Erdafitinib Latest News

On January 19, 2024, the U.S. Food and Drug Administration approved erdafitinib (Balversa, Janssen Biotech) for adult patients with locally advanced or metastatic urothelial carcinoma (mUC) who have predisposing FGFR3 genetic alterations and whose disease has progressed during or after at least one prior systemic therapy, as determined by an FDA-approved companion diagnostic test. Erdafitinib is not recommended for the treatment of eligible patients who have not previously received PD-1 or PD-L1 inhibitor therapy. This approval modifies the previously accelerated approval indication for mUC patients with predisposing FGFR3 or FGFR2 alterations after prior platinum-containing chemotherapy.

Study Efficacy was evaluated in BLC3001 Cohort 1, a randomized, open-label trial in 266 patients with mUC with specific FGFR3 alterations who had received 1-2 prior systemic therapies, including PD-1 or PD-L1 inhibitors. Patients were randomly assigned in a 1:1 ratio to receive erdafitinib or investigator's choice of chemotherapy (docetaxel or vinflunine). Randomization was stratified by region, performance status, and presence of visceral or bone metastases. In a central laboratory, FGFR3 alterations were identified in tumor tissue by the therascreen FGFR RGQ RT-PCR kit (Qiagen) in 75% of patients, whereas in the remaining patients they were identified by local next-generation sequencing assays.

The primary efficacy outcome measure is overall survival (OS). Investigator-assessed progression-free survival (PFS) and objective response rate (ORR) were additional outcome measures.

Compared with chemotherapy, erdafitinib demonstrated statistically significant improvements in OS, PFS, and ORR. Median OS was 12.1 months (95% CI: 10.3, 16.4) for patients who received erdafitinib and 7.8 months (95% CI: 6.5, 11.1) for patients who received chemotherapy (hazard ratio [HR] 0.64 [95% CI: 0.47, 0.88]; p-value=0.0050). The median number of patients treated with erdafitinibPFS was 5.6 months (95% CI: 4.4, 5.7) versus 2.7 months (95% CI: 1.8, 3.7) for patients who received chemotherapy (HR 0.58 [95% CI: 0.44, 0.78]; p-value=0.0002). The confirmed ORR was 35.3% (95% CI: 27.3, 43.9) in patients treated with erdafitinib and 8.5% (95% CI: 4.3, 14.6) in patients treated with chemotherapy (p-value<0.001).

The most common (>20%) adverse reactions, including laboratory abnormalities, were increased phosphate, nail disease, diarrhea, stomatitis, increased alkaline phosphatase, decreased hemoglobin, increased alanine aminotransferase, increased aspartate aminotransferase, decreased sodium, increased creatinine, dry mouth, decreased phosphate, palmoplantar erythrodysesthesia syndrome, dysgeusia, fatigue, dry skin, constipation, decreased appetite, increased calcium, alopecia, dry eyes, increased potassium, and weight loss.

The recommended dose of erdafitinib is 8 mg orally once daily, with the dose increased to 9 mg once daily on days 14 to 21 based on tolerability, including hyperphosphatemia. Treatment should be continued until disease progression or unacceptable toxicity occurs.

Erdafitinib is not currently on the market in China, and patients can only purchase it from abroad or contact regular overseas medical consulting companies. It is understood that the original drug specification of erdafitinib listed in Hong Kong is 4mg*14 tablets, and the price is more than 20,000 yuan. There are also related generic versions of erdafitinib.There are multiple versions of generic versions launched overseas in Bangladesh, including3mg, 4mg and 5mg. Taking 4mg as an example, the price of 28 tablets is around 1,000 yuan. If patients want to know more about the generic version of erdafitinib, please consult a regular overseas medical consulting company.

In summary, erdafitinib, as a new type of targeted therapy, has demonstrated significant efficacy in the treatment of urothelial cancer. However, since it is not yet available in mainland China, patients need to obtain the drug through other means.