Balversa Drug Information
Generic name: ERDAFITINIB
Kinase Inhibitor [EPC]
Uses of Balversa
is indicated for the treatment of adult patients with locally advanced or metastatic urothelial carcinoma (mUC) with susceptible FGFR3 genetic alterations whose disease has progressed on or after at least one line of prior systemic therapy. Select patients for therapy based on an FDA-approved companion diagnostic for BALVERSA . BALVERSA is a kinase inhibitor indicated for the treatment of adult patients with locally advanced or metastatic urothelial carcinoma (mUC) with susceptible FGFR3 genetic alterations whose disease has progressed on or after at least one line of prior systemic therapy. Select patients for therapy based on an FDA-approved companion diagnostic for BALVERSA. Limitations of Use BALVERSA is not recommended for the treatment of patients who are eligible for and have not received prior PD-1 or PD-L1 inhibitor therapy.
Limitations of Use BALVERSA is not recommended for the treatment of patients who are eligible for and have not received prior PD-1 or PD-L1 inhibitor therapy .
Dosage & Administration of Balversa
| 8 mg (two 4 mg tablets) | |
| 6 mg (two 3 mg tablets) |
Side Effects of Balversa
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. The pooled safety population described in the WARNINGS AND PRECAUTIONS reflects exposure to BALVERSA as a single agent at the recommended dose (8 to 9 mg orally daily) in 479 patients with advanced urothelial cancer and FGFR alterations in 42756493BLC3001 (NCT03390504), 42756493BLC2001 (NCT02365597), 42756493BLC2002 (NCT 03473743), and 42756493EDI1001 (NCT01703481). Among 479 patients who received BALVERSA, the median duration of treatment was 4.8 months (range: 0.1 to 43 months). In this pooled safety population, the most common (>20%) adverse reactions, including laboratory abnormalities, were increased phosphate, nail disorders, stomatitis, diarrhea, increased creatinine, increased alkaline phosphatase, increased alanine aminotransferase, decreased hemoglobin, decreased sodium, increased aspartate aminotransferase, fatigue, dry mouth, dry skin, decreased phosphate, decreased appetite, dysgeusia, constipation, increased calcium, dry eye, palmar-plantar erythrodysesthesia syndrome, increased potassium, alopecia, and central serous retinopathy. BLC3001 The safety of BALVERSA was evaluated in Cohort 1 of the BLC3001 study that included patients with locally advanced unresectable or metastatic urothelial carcinoma which had susceptible FGFR3 genetic alterations and were previously treated with a PD-1 or PD-L1 inhibitor . Patients received either BALVERSA (8 mg orally once daily with individualized up-titration to 9 mg) (n=135) or chemotherapy (docetaxel 75 mg/m 2 once every 3 weeks or vinflunine 320 mg/m 2 once every 3 weeks) (n=112). Among patients who received BALVERSA, median duration of treatment was 4.8 months (range: 0.2 to 38 months). Serious adverse reactions occurred in 41% of patients who received BALVERSA. Serious reactions in >2% of patients included urinary tract infection (4.4%), hematuria (3.7%), hyponatremia (2.2%), and acute kidney injury (2.2%). Fatal adverse reactions occurred in 4.4% of patients who received BALVERSA, including sudden death (1.5%), pneumonia (1.5%), renal failure (0.7%), and cardiorespiratory arrest (0.7%). Permanent discontinuation of BALVERSA due to an adverse reaction occurred in 14% of patients.
