The European Commission (EC) has approved UCB’s Fintepla (fenfluramine) oral solution as an add-on treatment for seizures associated with Lennox-Gastaut syndrome (LGS), a severe form of epilepsy.
The disease, which affects around two in 10,000 people in the EU, usually begins in early childhood and is characterised by multiple types of drug-resistant seizures with high morbidity, as well as serious impairment of neurodevelopmental, cognitive and motor functions.
“LGS is a developmental and epileptic encephalopathy where seizures are frequent, inducing high level of trauma injuries and negatively impacting development and quality of life,” said Rima Nabbout, professor of paediatric neurology at University Paris cité, APHP, Necker Enfants Malades, Institut Imagine.
“Seizures are often resistant to currently available medications, making this approval especially important for the individuals affected and their families,” Nabbout said.
The EC’s decision, which indicates the therapy for patients aged from two years, was based on results from a phase 3 trial in 263 LGS patients, aged two to 35 years.
Results showed that a daily dose of Fintepla, in combination with other anti-epileptic medicines, provided a significantly greater reduction in the frequency of seizures leading to falls compared to placebo.
The most common treatment-emergent adverse events were decreased appetite, fatigue and fever, the company reported, and no cases of valvular heart disease or pulmonary arterial hypertension were observed.
Along with its approval, the EC has also adopted the European Medicines Agency Committee for Orphan Medicinal Products’ recommendation that the orphan designation for fenfluramine be maintained.
Mike Davis, head of global epilepsy and rare syndromes at UCB, said: “With this approval, [Fintepla] is now an important additional treatment option for those impacted by this difficult to treat condition in Europe.
“This approval underscores our commitment to improving treatment outcomes, while addressing the high unmet need for new treatments for people living with LGS and rare epilepsies.”
Fintepla is already approved in the US for the treatment of seizures associated with LGS, as well as for Dravet syndrome – a rare and severe form of epilepsy – in the EU, the US and Japan.
Dravet syndrome generally begins in infancy and is marked by frequent, treatment-resistant seizures, significant developmental, motor and behavioural impairments, as well as an increased risk of mortality.