Johnson & Johnson’s Balversa therapy finally gets full FDA approval

The approval by the Federal Drug Agency (FDA) of the bladder cancer therapy Balversa (erdafitinib) has taken a notch higher the search for more treatment options for the nearly 600,000 people who are affected by the disease annually.

FDA says Balversa received full approval for its use in the treatment of adult patients who have advanced or metastatic urothelial carcinoma (mUC) "with susceptible fibroblast growth factor receptor 3 (FGFR3) genetic alterations, whose disease has progressed on or after at least one line of prior systemic therapy." ⁽¹⁾

It is estimated 20% of bladder cancer patients with mUC have FGFR3 genetic alterations.

In some bladder cancer cases, the cells have been observed to change in the FGFR genes, which controls how much of the FGFR proteins are made. Balversa is the latest drug, or FGFR inhibitor, that targets the cells with the FGFR gene changes in the treatment of bladder cancer.

However, FDA has clarified the therapy, a once-daily, oral FGFR kinase inhibitor, is only recommended for patients who are eligible and have not received prior programmed death-ligand 1 (PD-L1) or programmed cell death protein 1 (PD-1) inhibitor therapy. The inhibitors' purpose is to block the PD-L1 from interacting with its PD-1 receptors hence avert the likelihood of destroying healthy cells, hence the likelihood of triggering an autoimmune disease. ⁽²⁾

With the announcement, FDA has brought into fruition its April 2019 commitment to accelerate the full approval of Balversa based on the clinical and overall survival benefit observed during the therapy's Phase 3 THOR study. 

FDA says the approval of Balversa makes the therapy the first and only "targeted for patients with locally advanced or metastatic urothelial carcinoma and susceptible fibroblast growth factor receptor alterations."

The therapy, which is a product of American multinational, pharmaceutical, and medical technologies corporation, Johnson & Johnson, has shown potential to extend the overall survival compared to chemotherapy in the second line setting according to FDA.

Prior to the full approval of Balversa, as a targeted therapy drug for bladder cancer, the disease, depending on the stage of its development, has been treated through surgery, intravesical therapy, chemotherapy, radiation and immunotherapy among other options. 

The disease, ranked as the 10th most common cancer globally, has in previous studies been linked to smoking, infection from the parasitic worm schistosoma haematobium, occupational exposure to metalworking fluids, and exposure to aromatic amines and polyaromatic hydrocarbons according to the World Cancer Research Fund International. ⁽³⁾

In Africa, the International Agency for Research on Cancer says, "bladder cancer is common in sub-Saharan Africa, but very little research on this cancer type has previously been conducted in the region." In fact, the highest bladder cancer fatalities are found in the African countries of Egypt, Algeria and Tunisia.

Researchers, the agency says, are looking into the possibility of using an inexpensive urine test, developed at IARC, "to improve the early detection of bladder cancer in this setting."

Balversa therapy, FDA cautions comes with some adverse effects on patients such as ocular disorders, hyperphosphatemia and embryo-fetal toxicity. 

Other adverse reactions, but which are estimated to be less than 20% include "laboratory abnormalities, were increased phosphate, nail disorders, stomatitis, diarrhea, increased creatinine, increased alkaline phosphate, increased alanine aminotransferase, decreased hemoglobin, decreased sodium, increased aspartate aminotransferase, fatigue, dry mouth, dry skin, decreased phosphate, decreased appetite, dysgeusia, constipation, increased calcium, dry eye, palmar-plantar erythrodysesthesia syndrome, increased potassium, alopecia, and central serous retinopathy."

Distribution of Balversa, Johnson & Johnson says, will be done through a single-source specialty pharmacy provider, US Bioservices.

"This model is part of the Company's ongoing commitment to provide high-quality products, services, access, and support to healthcare professionals and patients," Johnson & Johnson said.

European countries of Germany, Italy, Spain, Greece, Belgium Hungary have the highest bladder cancer cases globally as well as The Netherlands, Denmark, Serbia and Croatia.

The European Medicines Agency (EMA) is yet to approve any targeted drug for the treatment of bladder cancer or officially recommend the use of Balversa.

Previously, EMA had advised against the use of Keytruda (pembrolizumab) and Tecentriq (atezolizumab) as first line-treatments after patients, especially those with low levels of PD-L1, showed low survival rates as did those who were undergoing chemotherapy. ⁽⁴⁾

References

1. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-erdafitinib-locally-advanced-or-metastatic-urothelial-carcinoma

2. https://www.jnj.com/media-center/press-releases/u-s-food-and-drug-administration-grants-full-approval-for-balversa-to-treat-locally-advanced-or-metastatic-bladder-cancer-with-select-genetic-alterations#:~:text=Pharmaceuticals-,U.S.%20Food%20and%20Drug%20Administration%20Grants%20Full%20Approval%20for%20BALVERSA,Cancer%20with%20Select%20Genetic%20Alterations

3. https://www.wcrf.org/cancer-trends/bladder-cancer-statistics/

4. https://www.ema.europa.eu/en/news/ema-restricts-use-keytruda-tecentriq-bladder-cancer

About the Author:

Shem Oirere

Shem Oirere graduated from the University of South Africa with a bachelor’s degree in International Relations and Diplomacy, and also holds a Diploma in Journalism from the London School of Journalism. He previously worked for the Kenya Times, Nation Media Group and The People Daily over a twenty-year span as a business writer and Sub-editor. He wishes to share a view of the scenes behind Africa's latest pharma market trends with the rest of the world.