Fintepla brings meaningful drop in Dravet seizures: Real-world study
Frequent monthly seizures for many patients fall by 50% or more with treatment
Two-thirds of 24 children and young adults with Dravet syndrome experienced clinically meaningful declines in seizure frequency after adding Fintepla (fenfluramine) to their treatment regimen, according to a real-world Danish analysis.
Fintepla’s use also was associated with reductions in healthcare utilization and the use of other anti-seizure medications.
The article, “Fenfluramine treatment in pediatric patients with Dravet syndrome reduces seizure burden and overall healthcare costs: A retrospective and observational real-world study,” was published in Epilepsia. The work was supported by UCB, which markets the treatment.
Fintepla is an approved add-on therapy for children and adults with Dravet
Fintepla has been approved in both the U.S. and Europe since 2020 as an add-on to standard anti-seizure medicines for Dravet patients, ages 2 and older.
While its anti-seizure mechanisms are poorly understood, the oral treatment is thought to enhance the release of serotonin, a brain signaling chemical, and to positively modulate proteins called sigma-1 opioid receptors.
International guidelines on diagnosing and managing Dravet have suggested that Fintepla could be used as a first- or second-line maintenance therapy for keeping seizures under control. Still, data are limited on the treatment’s efficacy in real-world settings.
Researchers in Denmark looked at findings from a retrospective analysis of clinical outcomes in 30 Dravet patients treated with Fintepla at an epilepsy center in Dianalund between 2017 and 2023.
Patients, ages 3-21 years, started treatment at a mean age of 9 and used the therapy for a mean of 29 months, or just under 2.5 years. At the time of treatment initiation, 33% of these patients had from five up to 10 generalized tonic-clonic seizures each month, and nearly half (47%) had 10 or more of these monthly seizures.
Six of 24 patients stopped having generalized tonic-clonic seizures
Six patients discontinued treatment due to a lack of efficacy or side effects, all within the first year of its use.
The remaining 24 patients were still on treatment at the study’s end in December 2023, and had been treated for up to six years.
Among them, six (25%) were free of generalized tonic-clonic seizures at that time. Three stopped having these seizures within six and 12 months of treatment, and the other three within two and three years of treatment.
Generalized tonic-clonic seizures are the seizure type most people are familiar with, characterized by convulsions and a loss of consciousness. While they are not the only type experienced by Dravet patients, they’re a good indicator of a treatment response because they’re easily recorded and often severe.
Two-thirds of patients saw at least a 50% reduction in these seizures, and most patients (83%) experienced at least a 30% reduction. According to the Danish Medicines Agency, a seizure reduction of at least 30% is required to warrant continued use of Fintepla, so the researchers considered those above this cutoff as treatment responders.
The four patients with less than a 30% reduction continued treatment in the study due to other reported benefits, such as shorter and less intense seizures or fewer frequent episodes of status epilepticus, a prolonged period of seizure activity that’s a medical emergency.
Researchers noted that these other outcomes are “of great importance when evaluating the response to treatment.”
Lesser need for other treatments, professional help seen with treatment
When starting Fintepla, patients were using a mean of three anti-seizure medications. Almost three-quarters (71%) of those who stayed on treatment were able to lower their medication use, with the most frequently discontinued therapy being Diacomit (stiripentol).
In addition, 75% experienced a reduction — by a mean of 52% — in epilepsy-related contact with nurses or pediatricians at the hospital while on Fintepla. The median number of yearly epilepsy-related contacts fell from 25 to six per patient over two years of treatment.
This decline helps in perceiving whether Fintepla is a cost-effective treatment option, the scientists noted, as hospital contacts are a measure of burden on a healthcare system.
“This indirectly proves the effectiveness of fenfluramine treatment and indicates cost-effective benefits,” the researchers wrote.
Among all 30 patients, a little over half (53%) reported Fintepla-related adverse events, including problems with balance, lesser appetite, increased seizure frequency, tiredness, and tremor. No patients reported adverse events after the first year of treatment.
A limitation to these findings was the lack of reporting on outcomes such as life quality or cognitive performance, the scientists noted.
“Studies designed for detecting non-seizure-related improvements … are needed and are planned to be conducted by this group … to further expand the knowledge of treatment with [Fintepla] in patients with [Dravet syndrome],” the team concluded.
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