KALYDECO (ivacaftor) - Cystic fibrosis

Opinions on drugs - Posted on Jan 10 2025

Reason for request

Modification of the listing conditions

Summary of opinion

Favourable opinion for reimbursement only in “the treatment of infants aged at least 1 month to less than 4 months, weighing 3 kg to less than 25 kg with cystic fibrosis who have one of the following gating (class III) mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.”

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

Clinical Benefit

Substantial

The Committee deems that the clinical benefit of KALYDECO (ivacaftor) 13.4 mg, 25 mg, 50 mg, 75 mg granules in sachet is substantial only in the indication “the treatment of infants aged at least 1 month to less than 4 months, weighing 3 kg to less than 25 kg with cystic fibrosis who have one of the following gating (class III) mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R”.

Insufficient

The Committee deems that the clinical benefit of KALYDECO (ivacaftor) 13.4 mg, 25 mg, 50 mg, 75 mg granules in sachet is insufficient to justify public funding in the other MA situations.

Clinical Added Value

important

Considering:

an efficacy and safety of ivacaftor in infants aged at least 1 month to less than 4 months similar to those observed in older children, based on the results of the same non-comparative phase 3 study,
the safety profile of ivacaftor, which appears to be acceptable in this age group,
the substantial medical need in the treatment of cystic fibrosis in patients who have one of the gating (class III) mutations in the CFTR gene mentioned in the MA, in the absence of any other treatment targeting the causes of the disease,

and despite:

the follow-up limited to 24 weeks of treatment not enabling evaluation of the long-term efficacy and safety of ivacaftor,
the small size of the cohort concerned by the 1 to 4-months age category, missing data and the absence of comparative data versus a historical cohort, for example,

the Committee considers that, as in children aged over 4 months, KALYDECO (ivacaftor) 13.4 mg, 25 mg, 50 mg, 75 mg granules in sachet provides a substantial clinical added value (CAV II) in the treatment of cystic fibrosis in infants aged at least 1 month to less than 4 months and weighing 3 kg to less than 25 kg who have one of the following gating (class III) mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.