Reason for request

First listing

Summary of opinion

Favourable opinion for reimbursement for the adjunctive treatment of epileptic seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients 2 to 17 years of age, only in the event of drug-resistant epilepsy.

Unfavourable opinion for reimbursement in the other situations covered by the MA indication.


Clinical Benefit

Moderate

The Committee deems that the clinical benefit of ZTALMY (ganaxolone) is moderate in the adjunctive treatment of epileptic seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients 2 to 17 years of age, only in the event of drug-resistant epilepsy.

Insufficient

The Committee deems that the clinical benefit of ZTALMY (ganaxolone) is insufficient to justify public funding cover in the other MA situations.


Clinical Added Value

no clinical added value

Considering:

  • demonstration of the superiority of ganaxolone compared to placebo, as adjunctive therapy, in a randomised, double-blind study, on a primary endpoint of variation in the frequency of major motor seizures in the short term (17 weeks), with a modest size effect (median difference of -27.08% in patients with a median number of 49 to 54 seizures per month at baseline), in a context of a high level of drug resistance,
  • the substantial medical need to have access to alternatives, due to the limited alternatives in this rare disease,

but in view of:

  • the absence of a demonstrated statistically significant difference compared to placebo for the percentage of patients presenting an at least 50% reduction in the number of seizures, a relevant ranked secondary endpoint,
  • the absence of robust data on quality of life, which is particularly impacted in this disease,
  • the absence of long-term efficacy and safety data for ganaxolone, in the context of a chronic disease,

the Committee considers that ZTALMY (ganaxolone) as adjunctive therapy provides no clinical added value (CAV V) in the treatment of drug-resistant seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients 2 to 17 years of age.

 


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