Hundreds of people in England are set to benefit after NICE today
(Thursday, 10 April), for the first time, gave 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Johnson, is recommended in final draft guidance for adults with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.
Helen Knight, director of medicines evaluation at NICE, said: “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.
“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."
Jeannie Rigby, Chief Executive, Action Bladder Cancer UK, said:
“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.”
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.
NICE's 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 NICE's 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.
Read the full final draft guidance for erdafitinib for treating
metastatic or unresectable FGFR-altered urothelial
cancer.