Adverse reactions which resulted in permanent discontinuation of BALVERSA in >2% of patients included nail disorders (3%) and eye disorders (2.2%). Dosage interruptions of BALVERSA due to an adverse reaction occurred in 72% of patients. Adverse reactions which required dosage interruption in >4% of patients included nail disorders (22%), stomatitis (19%), eye disorders (16%), palmar-plantar erythrodysesthesia syndrome (15%), diarrhea (10%), hyperphosphatemia (7%), increased aspartate aminotransferase (6%), and increased alanine aminotransferase (5%). Dose reductions of BALVERSA due to an adverse reaction occurred in 69% of patients. Adverse reactions which required dose reductions in >4% of patients included nail disorders (27%), stomatitis (19%), eye disorders (17%), palmar-plantar erythrodysesthesia syndrome (12%), diarrhea (7%), dry mouth (4.4%), and hyperphosphatemia (4.4%). Table 3 presents adverse reactions reported in ≥15% of patients treated with BALVERSA at 8 or 9 mg once daily versus chemotherapy.
Table 3: Adverse Reactions Reported in ≥15% of Patients Who Received BALVERSA Versus Chemotherapy (Study BLC3001) Adverse Reaction BALVERSA (N=135) Chemotherapy (N=112) All Grades (%) Grade 3–4 (%) All Grades (%) Grade 3–4 (%) Skin and subcutaneous tissue disorders Nail disorders Includes multiple terms 70 12 5 0 Palmar-plantar erythrodysesthesia syndrome 30 10 0.9 0 Dry skin 27 1.5 6 0 Alopecia 25 0.7 24 0 Gastrointestinal disorders Diarrhea 63 3 17
Stomatitis 56 10 18 1.8 Dry Mouth 39 0 3.6 0 Constipation
27 0 28
Nervous system disorders Dysgeusia 30 0.7 7 0 General disorders Fatigue 29
1.5 42 7 Metabolism and nutrition disorders Decreased appetite 27 3 21
Eye disorders Dry eye 25 0.7 3.6 0 Central serous retinopathy 18
2.2 0 0 Investigations Decreased weight 22 2 2.7 0 Clinically relevant adverse reactions in <15% of patients who received BALVERSA included nausea (15%), pyrexia (15%), epistaxis (13%), vomiting (10%), and arthralgia (10%). Table 4 presents laboratory abnormalities reported in ≥15% of patients treated with BALVERSA at 8 or 9 mg once daily versus chemotherapy. Table 4: Selected Laboratory Abnormalities Reported in ≥15% of Patients Who Received BALVERSA Versus Chemotherapy; Cohort 1 Safety Analysis Set (Study BLC3001) Laboratory Abnormality BALVERSA (N=135 The denominator used to calculate the rate varied from 52 to 131 based on the number of patients with a baseline value and at least one post-treatment value. ) Chemotherapy (N=112 The denominator used to calculate the rate varied from 11 to 102 based on the number of patients with a baseline value and at least one post-treatment value. ) All Grades Severity graded per NCI CTCAE v4.03. (%) Grade 3–4 (%) All Grades (%) Grade 3–4 (%) Chemistry Increased phosphate 76 5 0 0 Increased alkaline phosphatase 54 4.7 29 1 Increased alanine aminotransferase 46 3.8 15 1 Increased aspartate aminotransferase 44 3.1 13 0 Decreased sodium 44 16 25 6 Increased creatinine 43 1.5 17 0 Decreased phosphate 34 8 25
Increased calcium 27 8 9 0 Increased potassium 24 0 21 0
Hematology Decreased hemoglobin 50 12 57 12 Decreased platelet count 17 1.5 18 1 Decreased neutrophil count 16 0.8 40 26 BLC2001 The safety of BALVERSA was evaluated in the BLC2001 study that included 87 patients with locally advanced or metastatic urothelial carcinoma which had susceptible FGFR3 and other FGFR alterations, and which progressed during or following at least one line of prior chemotherapy including within 12 months of neoadjuvant or adjuvant chemotherapy . Patients were treated with BALVERSA at 8 mg orally once daily; with a dose increase to 9 mg in patients with phosphate levels <5.5 mg/dL on Day 14 of Cycle 1. Median duration of treatment was 5.3 months (range: 0 to 17 months). Serious adverse reactions occurred in 41% of patients. The most frequent (>3%) serious adverse reactions were central serous retinopathy (4.6%), urinary tract infection (3.4%), and general physical health deterioration (3.4%). Fatal adverse reactions occurred in 8% of patients, including acute myocardial infarction (1.1%). Permanent discontinuation of BALVERSA due to an adverse reaction occurred in 21% of patients. The most frequent (≥ 2%) reasons for permanent discontinuation included central serous retinopathy (4.6%), general physical health deterioration (3.4%), palmar-plantar erythrodysesthesia syndrome (2.3%), acute kidney injury (2.3%), and fatigue (2.3%). Dosage interruptions of BALVERSA occurred in 68% of patients.
