A new treatment for Duchenne
muscular dystrophy (DMD) is set to be available to around 1,700
people in England following positive final draft guidance from
NICE published today (Tuesday, 10 December
2024).
Vamorolone (also called
Agamree and made by
Santhera), is an alternative to
currently available corticosteroids used to
reduce muscle inflammation and
maintain muscle strength and function in people with
DMD.
NICE's previous draft guidance
concluded that uncertainties in the clinical evidence and
economic model meant that more
evidence was needed to establish the cost-effectiveness of
vamorolone.
New analysis presented by the company,
together with an improved discount to the price of vamorolone,
mean that NICE can now recommend it as a cost-effective use of
NHS resources for people
4 years and over.
Side effects of currently available
corticosteroids can include osteoporosis, reduced bone strength,
increased risk of spinal fractures, weight gain, negative
behaviour changes, growth restriction, reduced bone density and
delayed puberty.
Vamorolone could offer important
benefits because of its potential to reduce adverse events
associated with corticosteroids.
Vamorolone, becomes one of several
non-cancer treatments recommended by NICE following the
introduction of the new severity modifier. This allows NICE's
independent committees to give
greater weight to severe conditions, including conditions such as
DMD, that would not have qualified for additional weighting under
the previous end of life
criteria.
The committee agreed that a severity
weight of 1.7 was appropriate to reflect the severity of
DMD.
Helen Knight, director of
medicines evaluation at NICE, said:
“The committee felt
vamorolone could offer important benefits to patients and their families because of its potential to reduce adverse events
associated with current steroid
treatments.
“Given the significant burden this
disease places on children with the condition and on their
parents and carers, as well as the negative impact this can have
on their quality of life, a treatment with the potential to
reduce side effects associated with current treatments is to be
welcomed.
“We're therefore delighted the company
has been able to work with us to enable vamorolone to be made
available to people with
DMD.”
One of a group of muscular
dystrophies, DMD is a severe, progressive muscle-wasting genetic
condition caused by the lack of a protein called
dystrophin, a protein important
for muscle cells.
Present from birth and with symptoms
that usually appear at around age 3, DMD leads to gradual decline in muscle function with
children often needing to use a wheelchair by early adolescence
and eventually requiring artificial ventilation to
breathe.
Ends
Notes for
editors
An embargoed copy of the final draft
guidance for Vamorolone for treating Duchenne muscular dystrophy
is attached below:
ID4024 vamorolone DMD
final draft guidance to PM for appeal noCON.docx
