FINTEPLA (fenfluramine) - Epilepsy

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.

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.