (50%), increased alkaline phosphatase (47%), decreased lymphocytes (46%), increased aspartate aminotransferase (46%), decreased platelets (42%), increased activated partial thromboplastin time (36%), decreased leukocytes (33%), decreased albumin (31%), decreased neutrophils (31%), increased creatine kinase (31%), increased bilirubin (28%), decreased glucose (25%), increased prothrombin international normalized ratio (25%), and decreased potassium (22%).

DRUG INTERACTIONS
  • Dual P-gp and Strong CYP3A Inhibitors: Avoid concomitant use of drugs that are dual P-gp and strong CYP3A inhibitors.
  • Dual P-gp and Strong CYP3A Inducers: Avoid concomitant use of drugs that are dual P-gp and strong CYP3A inducers.

USE IN SPECIFIC POPULATIONS
  • Lactation: Because of the potential for serious adverse reactions from LYTGOBI in breastfed children, advise women not to breastfeed during treatment and for 1 week after the last dose.
  • Hepatic Impairment: Patients with hepatic impairment may have the potential for increased adverse reactions compared to patients with normal hepatic function.

Please see accompanying full Prescribing Information for complete details.

Important Safety Information

INDICATION AND USAGE

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.

This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS
  • Ocular Toxicity: LYTGOBI can cause Retinal Pigment Epithelial Detachment (RPED), which may cause symptoms such as blurred vision. RPED occurred in 9% of 318 patients who received LYTGOBI across clinical trials. 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. Perform a comprehensive ophthalmological examination, including optical coherence tomography (OCT) of the macula, prior to initiation of therapy, every 2 months for the first 6 months, and every 3 months thereafter. For onset of visual symptoms, refer patients for ophthalmologic evaluation urgently, with follow-up every 3 weeks until resolution or discontinuation of LYTGOBI. Withhold or reduce the dose of LYTGOBI as recommended. Dry Eye/Corneal Keratitis: Among 318 patients who received LYTGOBI across clinical trials, dry eye occurred in 15% of patients. Treat patients with ocular demulcents as needed.
  • Hyperphosphatemia and Soft Tissue Mineralization: LYTGOBI can cause hyperphosphatemia leading to soft tissue mineralization, calcinosis, nonuremic calciphylaxis, and vascular calcification. Hyperphosphatemia was reported in 88% of 318 patients treated with LYTGOBI across clinical trials with a median time of onset of 5 days (range 3‑117). Phosphate binders were received by 77% of patients who received LYTGOBI. Monitor for hyperphosphatemia throughout treatment. Initiate a low-phosphate diet and phosphate-lowering therapy when serum phosphate level is ≥5.5 mg/dL; initiate or intensify phosphate-lowering therapy when >7 mg/dL; reduce dose, withhold, or permanently discontinue LYTGOBI based on duration and severity of hyperphosphatemia.
  • Embryo-fetal Toxicity: Based on findings in an animal study and its mechanism of action, LYTGOBI can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to the fetus. Advise female patients of reproductive potential, and males with female partners of reproductive potential, to use effective contraception during treatment with LYTGOBI and for 1 week after the last dose.

ADVERSE REACTIONS
  • Serious adverse reactions occurred in 39% of patients receiving LYTGOBI, and in ≥2% of patients included pyrexia (3.9%), gastrointestinal hemorrhage (3.9%), ascites (2.9%), musculoskeletal pain (2.9%), and bile duct obstruction (2.9%).
  • The most common adverse reactions (≥20%) were nail toxicity (47%), musculoskeletal pain (43%), constipation (39%), diarrhea (39%), fatigue (37%), dry mouth (35%), alopecia (34%), stomatitis (30%), abdominal pain (30%), dry skin (29%), arthralgia (25%), dysgeusia (25%), dry eye (25%), nausea (24%), decreased appetite (23%), urinary tract infection (23%), palmar-plantar erythrodysesthesia syndrome (21%), and vomiting (20%).
  • The most common laboratory abnormalities (≥20%) were increased phosphate (97%), increased creatinine (58%), decreased hemoglobin (52%), increased glucose (52%), increased calcium (51%), decreased sodium (51%), decreased phosphate (50%), increased alanine aminotransferase (50%), increased alkaline phosphatase (47%), decreased lymphocytes (46%), increased aspartate aminotransferase (46%), decreased platelets (42%), increased activated partial thromboplastin time (36%), decreased leukocytes (33%), decreased albumin (31%), decreased neutrophils (31%), increased creatine kinase (31%), increased bilirubin (28%), decreased glucose (25%), increased prothrombin international normalized ratio (25%), and decreased potassium (22%).

DRUG INTERACTIONS
  • Dual P-gp and Strong CYP3A Inhibitors: Avoid concomitant use of drugs that are dual P-gp and strong CYP3A inhibitors.
  • Dual P-gp and Strong CYP3A Inducers: Avoid concomitant use of drugs that are dual P-gp and strong CYP3A inducers.

USE IN SPECIFIC POPULATIONS
  • Lactation: Because of the potential for serious adverse reactions from LYTGOBI in breastfed children, advise women not to breastfeed during treatment and for 1 week after the last dose.
  • Hepatic Impairment: Patients with hepatic impairment may have the potential for increased adverse reactions compared to patients with normal hepatic function.

Please see accompanying full Prescribing Information for complete details.