FenfluramineFintepla
Treatment of seizures associated with Dravet Syndrome in patients not adequately controlled despite treatment with at least 2 other antiepileptic drugs.
Decisions on record
- Meeting Nov 2025 Recommended Lennox-Gastaut Syndrome – add-on therapy in patients not adequately controlled with ≥2 other anti-seizure medications no PSD
- Meeting Nov 2024 Recommended Seizures associated with Dravet Syndrome
Access path
- TGA registered · Fintepla
TGA label narrower than the PBS population
- Nov 2024Recommended · restricted
vs cannabidiol
- PBS listing · Authority Required
From the public summary
The PBAC recommended the General Schedule, Authority Required (telephone/online) listing of fenfluramine for the treatment of seizures associated with Dravet Syndrome, to be used in combination with at least two other anti-epileptic drugs (AEDs). In making this recommendation, the PBAC recognised the high unmet clinical need for effective therapies for this refractory and severe form of epilepsy.PSD · Nov 2024
The Committee considered there remained a high and unmet clinical need for additional effective therapies for Dravet Syndrome. The PBAC acknowledged Dravet Syndrome was a rare and severe developmental and epileptic encephalopathy which manifests through frequent, severe seizures, as well as cognitive and physical developmental delays and regression, which has a considerable impact on patients, parents, families and caregivers.PSD · Nov 2024
The ESC noted the economic model used a weighted ICER approach based on two cohorts stratified by use of concomitant stiripentol (either 100% for both fenfluramine and cannabidiol, or none), which individually produced vastly different ICERs (dominant in the + stiripentol cohort and $155,000 to < $255,000 per QALY in the no stiripentol cohort) and considered this highlighted major uncertainties with the model.PSD · Nov 2024
Further, the ESC noted that the relevant inputs were inconsistent with prior PBAC considerations for stiripentol and cannabidiol including time horizon, starting age, cannabidiol dose and extent of combination use of cannabidiol and stiripentol.PSD · Nov 2024
Fenfluramine vs cannabidiol The submission described fenfluramine as superior in terms of effectiveness compared with cannabidiol and similar in terms of safety compared to cannabidiol.PSD · Nov 2024
The therapeutic conclusion regarding efficacy presented in the submission was reasonably supported for fenfluramine vs cannabidiol. The indirect comparisons indicated that fenfluramine 0.7 mg/kg/day (without stiripentol) resulted in a greater decrease in the 28-day CSF and a greater increase in the proportion of participants who achieved ≥50% reduction in CSF compared with cannabidiol 10 or 20 mg/kg/day.PSD · Nov 2024
The PBAC noted the input from health professionals that described fenfluramine as having high-quality evidence for reducing seizures in Dravet Syndrome and being more effective than other available treatment options.PSD · Nov 2024
The PBAC welcomed the experiences shared by individuals, parents, family members and caregivers discussing both life with Dravet Syndrome and treatment with fenfluramine and noted consistent themes expressed including the transformative effects of treatment, including improved seizure control, improved language and cognitive development, improved physical function and motor skills.PSD · Nov 2024
Cost-effectiveness
ICER value redacted/commercially sensitive; pricing details marked with $ placeholders in document
Decision context
PopulationPatients with Dravet Syndrome aged 2 years and older with generalised tonic-clonic seizures or generalised clonic seizures not adequately controlled with at least 2 other antiepileptic drugs.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Nov 2024 | Recommended · restricted | cannabidiol | — | RCT · Convulsive and total seizure frequency |
Consumer voice
Health professionals, patients, family members, caregivers, and consumer organisations reported that fenfluramine provided transformative seizure control in Dravet Syndrome with improved cognitive and physical development, though access and cost were significant concerns without PBS listing. Multiple organisations supported PBS listing, citing high-quality evidence and unmet clinical need.
The health professionals considered that better seizure control was likely to have additional benefits including a reduced risk of sudden unexpected death in epilepsy (SUDEP) and the ability to reduce or cease other anti-epileptic drugs that have significant side effects. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to Dravet Syndrome only, excludes LGS, and requires prior failure of ≥2 antiepileptic drugs.