Favourable opinion for reimbursement for the treatment of seizures associated with Lennox-Gastaut syndrome as an add-on therapy to other anti-epileptic medicines for drug-resistant patients 2 years of age and older, only in last-resort situations.
Unfavourable opinion for reimbursement in the other situations covered by the MA indication.
Clinical Benefit
Substantial
The clinical benefit of FINTEPLA (fenfluramine) is substantial in the treatment of seizures associated with Lennox-Gastaut syndrome as an add-on therapy to other anti-epileptic medicines for drug-resistant patients 2 years of age and older, only in last-resort situations.
Insufficient
The clinical benefit of FINTEPLA (fenfluramine) is insufficient to justify public funding in view of the available alternatives in the other MA situations.
Clinical Added Value
minor
Considering:
evidence of the superiority of fenfluramine at the recommended maintenance dose of 0.7 mg/kg compared to placebo, as add-on therapy, assessed during a randomised, double-blind study in children and adults with Lennox-Gastaut syndrome:
for the primary endpoint of change in drop seizure frequency in the short term over 14 weeks, with a moderate additional effect size compared to placebo (median difference of -19.9%),
for the ranked secondary endpoint of proportion of patients responding to treatment (i.e. ≥ 50% reduction in drop seizures) over 14 weeks,
the medical need that remains substantial, due to the limited alternatives in this rare disease,
the absence of robust indirect comparative data versus cannabidiol (EPIDYOLEX) in a concomitant development context,
but:
the absence of robust data on quality of life,
the risk of pulmonary arterial hypertension now classed as an important identified risk following a post-marketing case, justifying the maintenance of supervision along with the need for echocardiogram monitoring, taking into account the history of the drug in the obesity indication in adults,
the absence of long-term efficacy and safety data for fenfluramine, particularly in terms of the impact on the neurocognitive deterioration and psychomotor development of patients, in the context of a chronic disease,
the Committee deems that FINTEPLA (fenfluramine) provides a minor clinical added value (CAV IV) in the current last-resort care pathway for the treatment of seizures associated with Lennox-Gastaut syndrome as an add-on therapy to other anti-epileptic medicines for drug-resistant patients 2 years of age and older.