Approved for the treatment of adult patients with locally advanced or metastatic bladder cancer with an FGFR3 or FGFR2 alteration that has progressed on platinum-containing chemotherapy.
Trade name Balversa.
The first targeted therapy approved for metastatic bladder cancer.
The approval is based on the phase II BLC2001 trial, in which the drug induced an overall response rate of 32.2% in patients with FGFR2/FGFR3-positive locally advanced or metastatic bladder cancer.
The ORR comprised a complete response rate of 2.3% and a partial response rate of 29.9%.
Responders included patients who had been unresponsive to anti–PD-1/PD-L1 treatment.
A companion diagnostic device should be used in patient selection for erdafitinib.
The QIAGEN therascreen® FGFR RGQ Reverse-transcription (RT)-polymerase chain reaction (PCR) Kit has been approved as a companion diagnostic for use with erdafitinib.
The starting dose of erdafitinib was 8 mg once daily.
Patients whose serum phosphate levels were below the target of 5.5 mg/dL between days 14 and 17 had their dose increased to 9 mg once daily.
The median duration of response was 5.4 months.
Among 64 patients with an FGFR3 point mutation, the ORR was 40.6% and the ORR was 11.1% among 18 patients with an FGFR3 fusion.
There were no confirmed responses among the 6 patients with an FGFR2 fusion.
The most common adverse events: were phosphate increased (76%), stomatitis (56%), fatigue (54%), creatinine increased (52%), diarrhea (47%), dry mouth (45%), onycholysis (41%), ALT increased (41%), ALP increased (41%), sodium decreased (40%), decreased appetite (38%), albumin decreased (37%), dysgeusia (37%), hemoglobin decreased (35%), dry skin (34%), AST increased (30%), magnesium decreased (30%), dry eye (28%), alopecia (26%), palmar-plantar erythrodysesthesia syndrome (26%), constipation (28%), phosphate decreased (24%), abdominal pain (23%), calcium increased (22%), nausea (21%), and musculoskeletal pain (20%).
Grade ≥3 AEs that were the most common included onycholysis (10%), stomatitis (9%), palmar-plantar erythrodysesthesia syndrome (6%), and paronychia (3%).
FGFRs regulate important biological processes including cell growth and division during development and tissue repair.