Sep. 14, 2022
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The U.S. Food and Drug Administration (FDA) has approved fenfluramine (Fintepla®) for the treatment of seizures associated with Lennox-Gastaut syndrome in patients 2 years of age and older. Last year, the orphan drug was approved for treating seizures in children with Dravet syndrome.
Lennox-Gastaut syndrome is a rare neurologic condition that usually begins in childhood. It is characterized by multiple types of seizures and intellectual disability. This condition can be caused by brain malformations, perinatal asphyxia, severe head injury, central nervous system infection, and inherited degenerative or metabolic conditions. In about one-third of cases, no cause can be found.
The drug approval was largely based on results of a randomized, placebo-controlled phase 3 clinical trial in 263 patients with Lennox-Gastaut syndrome. The trial demonstrated that fenfluramine at a dose of 0.7 mg/kg/day significantly reduced monthly drop seizures frequency by a median of 23.7% from baseline compared to 8.7% placebo (P = .0037). Common adverse reactions in patients treated with fenfluramine were diarrhea, decreased appetite, fatigue, somnolence, and vomiting.
In a news release, Kelly Knupp MD of the Children’s Hospital Colorado stated, “Lennox-Gastaut syndrome is one of the most challenging epileptic encephalopathies to treat, and the vast majority of patients are not well controlled, despite a regimen of multiple antiepileptic drugs,” adding, “as a complementary therapy, fenfluramine offers a different mechanism of action and demonstrated ability to significantly reduce the number of seizures associated with a drop, a critical measure for managing this severe form of epilepsy.”
Last year, CheckRare talked to Arnold Gammaitoni PharmD about that phase 3 clinical trial that led to the approval (see video here).
Source: News Release
March 29, 2022