The most frequent (≥ 5%) adverse reactions requiring dosage interruption included hyperphosphatemia (24%), stomatitis (17%), nail disorders (16%), central serous retinopathy (9%), palmar-plantar erythro-dysesthesia syndrome (8%), and fatigue (8%). Dose reductions of BALVERSA occurred in 53% of patients. The most frequent (≥ 5%) adverse reactions for dose reductions included nail disorders (21%), stomatitis (15%), central serous retinopathy (14%), hyperphosphatemia (7%), palmar-plantar erythro-dysesthesia syndrome (7%), fatigue (6%), and blurred vision (6%). Table 5 presents adverse reactions reported in ≥15% of patients treated with BALVERSA at 8 mg or 9 mg once daily. Table 5: Adverse Reactions Reported in ≥15% of Patients (Study BLC2001) Adverse Reaction BALVERSA 8 mg daily (N=87) All Grades (%) Grade 3–4 (%) Gastrointestinal disorders Stomatitis Includes multiple terms 62 11 Diarrhea 48
Dry mouth 45 0 Constipation 28 1.1 Nausea 21 1.1 Skin and
subcutaneous tissue disorders Nail disorders 62 14 Dry skin 37 0 Alopecia 26 0 Palmar-plantar erythrodysesthesia syndrome 26 6 General disorders and admin. site conditions Fatigue, Includes fatal adverse reactions (n=2) 54 8 Decreased weight 16 0 Metabolism and nutrition disorders Decreased appetite 38
Nervous system disorders Dysgeusia 38 1.1 Eye disorders Dry eye 29 1.1
Central serous retinopathy 28
Blurred vision 17 0 Infections and Infestations Urinary tract infection 17 6
Clinically relevant adverse reactions in <15% of patients who received BALVERSA included pyrexia (14%), extremity pain (13%), vomiting (13%), and peripheral edema (10%). Table 6 presents laboratory abnormalities reported in ≥15% of patients treated with BALVERSA at 8 mg or 9 mg once daily. Table 6: Selected Laboratory Abnormalities Reported in ≥ 15% of Patients Laboratory Abnormality BALVERSA 8 mg daily (N=87 The denominator used to calculate the rate varied from 83 to 86 based on the number of patients with a baseline value and at least one post-treatment value. ) All Grades (%) Grade 3–4 (%) Chemistry Increased phosphate 76
Increased creatinine 52 4.7 Increased alanine aminotransferase 41 1.2 Increased alkaline phosphatase
41
Decreased sodium 40 16 Decreased magnesium 31 1.2 Increased aspartate aminotransferase 30
0 Decreased phosphate 24 9 Increased calcium 22
Hematology Decreased hemoglobin 35 3.5 Decreased platelets 19 1.2 Decreased leukocytes 17
0
Warnings & Cautions for Balversa
Ocular Disorders
BALVERSA can cause ocular disorders, including central serous retinopathy/retinal pigment epithelial detachment (CSR/RPED) resulting in visual field defect. In the pooled safety population , CSR/RPED occurred in 22% of patients treated with BALVERSA, with a median time to first onset of 46 days. In 104 patients with CSR, 40% required dose interruptions and 56% required dose reductions; 2.9% of BALVERSA-treated patients required permanent discontinuation for CSR. Of the 24 patients who restarted BALVERSA after dose interruption with or without dose reduction, 67% had recurrence and/or worsening of CSR after restarting.
