UCB’s FINTEPLA (fenfluramine) oral solution has been approved in the European Union (EU) for the treatment of seizures associated with Lennox-Gastaut Syndrome (LGS) as an add-on therapy to other anti-epileptic medicines for patients two years of age and older.
Key highlights:
- FINTEPLA (fenfluramine) oral solution has been approved in the EU for adjunctive treatment of seizures associated with Lennox-Gastaut syndrome (LGS).
- A Phase 3 clinical trial demonstrated that FINTEPLA (fenfluramine) significantly reduced the frequency of drop seizures, when given as an add-on therapy to other anti-epileptic medicines.
- LGS causes drug-resistant seizures and other severe symptoms, including impairment of neurodevelopmental, cognitive, and motor functions. Symptoms persist throughout childhood into adulthood, resulting in potential early mortality.
The approval by the European Commission (EC) was based on safety and efficacy data from a global, randomised, placebo-controlled Phase 3 clinical trial, in 263 patients with LGS (aged 2-35 years), that demonstrated adjunctive fenfluramine at a dose of 0.7/mg/kg/day provided a significantly greater reduction in the frequency of drop seizures (p=0.001) compared to placebo.
The most common treatment-emergent adverse events were decreased appetite, somnolence, fatigue, and pyrexia (fever). No cases of valvular heart disease or pulmonary arterial hypertension were observed.
Professor Rima Nabbout, MD, PhD, professor of Paediatric Neurology at University Paris cité, APHP, Necker Enfants Malades, Institut Imagine, Paris, France, said: “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. Seizures are often resistant to currently available medications, making this approval especially important for the individuals affected and their families.”
Mike Davis, head of global epilepsy & rare syndromes, UCB, said: “With this approval, fenfluramine 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 for the treatment of Dravet Syndrome but Lennox-Gastaut Syndrome affects a unique group of patients and caregivers. There are roughly 20-30k LGS patients in the EU – seizures remain largely uncontrolled on current medications, and there is a significant concern for sudden unexpected death.
LGS is a severe childhood-onset developmental and epileptic encephalopathy (DEE) characterised by multiple types of drug-resistant seizures with high morbidity, as well as serious impairment of neurodevelopmental, cognitive, and motor functions, affecting an estimated 2 in 10,000 people in European Union (EU).
Seizures leading to falls are common in LGS and tonic seizures are a hallmark feature of this syndrome. In addition, convulsive seizures (e.g. generalised tonic-clonic [GTC] seizures) are also commonly observed and usually occur in later stages of LGS, but sometimes may precede core seizure types.
In addition to being associated with bodily injury and hospitalisations, GTC seizures are a primary risk factor of sudden unexpected death in epilepsy (SUDEP). Patients with GTC seizures have an approximately 10-fold greater risk for SUDEP than patients with other seizure types.
Additionally, the EC has also adopted the EMA Committee for Orphan Medicinal Products (COMP) recommendation that the orphan designation for fenfluramine be maintained.