Scottish decision-makers have announced that fenfluramine (brand name Fintepla, developed by UCB Pharma) can be prescribed on NHS Scotland to treat seizures associated with Lennox-Gastaut Syndrome (LGS), a complex and very difficult to treat form of epilepsy that some people with TSC also have.

Funding fenfluramine in Scotland gives people with LGS, including those who also have TSC, a key option in improving epilepsy that hasn’t responded to other medicines. The TSA praises the Scottish Medicines Consortium (SMC) for coming to the right decision. Fenfluramine can now be used as an add-on therapy to other anti-epileptic medicines for patients 2 years of age and older.

The SMC’s decision highlights the importance of the TSC community voice in the decision-making process. As part of our evidence submission, we spoke to families affected by TSC-LGS, ensuring their experiences and voices were central to our arguments.

Dr Pooja Takhar, Joint Chief Executive, TSA, commented: “LGS can be incredibly difficult to treat, even more so for those who have co-diagnosis of LGS with other rare conditions like TSC. Many families will try as many as 20 anti-epileptic medicines until they reach the point where there is nothing left to try. We’re thankful to the SMC for their decision to approve this new medicine and are delighted to have another critically important treatment option in TSC-LGS.”

Nadeem Aurangzeb, Head of Rare & Epilepsy, UCB UK & Ireland, said: “LGS is a severe, rare form of childhood epilepsy which we know has a significant impact on not only those children living with it but their parents, caregivers and loved ones and UCB is delighted that eligible patients in Scotland can now access this treatment.”

Fenfluramine has been proven effective in significantly reducing ‘drop seizure’ frequency in people aged 2 to 35 years with LGS compared to placebo products. Drop seizures refer to a sudden loss of muscle strength that can cause a person to fall to the ground which can result in injuries, including broken bones.

Fenfluramine was reviewed by the New Drugs Committee (NDC) which considers the scientific evidence on new medicines, and provides recommendations to the SMC on the introduction of these medicines in Scotland. As part of their review of whether to fund a medicine, the SMC considers things like how well the medicine works, the impact it will have on individuals, and how much the medicine will cost.

Individuals and families in Scotland with TSC-LGS should contact their GP or healthcare professional to discuss whether fenfluramine is right for you. The TSA continues to work hard for the TSC community to achieve funding access to fenfluramine in other areas of the UK.