CSR was ongoing in 41% of the 104 patients at the time of last evaluation. Dry eye symptoms occurred in 26% of BALVERSA-treated patients. All patients should receive dry eye prophylaxis with ocular demulcents as needed.
Perform monthly ophthalmological examinations during the first 4 months of treatment and every 3 months afterwards, and urgently at any time for visual symptoms. Ophthalmological examination should include assessment of visual acuity, slit lamp examination, fundoscopy, and optical coherence tomography. Withhold or permanently discontinue BALVERSA based on severity and/or ophthalmology exam findings .
Hyperphosphatemia and Soft Tissue Mineralization
BALVERSA can cause hyperphosphatemia leading to soft tissue mineralization, cutaneous calcinosis, non-uremic calciphylaxis and vascular calcification. Increases in phosphate levels are a pharmacodynamic effect of BALVERSA. In the pooled safety population, increased phosphate occurred in 73% of BALVERSA-treated patients. The median onset time of increased phosphate was 16 days (range: 8–421) after initiating BALVERSA. Twenty-four percent of patients received phosphate binders during treatment with BALVERSA. Vascular calcification was observed in 0.2% of patients treated with BALVERSA. Monitor for hyperphosphatemia throughout treatment.
Restrict dietary phosphate intake (600–800 mg daily) and avoid concomitant use of agents that may increase serum phosphate levels. If serum phosphate is above 7.0 mg/dL, consider adding an oral phosphate binder until serum phosphate level returns to <7.0 mg/dL. Withhold, dose reduce, or permanently discontinue BALVERSA based on duration and severity of hyperphosphatemia according to Table 2.
Embryo-Fetal Toxicity
Based on the mechanism of action and findings in animal reproduction studies, BALVERSA can cause fetal harm when administered to a pregnant woman. In an embryo-fetal toxicity study, oral administration of erdafitinib to pregnant rats during the period of organogenesis caused malformations and embryo-fetal death at maternal exposures that were less than the human exposures at the maximum human recommended dose based on area under the curve (AUC). Advise pregnant women of the potential risk to the fetus. Advise female patients of reproductive potential to use effective contraception during treatment with BALVERSA and for one month after the last dose.
Advise male patients with female partners of reproductive potential to use effective contraception during treatment with BALVERSA and for one month after the last dose .
Drug Interactions with Balversa
Effect of Other Drugs on
BALVERSA Table 7 summarizes drug interactions that affect the exposure of BALVERSA or serum phosphate level and their clinical management. Table 7: Drug Interactions that Affect BALVERSA Moderate CYP2C9 or Strong CYP3A4 Inhibitors Clinical Impact Co-administration of BALVERSA with moderate CYP2C9 or strong CYP3A4 inhibitors increased erdafitinib plasma concentrations. Increased erdafitinib plasma concentrations may lead to increased drug-related toxicity.
Clinical Management Consider alternative therapies that are not moderate CYP2C9 or strong CYP3A4 inhibitors during treatment with BALVERSA. If co-administration of a moderate CYP2C9 or strong CYP3A4 inhibitor is unavoidable, monitor closely for adverse reactions and consider dose modifications accordingly. If the moderate CYP2C9 or strong CYP3A4 inhibitor is discontinued, resume the BALVERSA dose before dose modifications in the absence of drug-related toxicity. Strong CYP3A4 Inducers Clinical Impact Co-administration of BALVERSA with strong CYP3A4 inducers decreased erdafitinib plasma concentrations.
Decreased erdafitinib plasma concentrations may lead to decreased activity. Clinical Management Avoid co-administration of strong CYP3A4 inducers with BALVERSA. Moderate CYP3A4 Inducers Clinical Impact Co-administration of BALVERSA with moderate CYP3A4 inducers may decrease erdafitinib plasma concentrations. Decreased erdafitinib plasma concentrations may lead to decreased activity.
