New life-changing treatment option recommended to help control seizures for type of rare epilepsy
This marks a significant step forward for around 1,400 people living with this rare and severe form of epilepsy, which typically begins in early childhood.
In our final draft guidance published today (20 February) we've recommended fenfluramine (also called Fintepla and made by UCB) as an add-on to other antiseizure medicines for treating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. 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.
Today’s positive recommendation follows an appeal by the company as well as an offer of an improved discount to its price. It changes our previous final draft guidance that did not recommend the treatment.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 we recommend 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.
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.
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.
Professor Powis continued: “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."
We already recommend fenfluramine for Dravet syndrome, another type of rare childhood epilepsy.