Efficacy and Safety of Pemigatinib in Subjects With Advanced/Metastatic or Surgically Unresectable Cholangiocarcinoma Who Failed Previous Therapy - (FIGHT-202)

ORR was defined as the percentage of participants with a best overall response of complete response (CR) or partial response (PR) at any post-Baseline visit prior to first progressive disease (PD), per Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 (v1.1). CR: disappearance of all target and non-target lesions and no appearance of any new lesions. Any pathological lymph nodes (whether target or non-target) must have a reduction in the short axis to \<10 millimeters (mm). PR: complete disappearance or at least a 30% decrease in the sum of the diameters of target lesions, taking as a reference the baseline sum diameters, no new lesions, and no progression of non-target lesions. ORR was based on central genomics laboratory results. Response was based on review of scans by an independent centralized radiological review committee.

Time frame: up to 1527 days

Population: Efficacy Evaluable Population (EEP): all participants who received ≥1 dose of pemigatinib and had a known FGF/FGFR alteration or, in the United States, were negative for FGF/FGFR alterations based on central genomics laboratory results. Two participants were assigned to a group labeled Other and were excluded from the EEP because the local laboratory FGF/FGFR results could not be confirmed centrally. Confidence intervals were calculated based on the exact method for binomial distribution.

CL/F is defined as apparent oral clearance.

Time frame: Predose; 1-2 hours post-dose; 4-12 hours post-dose

Population: PK Population

DCR was defined as the proportion of participants with an overall response of CR, PR, or stable disease (SD), per RECIST v1.1. CR: disappearance of all target and non-target lesions and no appearance of any new lesions. Any pathological lymph nodes (whether target or non-target) must have a reduction in the short axis to \<10 mm. PR: complete disappearance or at least a 30% decrease in the sum of the diameters of target lesions, taking as a reference the baseline sum diameters, no new lesions, and no progression of non-target lesions. PD: progression of a target or non-target lesion or presence of a new lesion. SD: no change in target lesions to qualify for CR, PR, or PD.

Time frame: up to 1527 days

Population: Efficacy Evaluable Population. Two participants were assigned to a group labeled Other and were excluded from the EEP because the local laboratory FGF/FGFR results could not be confirmed centrally. Confidence intervals were calculated based on the exact method for binomial distribution.

DOR was defined as the time from the first overall response contributing to an objective response (CR or PR) as assessed by an independent centralized radiological review committee to the earlier of death or first overall response of PD occurring after the first overall response contributing to the objective response. CR: disappearance of all target and non-target lesions and no appearance of any new lesions. Any pathological lymph nodes (whether target or non-target) must have a reduction in the short axis to \<10 millimeters (mm). PR: complete disappearance or at least a 30% decrease in the sum of the diameters of target lesions, taking as a reference the baseline sum diameters, no new lesions, and no progression of non-target lesions. PD: progression of a target or non-target lesion or presence of a new lesion.

Time frame: up to 47.11 months

Population: Efficacy Evaluable Population. Two participants were assigned to a group labeled Other and were excluded from the EEP because the local laboratory FGF/FGFR results could not be confirmed centrally. The 95% confidence intervals were calculated using the Brookmeyer and Crowley's method. Only participants with a CR or PR were analyzed.

First-order absorption rate constant is defined as the rate at which a drug enters into the system.

Time frame: Predose; 1-2 hours post-dose; 4-12 hours post-dose

Population: Pharmacokinetic (PK) Population: all participants contributing at least one sample for PK analysis

An adverse event (AE) was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related, that occurred after a participant provided informed consent. Abnormal laboratory values or test results occurring after informed consent constituted AEs only if they induced clinical signs or symptoms, were considered clinically meaningful, required therapy (e.g., hematologic abnormality that required transfusion), or required changes in the study drug(s). A TEAE was defined as any adverse event either reported for the first time or the worsening of a pre-existing event after the first dose of study drug and within 30 days of the last dose of study drug.

