Fintepla safe, effective for Dravet patients, real-world study finds

Clinically meaningful drop in seizure frequency seen with year of treatment

Lindsey Shapiro, PhD avatar

by Lindsey Shapiro, PhD |

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Real-world use of Fintepla (fenfluramine) was found to be safe and effective for people with Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS), according to a recent report from Spain.

Data, which showed that 60.3% of Dravet patients experienced a clinically meaningful reduction in seizures, are generally consistent with findings from randomized and controlled clinical trials of the therapy.

“This study contributes to the growing body of evidence supporting the effectiveness of [Fintepla] in the real-world management of DS and LGS,” the researchers wrote.

The study, “What have we learned from the real-world efficacy of FFA in DS and LGS? A post-marketing study in clinical practice,” was published in Epilepsy & Behavior.

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Fintepla is approved for Dravet syndrome patients ages 2 and older

Fintepla, marketed by UCB, is an approved add-on treatment to standard anti-epileptic therapies for Dravet syndrome patients, ages 2 and older, in the U.S., Europe, and Japan. It’s also approved in the U.S. and Europe to treat LGS, a related form of childhood epilepsy.

While it is not clear exactly how Fintepla helps to prevent disease-related seizures, it is believed to do so by binding to certain receptor proteins, namely serotonin and sigma receptors, found in nerve cells.

Clinical trial evidence indicates that Fintepla eases seizure frequency and severity, and boosts life quality while maintaining a favorable safety profile. However, data related to its real-world use in routine clinical practice are lacking.

“It is essential to ascertain whether the promising outcomes observed in [clinical trials] can be translated into real-world clinical settings, where factors such as medication adherence, concomitant [anti-seizure medications] and other medications, and comorbidities may influence treatment outcomes,” the team wrote.

Scientists, all with various hospitals and institutes in Spain, conducted a retrospective analysis of 68 patients who initiated Fintepla at the Ruber International Hospital in Madrid between 2018 and 2022. About half  (51.5%) started on Fintepla under a compassionate use program, while the others began treatment in the open-label extension phase of a clinical trial.

Among the patients, whose median age was 12, 54 were diagnosed with Dravet or related forms of childhood epilepsy, and 14 had LGS. Patients were followed for a median of 25.1 months, or a little over two years.

The median daily dosage of the oral medication was 0.4 mg/kg of body weight in Dravet patients, with those also taking Diacomit (stiripentol) given a lower dose. Patients used Fintepla for a median of 755 days, or about two years.

A 50% or greater reduction in seizure frequency seen at one year

Prior to starting Fintepla, Dravet patients were experiencing an average of 24 seizures each month. For those who remained on Fintepla after a year, 60.3% achieved at least a 50% reduction in seizures and 10.5% were seizure free. No differences in sustained reduced and seizure-free rates were seen between patients younger than age 12 and those ages 12 and older at one year of treatment.

Subjective cognitive improvements were also reported for 73.3% of Dravet patients, while findings of motor or social gains were “relatively modest.”

Nearly three-quarters (72.2%) of Dravet patients were able to reduce doses of their other anti-seizure medications, and 31.4% were able to stop using at least one of those medications.

In general, Fintepla’s efficacy among Lennox-Gastaut patients was similar to those with Dravet, with no significant differences between the two groups.

Across the entire study population, 59.7% of patients experienced at least one side effect while using Fintepla, most of which (86.5%) were considered to be plausibly related to treatment. The most common effects were decreased appetite, sleepiness, irritability, dizziness, diarrhea, and nausea.

Four patients stopped using the medication due to side effects. There were no occurrences of heart valve disease or pulmonary arterial hypertension — two serious cardiovascular complications that have been associated with higher doses of the medication used for other purposes, but largely not observed in Dravet clinical trials.

Studies urged into the effectiveness of Fintepla at lower doses

Side effects often resolved without any intervention (43.1%), but some required a dose reduction or discontinuation. Patients with persistent side effects were significantly more likely to be receiving a higher dose of Fintepla. The median adjusted dose at which side effects became evident was 0.4 mg/kg (range, 0.2 to 0.6 mg/kg), the study noted.

“Further investigation to determine whether lower doses of the drug could be an effective option for the treatment of these patients is warranted,” the researchers noted.

Altogether, the safety and efficacy profile of Fintepla was consistent with the effects observed in controlled clinical trials, they added.

“The significant and sustained reduction in seizure frequency and the achievement of seizure freedom … highlight the potential of [Fintepla] as an effective treatment option,” the researchers wrote, noting that the side effect profile, “underscores the importance of careful monitoring and patient selection.”

The writing of this study was supported by UCB.