Clinical Management If a moderate CYP3A4 inducer must be co-administered at the start of BALVERSA treatment, administer BALVERSA at a dose of 9 mg daily. When a moderate CYP3A4 inducer is discontinued, continue BALVERSA at the same dose, in the absence of drug-related toxicity. Serum Phosphate Level-Altering Agents Clinical Impact Co-administration of BALVERSA with other serum phosphate level-altering agents may increase or decrease serum phosphate levels.
Changes in serum phosphate levels due to serum phosphate level-altering agents (other than erdafitinib) may interfere with serum phosphate levels needed for the determination of initial dose increased based on serum phosphate levels. Clinical Management Avoid co-administration of serum phosphate level-altering agents with BALVERSA before initial dose increase period based on serum phosphate levels (Days 14 to 21).
Effect of
BALVERSA on Other Drugs Table 8 summarizes the effect of BALVERSA on other drugs and their clinical management. Table 8: BALVERSA Drug Interactions that Affect Other Drugs P-glycoprotein (P-gp) Substrates Clinical Impact Co-administration of BALVERSA with P-gp substrates may increase the plasma concentrations of P-gp substrates. Increased plasma concentrations of P-gp substrates may lead to increased toxicity of the P-gp substrates.
Clinical Management If co-administration of BALVERSA with P-gp substrates is unavoidable, separate BALVERSA administration by at least 6 hours before or after administration of P-gp substrates with narrow therapeutic index.
Pregnancy Safety for Balversa
Pregnancy Risk Summary Based on the mechanism of action and findings in animal reproduction studies, BALVERSA can cause fetal harm when administered to a pregnant woman . There are no available data on BALVERSA use in pregnant women to inform a drug-associated risk. Oral administration of erdafitinib to pregnant rats during organogenesis caused malformations and embryo-fetal death at maternal exposures that were less than the human exposures at the maximum recommended human dose based on AUC (see Data ). Advise pregnant women and females of reproductive potential of the potential risk to the fetus. The estimated background risk of major birth defects and miscarriage for the indicated population is unknown.
All pregnancies have a background risk of birth defect, loss, or other adverse outcomes. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2–4% and 15–20%, respectively. Data Animal Data In an embryo-fetal toxicity study, erdafitinib was orally administered to pregnant rats during the period of organogenesis.
Doses ≥4 mg/kg/day (at total maternal exposures <0.1% of total human exposures at the maximum recommended human dose based on AUC) produced embryo-fetal death, major blood vessel malformations and other vascular anomalies, limb malformations (ectrodactyly, absent or misshapen long bones), an increased incidence of skeletal anomalies in multiple bones (vertebrae, sternebrae, ribs), and decreased fetal weight.
Pediatric Use of Balversa
Pediatric Use Safety and effectiveness of BALVERSA in pediatric patients have not been established. Skeletal adverse reactions have occurred in pediatric patients treated with BALVERSA. In a study of BALVERSA that included pediatric patients ages 6 to <18 years with FGFR-positive advanced solid tumors, epiphysiolysis and bone fractures occurred. In the postmarket setting and in literature reports, cases of slipped capital femoral epiphysis and accelerated linear growth in patients treated with BALVERSA have been reported.
Juvenile Animal Toxicity Data In 4 and 13-week repeat-dose toxicology studies in rats and dogs, toxicities in bone and teeth were observed at an exposure less than the human exposure (AUC) at the maximum recommended human dose. Chondroid dysplasia/metaplasia were reported in multiple bones in both species, and tooth abnormalities included abnormal/irregular denting in rats and dogs and discoloration and degeneration of odontoblasts in rats.
