The National Institute for Health and Care Excellence (NICE) has recommended erdafitinib (Balversa, Johnson & Johnson) as a treatment option for adults with unresectable or metastatic urothelial carcinoma.
The drug is approved for patients with fibroblast growth factor receptor 3 (FGFR3) genetic alterations who have previously received at least one therapy containing a PD-1 or PD-L1 inhibitor.
In a final draft guidance, NICE estimated that around 423 patients each year could benefit from the therapy.
Targeted Treatment for FGFR3 Mutations
Erdafitinib inhibits FGFR, a family of receptor tyrosine kinases that promote uncontrolled growth of urothelial carcinomas.
Alterations in the FGFR3 gene are found in 15%-20% of advanced urothelial cancer cases. These mutations are particularly common in the luminal subtype of the disease.
Clinical experts told NICE that such tumours may grow more slowly but respond less well to chemotherapy or immunotherapy.
Significant Disease Burden
Urothelial carcinomas originate in the lining of the bladder, urethra, ureter, or renal pelvis. They account for approximately 90% of all bladder cancers.
Around 10,000 new bladder cancer cases are diagnosed in the UK each year. Five-year survival ranges from 80% for stage 1 tumours to 10% for stage 4 tumours.
The disease is more common in men and in people over age 75. Smoking is linked to about half of all cases.
Symptoms can include haematuria, dysuria, and pain. Advanced disease may cause pelvic pain, bone pain, weight loss, leg swelling, lower back pain, and fatigue.
Improved Survival Outcomes
NICE said that erdafitinib had not been directly compared with standard treatments, such as paclitaxel, carboplatin, or best supportive care. However, indirect comparisons showed that the drug improved both progression-free and overall survival.
In a 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, median overall survival improved by over 4 months. Progression-free survival more than doubled — from 2.73 to 5.55 months.
Patients and Advocates Welcome Approval
Patients told NICE that current treatments involve frequent hospital visits and significant side effects. Those affected are often shocked to discover the disease’s poor prognosis and the limited treatment options available.
Helen Knight, director of medicines evaluation at NICE, said the oral drug can be taken at home, sparing patients travel to hospital and improving quality of life. The targeted treatment option would be welcomed by patients and would make “a significant difference to people’s lives,” she said.
Jeannie Rigby, chief executive of Action Bladder Cancer UK, also welcomed the decision. “We hope this is just the start of new hope for those with bladder cancer and their families,” she said.
Cost and Availability
The list price for erdafitinib is £12,750 per 28-day cycle, excluding VAT.
The company has agreed to supply the drug under a commercial arrangement with the NHS, offering a confidential discount.
NICE initially declined to recommend the drug due to uncertainty around comparators and economic modelling. Following additional data, NICE said the cost-effectiveness estimates now fall within its acceptable range.
The therapy will be available on the NHS immediately. Final guidance is due on 12 May 2025.
Dr Sheena Meredith is an established medical writer, editor, and consultant in healthcare communications, with extensive experience writing for medical professionals and the general public. She is qualified in medicine and in law and medical ethics.