FDA approves first targeted therapy for metastatic bladder cancer

FDAThe U.S. Food and Drug Administration today granted accelerated approval to Balversa (erdafitinib), a treatment for adult patients with locally advanced or metastatic bladder cancer that has a type of susceptible genetic alteration known as FGFR3 or FGFR2, and that has progressed during or following prior platinum-containing chemotherapy. Patients should be selected for therapy with Balversa using an FDA-approved companion diagnostic device.

"We're in an era of more personalized or precision medicine, and the ability to target cancer treatment to a patient’s specific genetic mutation or biomarker is becoming the standard, with advances being made in new disease types. Today's approval represents the first personalized treatment targeting susceptible FGFR genetic alterations for patients with metastatic bladder cancer," said Richard Pazdur, M.D., director of the FDA's Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. "FGFRs regulate important biological processes including cell growth and division during development and tissue repair. This drug works by targeting genetic alterations in FGFRs."

The most common type of bladder cancer is transitional cell carcinoma, also called urothelial carcinoma. Bladder cancers are associated with genetic mutations that are present in the patient's bladder or entire urothelium (the lining of the lower urinary tract). Bladder cancer is the sixth most common cancer in the United States. Fibroblast growth factor (FGFR) alterations are present in approximately one in five patients with recurrent and refractory bladder cancer.

The efficacy of Balversa was studied in a clinical trial that included 87 patients with locally advanced or metastatic bladder cancer, with FGFR3 or FGFR2 genetic alterations, that had progressed following treatment with chemotherapy. The overall response rate in these patients was 32.2%, with 2.3% having a complete response and almost 30% having a partial response. The response lasted for an average of approximately five-and-a-half months. About a quarter of patients in the study were previously treated with anti PD-L1/PD-1 therapy, which is a standard treatment for patients with locally advanced or metastatic bladder cancer. Responses to Balversa were seen in patients who had previously not responded to anti PD-L1/PD-1 therapy.

Common side effects reported by patients taking Balversa were increased phosphate level, mouth sores, feeling tired, change in kidney function, diarrhea, dry mouth, nails separating from the bed or poor formation of the nail, change in liver function, low salt (sodium) levels, decreased appetite, change in sense of taste, low red blood cells (anemia), dry skin, dry eyes and hair loss. Other side effects include redness, swelling, peeling or tenderness on the hands or feet (hand foot syndrome), constipation, stomach pain, nausea and muscle pain.

Balversa may cause serious eye problems, including inflamed eyes, inflamed cornea (front part of the eye) and disorders of the retina, an internal part of the eye. Patients are advised to have eye examinations intermittently and to tell their health care professional right away if they develop blurred vision, loss of vision or other visual changes. Health care professionals are advised to check patients' blood phosphate level between 14 and 21 days after starting treatment and monthly, and to increase the dose Balversa in patients whose serum phosphate is below the target level.

Health care professionals are advised to tell male patients with female partners of reproductive potential to use effective contraception during treatment with Balversa and for one month after the last dose. Pregnancy testing is recommended for females of reproductive potential prior to initiating treatment with Balversa. Women who are pregnant or breastfeeding should not take Balversa because it may cause harm to a developing fetus or newborn baby. Balversa must be dispensed with a patient Medication Guide that describes important information about the drug’s uses and risks.

Balversa received an Accelerated Approval, which enables the FDA to approve drugs for serious conditions to fill an unmet medical need using clinical trial data that is thought to predict a clinical benefit to patients. Further clinical trials are required to confirm Balversa’s clinical benefit and the sponsor is conducting or plans to conduct these studies. Balversa was also granted Breakthrough Therapy designation.

The FDA granted the approval of Balversa to Janssen Pharmaceutical.

The FDA also approved the therascreen FGFR RGQ RT-PCR Kit, developed by QIAGEN Manchester, Ltd., for use as a companion diagnostic with Balversa for this therapeutic indication.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

Most Popular Now

Sanofi and GSK sign agreements with the Government…

Sanofi and GSK have signed agreements with the Government of Canada for the supply of up to 72 million doses of an adjuvanted COVID-19 vaccine, beginning in 2021. Thom...

BioNTech and Pfizer initiate rolling submission to…

Pfizer Inc. (NYSE: PFE) and BioNTech SE (Nasdaq: BNTX) today announced the initiation of a rolling submission to the European Medicines Agency (EMA) for BNT162b2, the lea...

Johnson & Johnson initiates pivotal global Pha…

Johnson & Johnson (NYSE: JNJ) (the Company) announced the launch of its large-scale, pivotal, multi-country Phase 3 trial (ENSEMBLE) for its COVID-19 vaccine candidate, J...

Scientists trace severe COVID-19 to faulty genes a…

More than 10 percent of young and healthy people who develop severe COVID-19 have misguided antibodies that attack not the virus, but the immune system itself, new resear...

Vir Biotechnology and GSK announce global expansio…

Vir Biotechnology, Inc. (Nasdaq: VIR) and GlaxoSmithKline plc (LSE/NYSE: GSK) announced the global expansion to Phase 3 of the COMET-ICE (COVID-19 Monoclonal antibody Eff...

Johnson & Johnson announces European Commissio…

Johnson & Johnson (NYSE: JNJ) (the Company) announced the European Commission (EC), acting on behalf of the European Union (EU) Member States, has approved an Advance Pur...

Johns Hopkins researchers publish COVID-19 'predic…

Using a combination of demographic and clinical data gathered from seven weeks of COVID-19 patient care early in the coronavirus pandemic, Johns Hopkins researchers today...

Web resources bring new insight into COVID-19

Researchers around the world are a step closer to a better understanding of the intricacies of COVID-19 thanks to two new web resources developed by investigators at Bayl...

COVID-19 vaccine AZD1222 clinical trial resumed in…

The Phase I/II clinical trial for the COVID-19 vaccine AZD1222 has resumed in Japan after discussion with the Japanese Pharmaceuticals and Medical Devices Agency (PMDA). ...

COVID-19 Long-Acting AntiBody (LAAB) combination A…

AstraZeneca's long-acting antibody (LAAB) combination, AZD7442, will advance into two Phase III clinical trials in more than 6,000 participants at sites in and outside th...

Adequate levels of vitamin D reduces complications…

Hospitalized COVID-19 patients who were vitamin D sufficient, with a blood level of 25-hydroxyvitamin D of at least 30 ng/mL (a measure of vitamin D status), had a signif...

Protective antibodies persist for months in surviv…

People who survive serious COVID-19 infections have long-lasting immune responses against the virus, according to a new study led by researchers at Massachusetts General ...