How effective is Erdafitinib, the first targeted drug, in the treatment of advanced bladder cancer?

The first targeted drug Erdafitinib (Erdafitinib, trade name Balversa) has a remarkable clinical therapeutic effect in advanced bladder cancer, providing new treatment options and hope for such patients.

Erdafitinib is a potent and selective pan-fibroblast growth factor receptor (FGFR) tyrosine kinase inhibitor. FGFRs are a family of receptor tyrosine kinases. Gene mutations that appear in different tumors can cause them to be activated, thereby promoting the survival and proliferation of tumor cells. Erdafitinib blocks the FGFR signaling pathway by inhibiting mutations or fusions of FGFR2, FGFR3 and other genes, thereby inhibiting the growth and spread of tumor cells.

Multiple clinical studies have shown that erdafitinib exhibits good therapeutic effects in patients with advanced bladder cancer. BLC2001The study was conducted in 1412 countries in Asia, Europe and North America. An open-label, uncontrolled Phase 2 study of BLC2001 at 6 medical centers. Eligible patients were 18 years or older, had locally advanced unresectable or metastatic bladder cancer with prespecified FGFR alterations, and had received at least one dose of systemic chemotherapy or had progressive disease within 12 months after neoadjuvant or adjuvant chemotherapy, or were ineligible for cisplatin treatment.

Among 2328 patients, 212 were selected, of which 101 received 8 mg/ days of erdafitinib treatment. The investigator-assessed objective response rate was 40%. Erdafitinib demonstrated sustained activity and a manageable safety profile with extended follow-up in patients with locally advanced or metastatic bladder cancer.

Another clinical trial enrolled 87 patients with locally advanced or metastatic bladder cancer who had disease progression during or after at least one chemotherapy regimen and who had FGFR3 gene mutations orFGFRgene fusion.

The objective response rate (ORR, tumor shrinkage 30% or more) for all patients was 32.2%, and the complete response rate ( CR, complete tumor disappearance) was 2.3%, and the partial response rate (PR) was 29.9%. The median duration of response (DOR) was 5.4 months. The efficacy of erdafitinib varies for different types of genetic mutations. For patients with FGFR3 point mutations, ORR is 40.6%; for patients with FGFR3 fusion mutations, The ORR is 11.1%; while for patients with FGFR2 fusion mutations, the ORR is 0%, that is, the treatment is ineffective.

Erdafitinib can significantly prolong the progression-free survival of patients with bladder cancer, which is of great significance for improving patients' quality of life and extending survival time. In some patients, erdafitinib resulted in partial or complete tumor response, which is a very positive treatment response for patients with advanced bladder cancer. Erdafitinib has different effects on different FGFR gene mutations. It is effective in patients with FGFR3 point mutations, but is ineffective in patients with FGFR2 fusion mutations. This suggests the importance of FGFR gene testing before using erdafitinib.

Some side effects may occur during treatment with erdafitinib, but most are manageable. Here are some common side effects:

1.Stomatitis: incidence rate56%, is one of the most common side effects of erdafitinib treatment.

2.Fatigue: the incidence rate is 54% and may affect patients’ daily life and treatment compliance.

3.Diarrhea: The incidence rate is 47% and may lead to dehydration and other complications.

4.Dry mouth: the incidence rate is 45%, which may affect the patient's diet and oral health.

5.Nail bed separation: The incidence rate is 41% and may cause discomfort and pain in the fingers or toes.

In addition, erdafitinib may also cause side effects such as dysgeusia, dry skin, constipation, dry eye syndrome, and hair loss. During treatment, doctors will closely monitor the patient's side effects and adjust drug dosage or take other treatment measures as necessary to reduce side effects.