The US Food and Drug Administration today granted accelerated approval for Balversa (erdafitinib), a treatment for adult patients with locally advanced or metastatic bladder cancer who have a type of genetic alteration called FGFR3 or FGFR2, who progressed during or chemotherapy containing platinum. Patients should be selected for treatment with Balversa with the aid of an FDA-approved companion diagnostic device.
"We are in a time of more personalized or precision medicine, and the ability to target cancer treatment on a specific genetic mutation or biomarker of the patient is becoming the norm, with advances in new types of diseases. Today's approval is the first personalized treatment targeting sensitive FGFR genes. alterations for patients with metastatic bladder cancer, "said Richard Pazdur, MD, director of the FDA's Center of Excellence in Oncology and acting director of the Bureau of Hematology and Oncology Products. from the FDA's Center for Drug Evaluation and Research. "FGFRs regulate important biological processes, including cell growth and division during tissue development and repair. This medication works by targeting genetic alterations in FGFRs. "
The most common type of bladder cancer is transitional cell carcinoma, also known as urothelial carcinoma. Bladder cancer is associated with genetic mutations in the patient's bladder or urothelium (the lining of the lower urinary tract). Bladder cancer is the sixth most common cancer in the United States. Changes in fibroblast growth factor (FGFR) are present in about one in five patients with recurrent or refractory bladder cancer.
The effectiveness of Balversa has been studied in a clinical trial involving 87 patients with locally advanced or metastatic bladder cancer, with genetic alterations FGFR3 or FGFR2, which had progressed afterwards. chemotherapy treatment. The overall response rate in these patients was 32.2%, with 2.3% complete responses and almost 30% partial responses. The answer lasted on average about five and a half months. About a quarter of the study's patients had previously received anti-PD-L1 / PD-1 treatment, standard treatment for patients with locally advanced or metastatic bladder cancer. Balversa responses have been observed in patients who have never responded to anti-PD-L1 / PD-1 therapy.
The common side effects reported by patients taking Balversa have been increased phosphate levels, mouth sores, tiredness, impaired renal function, diarrhea, dry mouth, and nails separating from the bed. or poor nail formation, impaired liver function, decreased appetite, change in sense of taste, deficiency of red blood cells (anemia), dryness of the skin, dryness of the eyes and hair loss. Other side effects: redness, swelling, flaking or tenderness of hands or feet (foot and foot syndrome), constipation, stomach pain, nausea and muscle aches.
Balversa can cause serious eye problems, including inflamed eyes, an inflamed cornea (anterior part of the eye) and retinal disorders, an internal part of the eye. Patients are advised to undergo intermittent eye exams and to immediately notify their health care professional if they develop blurred vision, vision loss or other visual changes. Health care professionals are advised to check blood phosphate levels in patients' blood between 14 and 21 days after starting treatment and every month, and to increase the dose of Balversa in patients with Serum phosphate is below the target level.
Health professionals are advised to inform male patients with female reproductive potential partners to use effective contraception during treatment with Balversa and for the month following the last dose. The pregnancy test is recommended for women of childbearing age before starting treatment with Balversa. Pregnant or nursing women should not take Balversa as this may affect the development of the fetus or newborn. Balversa should be exempted from the patient's medication guide, which describes important information about the use and risks of the drug.
Balversa has received accelerated approval, which allows the FDA to approve drugs to treat serious conditions, to address an unmet medical need, using clinical trial data believed to predict clinical benefit. for patients. Additional clinical trials are needed to confirm the clinical benefit of Balversa and the sponsor is conducting or considering conducting these studies. Balversa has also obtained the designation of rupture therapy.
The FDA has granted Balversa approval to Janssen Pharmaceutical.
The FDA has also approved the therascreen FGFR RGQ RT-PCR kit, developed by QIAGEN Manchester, Ltd., to be used as a complementary diagnostic with Balversa for this therapeutic indication.
The FDA, an agency of the US Department of Health and Social Services, protects public health by ensuring the safety, efficacy and safety of human and veterinary drugs, vaccines and other biological products for human use and medical devices. The agency is also responsible for the safety of food products, cosmetics, food supplements, products emitting electronic radiation and the regulation of tobacco products.