Hundreds of people in England to benefit from take-at-home tablet bladder cancer treatment

10 Apr 2025 08:43 AM

Today, for the first time, we have given the green light to a targeted treatment for the most common form of bladder cancer.

Erdafitinib, also known as Balversa and made by Johnson & Johnson, is recommended in final draft guidance for adults with metastatic or unresectable FGFR-altered urothelial cancer who have previously received at least one line of therapy containing a PD1 or PD-L1 inhibitor. 

Urothelial cancer occurs in the cells that form the inner lining of the bladder, urethra, ureter, or renal pelvis, with most cases originating in the bladder. Urothelial cancer accounts for around 90% of all bladder cancers. 

In advanced bladder cancer, symptoms may include pelvic or bone pain, unintentional weight loss, swelling in the legs, difficulty urinating, lower back pain, and fatigue. 

Just over 18,000 people in England are diagnosed with bladder cancer each year and it is estimated that around 423 people would be able to benefit from this new drug, which is available in the NHS from today.

Erdafitinib, which is given as a tablet taken once a day, works by blocking proteins in the body called FGFR3 tyrosine kinases which may be overactive in some urothelial cancers due to mutations. This helps to slow down or stop the growth of cancer cells. It is the first targeted treatment for unresectable (meaning it cannot be removed by surgery) or metastatic (meaning it has spread to other parts of the body) urothelial cancer. 

The standard treatment for unresectable or metastatic urothelial cancer is either platinum-based chemotherapy (cisplatin or carboplatin) or, for people whose cancer is PD-L1 positive, atezolizumab. Once these treatment options have been exhausted, people may also be treated with paclitaxel with or without carboplatin chemotherapy or best supportive care. 

A clinical trial comparing erdafitinib with chemotherapy (vinflunine or docetaxel) in people with advanced urothelial cancer and FGFR alterations whose condition had progressed on or after one or two prior treatments found that overall survival increased by over four months for people taking erdafitinib compared with chemotherapy. 

The trial also found that progression-free survival more than doubled, with people taking erdafitinib averaging 5.55 months, compared with an average of 2.73 months for people on chemotherapy. 

I am pleased we have been able to recommend this effective, targeted treatment option which will make a significant difference to people’s lives. 

There are limited treatment options for this devastating and debilitating disease, which in many cases responds poorly to immunotherapies, so I’m sure today’s guidance will be welcomed by patients. 

Helen Knight, director of medicines evaluation at NICE

Helen continued: "One of the benefits of this drug is it can be taken in the comfort of the patients’ own home rather than having to travel to hospital to receive the treatment. NICE is determined to ensure NHS patients in England have access to the most promising new treatments while providing value for the taxpayer."

ABC UK and bladder cancer patients welcome the approval of erdafitinib for use in England. There are very few treatments available for bladder cancer in general, and particularly for people with advanced disease. It’s time that bladder cancer patients had some kind of new treatment available – we hope this is just the start of new hope for those with bladder cancer and their families.

Jeannie Rigby, chief executive, Action Bladder Cancer UK

The drug was not recommended at draft guidance due to uncertainties in comparators and economic modelling but after the company provided additional data and information the medicine was approved.

Our independent appraisal committee agreed that a severity weight of 1.7 was appropriate to reflect the seriousness of the disease and the lack of treatment options.

This allows our independent committees to give greater weight to severe conditions, which would not have qualified for additional weighting under the previous end of life criteria.  

The company has a confidential commercial arrangement through a simple discount patient access scheme which makes erdafitinib available to the NHS.