A new treatment option has become available for severe epilepsy: fenfluramine, which is now available on the NHS for children and adults living with Lennox–Gastaut syndrome (LGS)
The drug, which has been approved by the National Institute for Health and Care Excellence (NICE), is expected to significantly reduce seizures and help families and patients battling this condition.
What is Lennox–Gastaut syndrome?
Lennox–Gastaut syndrome is a rare and treatment-resistant form of epilepsy, often beginning between the ages of three and five.
Distinguished by frequent, unexpected seizures, including dangerous drop seizures, LGS can severely impact a child’s life.
These seizures result in a sudden loss of muscle control and consciousness, leading to falls and a high risk of injury. Many children with LGS face cognitive challenges and mobility issues, with traditional treatments often failing to provide proper relief.
Treating Lennox–Gastaut syndrome
Now, the liquid oral medication fenfluramine is available, and patients are able to work on increasing serotonin levels in the brain, helping to reduce seizure frequency and severity.
This medication is to be taken daily with a dosage tailored to each patient based on their weight and will overall help families struggling with seizure control.
It is the first non-cannabis-based treatment approved for LGS, providing a good alternative for children who cannot tolerate existing treatments, including cannabidiol (Epidyolex®) with clobazam, which has been the only NICE-approved option until now.
Reducing seizure frequency
Clinical trials have shown the effectiveness of fenfluramine in reducing drop seizures. On average, patients experienced a 26.5% reduction in seizure frequency, with over a quarter of participants seeing a 50% or greater reduction.
In comparison, only 10.3% of those in the placebo group showed such improvements. Further reducing seizure frequency, fenfluramine has also been shown to lower the risk of hospitalisations and emergency interventions, offering a significant quality-of-life improvement for patients and their families.
The approval of fenfluramine comes as part of an ongoing effort by the NHS to provide access to innovative treatments. The NHS is fast-tracking the drug’s availability through an interim arrangement, allowing eligible patients to access it three months earlier than usual. This move is part of the NHS’s commitment to making effective therapies accessible to those in need. Fenfluramine is also being used to treat Dravet syndrome, another rare form of epilepsy, having been commissioned for this purpose in 2022.
A new option to treat LGS
Around 60,000 children in England live with epilepsy, and approximately 1-2% of them are affected by LGS. While LGS is known for being difficult to treat, fenfluramine’s success in clinical trials offers a new option for patients who have not responded to other treatments.
For those unable to take clobazam due to its side effects, fenfluramine represents a crucial breakthrough. Clobazam can cause drowsiness, irritability, and long-term dependence, making it unsuitable for some patients.
Fenfluramine’s approval also brings relief to families like that of Levi, a child suffering from LGS, who has struggled with severe seizures despite a range of medications.
The treatment’s potential to reduce the frequency of life-threatening seizures is seen as a life-changing development, offering hope to families facing the daily challenges of managing the condition.
This new treatment for LGS is part of a bigger trend of innovative therapies being introduced to the NHS. Earlier in the year, the NHS also announced plans to provide curative gene therapy for sickle cell disease and a new treatment for eye cancer, reinforcing its commitment to offering cutting-edge medical solutions to improve patient outcomes.
Fenfluramine is manufactured by UCB Pharma, a major player in the pharmaceutical industry, and is now available to eligible patients across England, Wales, and Northern Ireland.
