Fenfluramine (Fintepla) oral solution has been approved by the European Commission (EC) as adjunctive therapy for seizures associated with Lennox-Gastaut syndrome (LGS) in children aged 2 years and older, the company has announced.
The EC approval comes just under a year after US Food and Drug Administration (FDA) approval for this indication, as reported last March by Medscape Medical News.
Fenfluramine is already approved in the US and Europe to treat seizures associated with Dravet syndrome.
LGS is a severe form of epilepsy characterized by multiple types of seizures, including "drop" seizures and convulsive seizures, leading to high morbidity and impairment in neurodevelopmental and cognitive and motor function. The disorder is also associated with significantly increased risk for sudden unexpected death in epilepsy (SUDEP).
Seizures associated with LGS are often resistant to currently available medications, "making this approval especially important for the individuals affected and their families," Rima Nabbout, MD, PhD, Necker Enfants Malades Hospital, Paris, France, said in a company news release.
The approval was supported by safety and efficacy data from a global, randomized, placebo-controlled, phase 3 clinical trial involving 263 patients aged 2-35 with LGS. Its results were published in JAMA Neurology last year.
As reported by Medscape Medical News, the trial met its primary efficacy endpoint. Children taking fenfluramine at a dose of 0.7 mg/kg/day had significantly fewer drop seizures than peers taking placebo. The estimated mean between-group difference in drop-seizure frequency was –19.9% (P = .001).
The median percentage reduction in the frequency of drop seizures with fenfluramine was 26.5% vs 7.6% with placebo (P = .09). One quarter of children taking the drug experienced at least a 50% reduction in drop-seizure frequency compared with 10% of children taking placebo.
The most common treatment-emergent adverse events were decreased appetite, somnolence, fatigue, and fever. No cases of valvular heart disease or pulmonary arterial hypertension were observed.
In Europe, fenfluramine oral solution is available under a controlled access program to ensure regular cardiac monitoring and to mitigate potential off-label use.
In addition to approving fenfluramine for LGS, the EC also adopted the EMA Committee for Orphan Medicinal Products (COMP) recommendation that the orphan designation for the drug be maintained. LGS affects about 2 in 10,000 people in the European Union (EU).
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Cite this: EU Approves Fenfluramine for Lennox-Gastaut Syndrome - Medscape - Feb 10, 2023.