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Lirafugratinib (Lyrfigtu)

Previously treated FGFR2 fusion or rearrangement-positive cholangiocarcinoma

Also known as RLY-4008
Regulatory submission
NDA submitted January 2026
Last updated
158 sources

Economic information and modeling reportBudget impact model

Epidemiology and eligible population inputs

11 evidence topics · 13 sources

Budget impact model of lirafugratinib: eligible population inputs

No evidence found.

Manufacturer estimate of the United States population with FGFR2-mediated cancers

“We believe FGFR2-mediated cancers affect approximately 11,000 late-line patients annually in the United States.” (opens the source at this quote in a new tab)

“In the future, if lirafugratinib advances to earlier lines of treatment, we believe it could potentially address approximately 35,000 patients annually in the United States.” (opens the source at this quote in a new tab)

“These numbers reflect the inclusion of patients with additional FGFR2 gene fusions and rearrangements that result from truncation of the protein at exon 18 based on recently published research suggesting that patients with these truncations should be considered for FGFR-targeted therapies.” (opens the source at this quote in a new tab)

Frequency of FGFR2 fusions or rearrangements used for eligible population sizing

“The FGFR2 fusions or rearrangements are found in 10% to 20% of patients with intrahepatic CCA, while they rarely occur in extrahepatic CCA.” (opens the source at this quote in a new tab)

“Of intrahepatic CCAs (iCCAs), ≤10% harbor fibroblast growth factor receptor-2 (FGFR2) fusions or rearrangements.” (opens the source at this quote in a new tab)

“this study is likely to have included patients with iCCA harboring FGFR2 alterations based on the reimbursement of pemigatinib, a subgroup that comprises ∼10%-15% of all iCCA cases” (opens the source at this quote in a new tab)

FGFR2 fusion prevalence and incidence inputs in the Taiwanese cost-effectiveness analysis

“According to the Taiwan Cancer Registry annual report, there were 1045 new incidences of ICC in 2019” (opens the source at this quote in a new tab)

“The proportion of FGFR2 fusions among Taiwanese patients was assumed to be 7.7% (beta distribution)” (opens the source at this quote in a new tab)

“Among patients with ICC, more than 90% did not have FGFR2 fusions, and therefore, the CEA result of using pemigatinib for patients with FGFR fusions in our study was highly dependent on the CEA of mFOLFOX for patients without FGFR2 fusions.” (opens the source at this quote in a new tab)

Eligible population assumptions in the Canadian budget impact reanalysis of pemigatinib

“CDA-AMC conducted a reanalysis that included: increasing pemigatinib uptake, changing the relative dose intensity to 100%, using the growth rate associated with intrahepatic CCA, removing market shares for clinical trials, assuming 85% of patients were diagnosed and unresectable and using component mFOLFOX prices sourced from DeltaPA.” (opens the source at this quote in a new tab)

Share of patients with advanced biliary tract cancer who reach second-line therapy in United States claims data

“Of 413 patients, 84.5% received gemcitabine-based first-line (1L) treatment, 46% received second-line treatment, and 16.5% received third-line (3L) treatment.” (opens the source at this quote in a new tab)

“A total of 514 patients (39.6%) died during the follow-up period without receiving a second-line therapy.” (opens the source at this quote in a new tab)

Incidence of intrahepatic cholangiocarcinoma in the SEER 22 registries, 2000 to 2020

“In this analysis of 28,918 iCCA patients, the AAIR increased from 0.49 per 100,000 in 2000 to 1.38 in 2020 [annual percent change (APC) 6.94, 95% confidence interval (CI): 6.32 to 7.56], with a notable decline from 2019 to 2020.” (opens the source at this quote in a new tab)

“The AAIR per 100,000 was 0.49 (95% CI: 0.45–0.53) in 2000, 1.39 (95% CI: 1.33–1.44) in 2017, 1.32 (95% CI: 1.26–1.37) in 2018, 1.43 (95% CI: 1.38–1.49) in 2019, and 1.38 (95% CI: 1.32–1.43) in 2020.” (opens the source at this quote in a new tab)

“Incidence rates for 2000–2020 were age-standardized to the 2000 US population in 5-year age groups.” (opens the source at this quote in a new tab)

Age range and age adjustment of the SEER 18 incidence rates for cholangiocarcinoma

“Patients were at least 18 years old at diagnosis and had iCCA (ICD-O-3 topography code C22.1) or eCCA (ICD-O-3 topography codes C24.0, C24.1, and C24.9).” (opens the source at this quote in a new tab)

“Incidence rates (per 100 000 person-years [p-y]) were age-adjusted to the US Census bureau’s population for the year 2000.” (opens the source at this quote in a new tab)

Advanced disease, palliative systemic chemotherapy, and genomic profiling in intrahepatic cholangiocarcinoma at 8 United States centers
Measure in patients with intrahepatic cholangiocarcinomaValue
Total number of patients with advanced disease (locally advanced, primary metastatic, recurrent metastatic)“543 (88.9%)”
Treated with palliative systemic chemotherapy“388/589 (65.9%)”

“Genomic profiling data were available for 64.3% (n = 349/543) of patients with advanced ICC and 50.3% (n = 92/183) of patients with advanced ECC (P < .001) (Figure 1).” (opens the source at this quote in a new tab)

Eligibility for resection at presentation and recurrence after resection

“Most patients with CCA have metastatic or locally advanced (that is, unresectable) disease at presentation, and only ∼25% are eligible for resection” (opens the source at this quote in a new tab)

“Despite the importance of surgical resection to overall prognosis, recurrence is common, occurring in up to 75% of patients after hepatectomy at 5 years, highlighting the importance of developing multimodality therapeutic approaches in order to improve the long-term outcomes for this aggressive cancer” (opens the source at this quote in a new tab)

Receipt of second-line therapy among patients with advanced cholangiocarcinoma and FGFR2 fusions or rearrangements in a United States clinico-genomic database

“This study used the nationwide (US-based) de-identified Flatiron Health-Foundation Medicine clinico-genomic database.” (opens the source at this quote in a new tab)

“As of May 2020, 571 patients met the inclusion criteria; 75 patients with FGFR2 fusions/rearrangements (median age 63 years; 64% female; 95% iCCA; 68% stage IV at initial diagnosis), and 496 patients with WT FGFR2 (median age 65 years; 48% female; 74% iCCA; 55% stage IV at initial diagnosis).” (opens the source at this quote in a new tab)

“50 patients with FGFR2 fusions/rearrangements (median age 62 years; 70% female; 94% iCCA; 70% stage IV at diagnosis) received second-line therapy.” (opens the source at this quote in a new tab)

Patients with intrahepatic cholangiocarcinoma and FGFR2 fusions evaluable for first-line and second-line treatment at one United States center
Treatment lineEvaluable patients with FGFR2 fusions, n
First-line treatment“15”
Second-line treatment“11”