JOHNSON & JOHNSON Erdafitinib is the first targeted therapy to receive a positive CHMP recommendation in this patient population.3
'FGFR alterations are important oncogenic drivers in urothelial carcinoma and can be associated with adverse clinical outcomes,' said Yohann Loriot, M.D., Ph.D.,
'Today's recommendation from the CHMP marks important progress towards transforming outcomes for patients diagnosed with bladder cancer with FGFR alterations. There is a critical need for a multidisciplinary care team approach, to identify patients who may benefit from erdafitinib through biomarker testing, ensuring the right treatment reaches the right patient at the right time,' said Henar Hevia, Ph.D, Senior Director, EMEA Therapeutic Area Lead, Oncology, Johnson & Johnson Innovative Medicine. 'Pending approval, we look forward to providing eligible patients across the region with a new treatment option as soon as possible.'
The positive CHMP opinion is supported by data from Cohort 1 of the randomised, controlled, open-label multicentre Phase 3 THOR study (NCT03390504),9 evaluating the efficacy and safety of erdafitinib (n=136) versus chemotherapy (n=130) in patients with advanced or mUC with select FGFR alterations who have progressed on or after one or two prior treatments, at least one of which includes an anti-PD-(L) 1 agent.9 In
Serious treatment-related adverse events (TRAEs) were observed in 13.3 percent of patients who received erdafitinib and 24.1 percent of patients randomised to chemotherapy.2 Grade 3 or higher adverse events were observed in 45.9 percent of patients on erdafitinib and 46.4 percent on chemotherapy.2 Amongst patients who received erdafitinib, 8.1 percent had TRAEs that led to discontinuation of therapy, versus 13.4 percent of patients who received chemotherapy.2 TRAEs leading to death were reported in one patient who received erdafitinib and six patients who received chemotherapy.2
'Erdafitinib has been shown to significantly improve outcomes for patients with FGFR3-altered urothelial carcinoma based on the THOR study results and represents an important new therapeutic option for these patients,' said
About the THOR Study
THOR (NCT03390504) is a Phase 3 randomised, open-label, multicentre study evaluating the efficacy and safety of erdafitinib. All patients included in the study had metastatic or unresectable UC,9 with selected FGFR genetic alterations and showed disease progression during or after one or two prior lines of treatment.9 The study compared erdafitinib in two cohorts; erdafitinib versus standard of care chemotherapy (investigators choice of docetaxel or vinflunine) after at least one line of treatment including an anti-PD-(L)1 agent (Cohort 1);9 and erdafitinib compared to pembrolizumab after one prior treatment not containing an anti-PD-(L)1 agent (Cohort 2).9 The trial consists of a screening step, a treatment phase (from randomisation until disease progression, intolerable toxicity, withdrawal of consent or decision by investigator to discontinue treatment) and a post-treatment follow-up (from end-of-treatment to participants death, withdraws consent, lost to follow-up study completion for the respective cohort, whichever comes first).9 A long-term extension period is already operational following the clinical cut-off date of the final analysis of each cohort and eligible patients are continuing to benefit from the study intervention.10 The primary endpoint of the study is OS, with secondary endpoints being PFS, ORR, duration of response (DOR), patient-reported outcomes, safety and pharmacokinetics (PK).9
Results from Cohort 1 were presented at the 2023
About Erdafitinib
Erdafitinib is a pan-fibroblast growth factor receptor (FGFR) tyrosine kinase inhibitor being evaluated by
In addition to the Phase 3 THOR/BLC3001 (NCT03390504) study,9 erdafitinib is being studied as a once-daily oral tablet in: the Phase 2 THOR-2/BLC2003 (NCT04172675)13 study examining erdafitinib versus investigator choice of intravesical chemotherapy in participants who received Bacillus Calmette-Guerin and recurred with high risk non-muscle-invasive bladder cancer; the Phase 1b/2 NORSE/BLC2002 (NCT03473743)14 study of erdafitinib in combination with cetrelimab in patients with locally advanced or mUC and FGFR3 or FGFR2 gene alterations and the Phase 2 RAGNAR/CAN2002 (NCT04083976)15 study evaluating the safety and efficacy of erdafitinib in patients with advanced solid tumours, regardless of cancer type or tumour location (tumour-agnostic), driven by FGFR1-4 alterations.
Erdafitinib is also being investigated in the Phase 1 study/BLC1003 (NCT05316155)16 evaluating erdafitinib in patients with non-muscle invasive or muscle invasive bladder cancer with selected FGFR alterations, given via the intravesical targeted releasing system (TAR-210)16 and the Phase 3 MoonRISe-1 /BLC3004 (NCT06319820) study evaluating TAR-210 versus single agent intravesical cancer treatment in participants with bladder cancer.17
In addition to the marketing authorisation submitted to the EMA in
In 2008,
About Urothelial Carcinoma
Urothelial carcinoma (UC), also known as transitional cell carcinoma, starts in the innermost lining of the bladder.20 Almost all bladder cancers - more than 90 percent - are UCs.21 Up to one in five patients (20 percent) diagnosed with mUC have a fibroblast growth factor receptor (FGFR) genetic alteration.8 FGFRs are a family of receptor tyrosine kinases that can be activated by genetic alterations in a variety of tumour types, and these alterations may lead to increased tumour cell growth and survival.22 FGFRs play a key role in several biological processes, including tissue repair, inflammatory response and metabolism.23 Fusions or mutations in the genes that control FGFR (known as FGFR1-4 alterations) may lead to the development and progression of certain cancers by increasing tumour cell growth and survival.24 Patients with advanced UC, including FGFR-driven tumours, face a poor prognosis and the need for innovative therapies remains high.25 The five-year survival rate for patients with metastatic bladder cancer that has spread to distant parts of the body is currently 8 percent.26
About FGFR Testing
Testing for somatic mutations and fusions in FGFR genes can identify patients with urothelial carcinoma who may be eligible for treatment with FGFR-targeted therapies such as erdafitinib.27 Identification of actionable FGFR alterations may allow for utilisation of biomarker-guided therapy.28 FGFR testing to detect gene mutations, fusions and amplifications can be performed using validated laboratory developed tests involving polymerase chain reaction (PCR) and next-generation sequencing (NGS) techniques as recommended in the ESMO 2024 and EAU 2024 guidelines.28,29,30 These guidelines recommend early molecular/genomic testing, ideally at diagnosis of advanced bladder cancer, to facilitate treatment decision-making and prevent delays in administering later lines of therapy.30,31
About
At
Cautions Concerning Forward-Looking Statements
This press release contains 'forward-looking statements' as defined in the Private Securities Litigation Reform Act of 1995 regarding product development and the potential benefits and treatment impact of erdafitinib. The reader is cautioned not to rely on these forward-looking statements. These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialise, actual results could vary materially from the expectations and projections of
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