NHS Funds “Potentially Life-changing” Treatment for Severe Form of Epilepsy

There’s some exciting news in the world of epilepsy treatments, particularly for children suffering from a severe form of epilepsy. On February 20, 2025, the epilepsy medication fenfluramine became available for NHS patients with Lennox-Gastaut syndrome. This ground-breaking treatment is seen as “potentially life-changing” for hundreds of families across the UK.

What is Lennox-Gastaut syndrome?

Lennox-Gastaut syndrome, or LGS, is a rare, lifelong form of epilepsy that causes unpredictable and severe seizures, typically starting in early childhood (between ages 2 and 5).

In severe cases, children may suffer from multiple seizures daily, significantly impacting their quality of life. These seizures can lead to cognitive difficulties, mobility issues, and a higher risk of injury from sudden muscle control loss.

LGS affects about 1 to 2% of the 60,000 children living with epilepsy in the UK. That’s a significant number of families facing these challenges every day.

Here’s what some of those families have to say…

Michael and Paul Atwal-Brice are the parents of two boys who struggle with severe epilepsy. One of them, Levi, has Lennox-Gastaut Syndrome (LGS). Micheal explains they’ve faced many challenges in managing Levi’s seizures, such as navigating different medications and dealing with their many side effects. When asked about this new medication, he states, “Fenfluramine is potentially life-changing for Levi and our family.”

Lisa Suchet is a devoted mother and caregiver to her 10-year-old son, who lives with LGS. She shared the constant fear that comes with his unpredictable drop seizures and the potential injuries associated with them. This new treatment brings them a renewed opportunity for a better life. As she expressed, “It is essentially providing a chance of seizure freedom, which is essentially another chance at life really…”

“Potentially life-changing” Medication

So what is this new medication making headlines? It’s called fenfluramine, and it’s an oral medication taken daily as a liquid.

How does it work?
Fenfluramine boosts serotonin levels in the brain, also known as the “feel-good” chemical. While it plays a significant role in enhancing mood, it’s also essential for various brain functions. Essentially, it helps fine-tune electrical activity in the brain. Disruptions in these electrical signals can cause seizures, but fenfluramine aids in restoring balance, helping everything to run more smoothly.

Before fenfluramine, the only treatment approved by NICE* for Lennox-Gastaut syndrome was a combination of cannabidiol (Epidyolex) and clobazam, approved in 2019. With the introduction of fenfluramine, we now have the first non-cannabis option for treating LGS.

LGS is particularly resistant to a variety of anti-seizure medications. Therefore, many children with LGS don’t respond to existing treatments or cannot tolerate the side effects, such as drowsiness and irritability. However, fenfluramine now offers a vital new option for patients who haven’t found relief with other treatments, or for those who are unable to take clobazam due to its side effects.

Of course, no medication is completely free from risks. It’s always important to weigh both the potential downsides and benefits of a new medication. Some issues may be manageable, while others could be more serious. Regular monitoring and effective communication between patients, their families, and healthcare providers can help minimise side effects. Ongoing research is still looking into the long-term effects of fenfluramine.

Clinical trials show impressive results, significantly reducing the frequency of Atonic seizures. (Atonic seizures, or drop seizures, cause a sudden loss of muscle control in a person. This can cause the person to feel very weak and limp, which may result in a fall.)

By reducing the frequency of these seizures, the risk of injury from falls is also reduced, leading to an improved quality of life. This sets off a domino effect of positive outcomes, benefiting both children and their families.

According to the clinical trials, fenfluramine has been shown on average to lower the frequency of drop seizures by 26.5%, which is more than three times the improvement seen with a placebo. Additionally, over a quarter of patients (25.3%) saw their drop seizures cut in half or more, compared to only 10.3% of those taking the placebo.

The NHS is using its innovative medicines fund to allow patients access to fenfluramine three months sooner than usual. Before this, the NHS began commissioning fenfluramine for treating another form of epilepsy, known as Dravet syndrome.

While Dravet and Lennox-Gastaut syndrome (LGS) are different conditions, they share some molecular similarities that fenfluramine can effectively target. This medication does not merely address symptoms; it tackles the root cause of these conditions.

The approval of such treatment marks a significant turning point for rare diseases, which have long been overlooked due to their impact on a small number of individuals. While these diseases may seem rare in isolation, together, they affect millions of people worldwide.

The personal stories we’ve discussed highlight that research and development go beyond mere data; they are about real lives and providing hope for families who have faced challenges for too long. This reinforces the need to advocate for those with rare diseases. Everyone deserves access to effective treatments.

*The National Institute for Health and Care Excellence (NICE) is an organisation that offers advice and guidance to improve health and social care across the country. Its main goal is to help doctors, nurses, and other health professionals by creating recommendations based on the best research and information available.

This information is for informational purposes only and should not replace professional medical advice. Always consult a healthcare professional for proper diagnosis and appropriate treatment.