Clinical Studies of Balversa
Urothelial Carcinoma with Susceptible
FGFR3 Genetic Alterations The efficacy of BALVERSA was evaluated in Study BLC3001 (NCT03390504) Cohort 1, a randomized, open-label, multicenter study in which 266 patients with advanced urothelial cancer harboring selected FGFR3 alterations were randomized 1:1 to receive BALVERSA (8 mg with titration up to 9 mg) versus chemotherapy (docetaxel 75 mg/m 2 once every 3 weeks or vinflunine 320 mg/m 2 once every 3 weeks) until unacceptable toxicity or progression. Randomization was stratified by region (North America vs. Europe vs. rest of world), Eastern Cooperative Oncology Group (ECOG) performance status (0 or 1 vs. 2) and visceral or bone metastases (yes vs. no). All patients needed to have had disease progression after 1 or 2 prior treatments, at least 1 of which included a PD-1 or PD-L1 inhibitor.
FGFR3 genetic alterations were identified from tumor tissue in a central laboratory by the QIAGEN therascreen ® FGFR RGQ RT-Polymerase Chain Reaction (PCR) kit in 75% of patients while the remainder (25%) were identified by local next generation sequencing (NGS) assays. The major efficacy outcome measures were overall survival (OS), progression-free survival (PFS), and objective response rate (ORR) assessed by investigator using RECIST (Response Evaluation Criteria in Solid Tumors) Version 1.1. The median age was 67 years (range: 32 to 86 years) and 71% were male; 54% were White, 29% Asian, 0.4% Black, 0.4% multiple races, 16% not reported; 2% were Hispanic/Latino; and baseline ECOG performance status was 0 (43%), 1 (48%), or 2 (9%). Eighty-one percent of patients had FGFR3 mutations, 17% had fusions, and 2% had both mutations and fusions. Ninety-five percent of patients had pure transitional cell carcinoma (TCC) and 5% had TCC with other histologic variants.
The primary tumor location was the upper tract for 33% of subjects and lower tract for 67%; 74% of patients had visceral or bone metastases. Eighty-eight percent of patients received platinum-containing chemotherapy previously. PD-1 or PD-L1 inhibitor therapy was received only in the neoadjuvant or adjuvant setting in 7% of patients.
Statistically significant improvements in OS, PFS, and ORR were demonstrated for BALVERSA compared with chemotherapy. Table 9 and Figures 1 and 2 summarize the efficacy results for BLC3001 Cohort 1. Table 9: Efficacy Results for Study BLC3001 Cohort 1 BALVERSA N=136 Chemotherapy N=130 All p-values reported are 2-sided and compared with 0.019 of the allocated alpha for the interim analysis. ORR = confirmed objective response (CR + PR) CI = Confidence Interval Overall Survival (OS) Number of events (%) 77 (56.6%) 78 (60.0%) Median Based on Kaplan-Meier estimates, months (95% CI) 12.1
Hazard ratio
Based on an unstratified Cox proportional hazard model (95% CI) 0.64 p-value Based on an unstratified log-rank test 0.0050 Progression-free survival (PFS) Number of events (%) 101 (74.3%) 90 (69.2%) Median, months (95% CI) 5.6
Hazard ratio (95% CI) 0.58 p-value 0.0002 Objective response rate (ORR)
ORR (95% CI) 35.3% 8.5% p-value p-value is estimated using Cochran-Haenszel (CMH) test with ECOG performance status (0 or 1 vs 2) as a stratification factor. <0.001 Complete response, CR (%) 5.1% 0.8% Partial response, PR (%) 30.1% 7.7% Figure 1: Kaplan-Meier Plot of Overall Survival (Study BLC3001 Cohort 1) Figure 2: Kaplan-Meier Plot of Progression-free Survival (Study BLC3001 Cohort 1) Figure 1 Figure 2 Study BLC3001 Cohort 2 Study BLC3001 (NCT03390504) Cohort 2 was a multicenter, open-label, randomized study in 351 patients with locally advanced or metastatic urothelial carcinoma with selected FGFR3 alterations who received 1 prior line of systemic therapy and no prior PD-1 or PD-L1 inhibitor. Patients were randomized 1:1 to receive BALVERSA (8 mg with titration up to 9 mg) or pembrolizumab 200 mg every 3 weeks. The study did not meet its major efficacy outcome measure for superiority of OS at the pre-specified final analysis.
