In final draft guidance published
today (Thursday, 20 February 2025) NICE has recommended a new
treatment option for around 1,400 people with a type of rare,
hard-to-treat epilepsy.
The final draft guidance recommends
fenfluramine (also called Fintepla and made by UCB) as an add-on to other antiseizure medicines for treating
seizures associated with Lennox–Gastaut syndrome (LGS) for people
2 years and over.
LGS is a severely debilitating form of
epilepsy that begins in early childhood between the ages of 2 and
7 years. It is characterised by frequent seizures of different
types. Drop seizures result in a loss of muscle tone or
stiffening of muscles, and people can fall suddenly to the
ground. This may result in severe injuries and hospitalisation.
The condition is also associated with severe learning and
behavioural disorders.
People with the condition need
round-the-clock care and help with almost all aspects of daily
life. Approximately 5% of children with LGS will die during
childhood.
Today's positive recommendation
follows an appeal by the company as well as an offer of an
improved discount to its price. It changes NICE's previous final
draft guidance that did not recommend the
treatment.
At its subsequent meeting the
committee accepted the company's new proposal that fenfluramine
be considered using a cost comparison
approach. To get a positive
recommendation the company needed to demonstrate that
fenfluramine provides similar or greater health benefits to
cannabidiol plus clobazam (which is recommended by NICE for this
population and is already in use in the NHS) at a similar or
lower cost.
Fenfluramine is recommended if the
frequency of drop seizures is checked every 6 months and is
stopped if the frequency is not reduced by at least 30% compared
with the 6 months before starting
treatment.
Helen Knight, director of
medicines evaluation at NICE, said:
“The often distressing
and life-limiting nature of this very difficult to control
epilepsy means that any new treatment options are particularly
welcome.
“In recommending fenfluramine the
independent committee took into account the rarity and severity
of Lennox-Gastaut syndrome, the significant impact it has
on the quality of life of
people with the condition and their families and carers,
and the high need for effective
treatments, particularly for people who aren't able to take
cannabidiol with clobazam.”
Current treatments often do not
control seizures caused by LGS. The patient carer experts who
gave evidence to the committee noted that the currently available
drugs that make up standard care become less effective over
time.
Professor Stephen Powis,
National Medical Director for NHS England
said: “For
children and families living with Lennox–Gastaut syndrome, every
day can be challenging, facing unpredictable and life-limiting
seizures, and this new treatment option on the NHS will now offer new hope,
giving many the chance for greater stability and a better quality
of life.
“To have a proven, evidence-based new
medicine that can be taken at home to help control and reduce
their child's seizures, and for example lower the risk of them
experiencing injuries and needing to go to hospital, is fantastic
news for hundreds of families.
“Fenfluramine will offer a vital
alternative for those who can't tolerate existing cannabis-based
treatment and the fast-tracking of this treatment to be available
from today is another example of the NHS' commitment to ensuring
access to the best therapies that deliver real benefits to
patients as well as value for the
taxpayer."
NICE already recommends fenfluramine for
Dravet syndrome, another type of rare childhood
epilepsy.
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The final draft guidance for
fenfluramine is available on the NICE website (from 00:01,
Thursday 20 February 2025) at: https://www.nice.org.uk/guidance/indevelopment/gid-ta10653/documents
