Hundreds of children with severe epilepsy will be able to benefit from a groundbreaking new treatment on the NHS that could significantly reduce their seizures, providing new hope for them and their families.
The drug, fenfluramine, will be funded for NHS patients in England from today (Thursday 20 February) after the National Institute for Health and Care Excellence (NICE) recommended it for seizures associated with Lennox–Gastaut syndrome (LGS) in people aged 2 years and over.
It is the first non-cannabis based treatment approved for this form of epilepsy.
LGS is a rare, lifelong, and treatment-resistant form of epilepsy that causes frequent, unpredictable, and debilitating seizures. In severe cases, children may experience multiple seizures throughout the day, significantly impacting their quality of life. These seizures often lead to cognitive issues, difficulties with mobility, and an increased risk of injury due to sudden loss of muscle control.
Fenfluramine is an oral liquid medicine taken daily, with the dose tailored to each patient based on their weight. It works by increasing the levels of serotonin in the brain, which helps to reduce seizure activity.
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’s commitment to ensuring access to the best therapies that deliver real benefits to patients as well as value for the taxpayer.”
Around 60,000 children in England are living with epilepsy and it is estimated that between 1 and 2% of those have Lennox-Gastaut syndrome.
Previously, the only treatment recommended by NICE for LGS was cannabidiol (Epidyolex®) with clobazam, which was approved in 2019.
LGS is highly resistant to many anti-epileptic drugs, making seizure control extremely challenging — fenfluramine now provides a crucial new option for patients who have not responded to existing treatments or for people who cannot take clobazam, due to its side effects, which can include excessive drowsiness, sedation, irritability, and dependence with long-term use.
Clinical trials have demonstrated that fenfluramine can reduce the frequency of drop seizures – which cause a loss of consciousness and muscle control – by 26.5% on average, more than three times the reduction seen in the placebo arm. More than a quarter (25.3%) of patients experienced a 50% or greater reduction in drop seizure frequency, compared to just 10.3% in the placebo group.
Clinical evidence suggests that fenfluramine not only reduces seizure frequency but also significantly reduces the risk of hospitalisation and needing emergency care.
Michael and Paul Atwal-Brice have two older boys with severe epilepsy. One – Levi – suffers from LGS. Michael said: “This is really positive news for families like ours. Levi suffers from extremely serious seizures, and having to use a cocktail of drugs to treat them has been really difficult. The side-effects have been impossible to manage as it’s hard to know which medication is causing them. Fenfluramine is potentially life-changing for Levi and our family.”
Lisa Suchet is a mum and carer of her 10-year-old son, who lives LGS. She said: “It’s wonderful news that NICE has approved Fenfluramine for patients with Lennox Gastaut syndrome.
“My son has suffered with seizures since he was 5 weeks of age, including drop seizures which are the most devastating. They occur without warning, cause immediate collapse and the risk of head injury is high due to the sudden fall. There are so few effective drugs for seizure control. Many don’t work for all patients; many have debilitating side effects or interactions with other meds a patient might have to take.
“That there is another option now available, which has shown effective results and minimal side effects is a huge comfort. It is essentially providing a chance of seizure freedom, which is essentially another chance at life really; for the patient and the family or carers who are impacted so significantly by this awful disease.”
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.”
NHS England is using its Innovative Medicines Fund to fast-track the treatment to eligible patients through an interim arrangement that will be three months faster.
This is the second indication that the NHS will offer the drug for, having commissioned fenfluramine for treating seizures caused by another rare form of epilepsy known as Dravet syndrome since 2022.
While Lennox–Gastaut syndrome most commonly develops between the ages of three and five years, today’s approval will provide access to all clinically eligible patients aged two years and over, including adult patients, with most children and young people with LGS continuing to have seizures into adult life.
Fenfluramine is manufactured by UCB Pharma, a Belgium-based pharmaceutical company and one of the UK’s largest life sciences investors.
Nadeem Aurangzeb, Head of Rare & Epilepsy, UCB UK & Ireland, said: “LGS is a rare and severe form of epilepsy which significantly impacts the quality of life of those living with it as well as those living with and caring for them. We are delighted that eligible patients in England, Wales and Northern Ireland are now able to access this treatment.”
This is the latest innovative treatment to be offered by the NHS. In January, the NHS announced a deal to offer curative sickle cell gene therapy, Casgevy®, which is being made in the UK, and follows a deal in December for the eye cancer drug tebantafusp, developed by the Oxford-based company Immunocore.