The OS hazard ratio (HR) was 1.18 (95% CI: 0.92, 1.51; p=0.18), median 10.9 (95% CI: 9.2, 12.6) months for BALVERSA versus 11.1 (95% CI: 9.7, 13.6) months for pembrolizumab . Study BLC2001 Study BLC2001 (NCT02365597) was a multicenter, open-label, single-arm study to evaluate the efficacy and safety of BALVERSA in patients with locally advanced or metastatic urothelial carcinoma (mUC). FGFR mutation status for screening and enrollment of patients was determined by a clinical trial assay (CTA). The efficacy population consists of a cohort of eighty-seven patients who were enrolled in this study with disease that had progressed on or after at least one prior chemotherapy and that had at least 1 of the following genetic alterations: FGFR3 gene mutations ( R248C, S249C, G370C, Y373C ) or FGFR gene fusions ( FGFR3-TACC3, FGFR3-BAIAP2L1, FGFR2-BICC1, FGFR2-CASP7 ), as determined by the CTA performed at a central laboratory. Tumor samples from 69 patients were tested retrospectively by the QIAGEN therascreen ® FGFR RGQ RT-PCR Kit, which is the FDA-approved test for selection of patients with mUC for BALVERSA. Patients received a starting dose of BALVERSA at 8 mg once daily with a dose increase to 9 mg once daily in patients whose serum phosphate levels were below the target of 5.5 mg/dL between days 14 and 17; a dose increase occurred in 41% of patients. BALVERSA was administered until disease progression or unacceptable toxicity.
The major efficacy outcome measures were ORR and duration of response (DoR), as determined by blinded independent review committee (BIRC) according to RECIST v1.1. The median age was 67 years (range: 36 to 87 years), 79% were male, and 74% were Caucasian. Most patients (92%) had a baseline Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1. Sixty-six percent of patients had visceral metastases. Eighty-four (97%) patients received at least one of cisplatin or carboplatin previously.
Fifty-six percent of patients only received prior cisplatin-based regimens, 29% received only prior carboplatin-based regimens, and 10% received both cisplatin and carboplatin-based regimens. Three (3%) patients had disease progression following prior platinum-containing neoadjuvant or adjuvant therapy only. Twenty-four percent of patients had been treated with prior anti PD-L1/PD-1 therapy.
Efficacy results are summarized in Table 10 and Table 11. ORR was 32.2%. Responders included patients who had previously not responded to anti PD-L1/PD-1 therapy. Table 10: Efficacy Results Endpoint BIRC BIRC: Blinded Independent Review Committee Assessment N=87 ORR = CR + PR CI = Confidence Interval ORR (95% CI) 32.2% Complete response (CR) 2.3% Partial response (PR) 29.9% Median DoR in months (95% CI)
Table 11: Efficacy Results by
FGFR Genetic Alteration BIRC BIRC: Blinded Independent Review Committee Assessment ORR = CR + PR CI = Confidence Interval FGFR3 Point Mutation N=64 ORR (95% CI) 40.6% FGFR3 Fusion Both responders had FGFR3-TACC3_V1 fusion, One patient with a FGFR2-CASP7/FGFR3-TACC3_V3 fusion is reported in both FGFR2 fusion and FGFR3 fusion above N=18 ORR (95% CI) 11.1% FGFR2 Fusion N=6 ORR 0
Drug information sourced from the FDA. This content is for informational purposes only and does not constitute medical advice. Consult a healthcare professional before making any medication decisions.
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