Time frame: up to 1584 days

Population: Safety Population: all enrolled participants who received at least 1 dose of pemigatinib

ORR was defined as the percentage of participants with a best overall response of CR or PR at any post-Baseline visit prior to first PD, per RECIST v1.1. CR: disappearance of all target and non-target lesions and no appearance of any new lesions. Any pathological lymph nodes (whether target or non-target) must have a reduction in the short axis to \<10 mm. PR: complete disappearance or at least a 30% decrease in the sum of the diameters of target lesions, taking as a reference the baseline sum diameters, no new lesions, and no progression of non-target lesions. ORR was based on central genomics laboratory results. Response was based on review of scans by an independent centralized radiological review committee.

Time frame: up to 1527 days

Population: Efficacy Evaluable Population. Two participants were assigned to a group labeled Other and were excluded from the EEP because the local laboratory FGF/FGFR results could not be confirmed centrally. Confidence intervals were calculated based on the exact method for binomial distribution.

ORR was defined as the percentage of participants with a best overall response of CR or PR at any post-Baseline visit prior to first PD, per RECIST v1.1. CR: disappearance of all target and non-target lesions and no appearance of any new lesions. Any pathological lymph nodes (whether target or non-target) must have a reduction in the short axis to \<10 mm. PR: complete disappearance or at least a 30% decrease in the sum of the diameters of target lesions, taking as a reference the baseline sum diameters, no new lesions, and no progression of non-target lesions. ORR was based on central genomics laboratory results. Response was based on review of scans by an independent centralized radiological review committee.

Time frame: up to 424 days

Population: Efficacy Evaluable Population. Two participants were assigned to a group labeled Other and were excluded from the EEP because the local laboratory FGF/FGFR results could not be confirmed centrally. Confidence intervals were calculated based on the exact method for binomial distribution.

ORR was defined as the percentage of participants with a best overall response of CR or PR at any post-Baseline visit prior to first PD, per RECIST v1.1. CR: disappearance of all target and non-target lesions and no appearance of any new lesions. Any pathological lymph nodes (whether target or non-target) must have a reduction in the short axis to \<10 mm. PR: complete disappearance or at least a 30% decrease in the sum of the diameters of target lesions, taking as a reference the baseline sum diameters, no new lesions, and no progression of non-target lesions. ORR was based on central genomics laboratory results. Response was based on review of scans by an independent centralized radiological review committee.

Time frame: up to 143 days

Population: Efficacy Evaluable Population. Two participants were assigned to a group labeled Other and were excluded from the EEP because the local laboratory FGF/FGFR results could not be confirmed centrally. Confidence intervals were calculated based on the exact method for binomial distribution.

Overall survival was defined as the length of time from the first dose of study drug (Day 1) until the date of death due to any cause.

Time frame: up to 51.32 months

Population: Efficacy Evaluable Population. Two participants were assigned to a group labeled Other and were excluded from the EEP because the local laboratory FGF/FGFR results could not be confirmed centrally. The 95% confidence intervals were calculated using the Brookmeyer and Crowley's method.

PFS was defined as the length of time from the first dose of study drug (Day 1) to the earlier of death or disease progression by RECIST v1.1, as assessed by the independent centralized radiological review committee.

Time frame: up to 50.17 months

Population: Efficacy Evaluable Population. Two participants were assigned to a group labeled Other and were excluded from the EEP because the local laboratory FGF/FGFR results could not be confirmed centrally. The 95% confidence intervals were calculated using the Brookmeyer and Crowley's method.

Vc/F is defined as the apparent volume of distribution for the central compartment of pemigatinib.

Time frame: Predose; 1-2 hours post-dose; 4-12 hours post-dose

Population: PK Population

Vp/F is defined as the apparent volume of distribution for the tissue (peripheral) compartment.

Time frame: Predose; 1-2 hours post-dose; 4-12 hours post-dose

Population: PK Population