KALYDECO (ivacaftor) (mucoviscidose nourrissons 4-6 mois)

Opinions on drugs - Posted on Oct 04 2021

Reason for request

Extension d'indication

Key points

Favourable opinion for reimbursement in the treatment of infants aged at least 4 months to less than 6 months, weighing 5 kg to less than 25 kg with cystic fibrosis who have one of the gating (class III) mutations in the CFTR gene mentioned in the MA.

What therapeutic improvement?

Therapeutic improvement in the treatment of cystic fibrosis in infants aged at least 4 months to less than 6 months who have one of the gating (class III) mutations in the CFTR gene mentioned in the MA.

Role in the care pathway?

The management of cystic fibrosis patients requires the intervention of a multidisciplinary team

(primary care physicians, specialist centres, paramedical team with physiotherapist and nurse).

Symptomatic treatment, which needs to be life-long, is based on 4 complementary symptomatic

interventions:

respiratory management: physiotherapy, inhaled dornase alfa (which cannot be administered in patients under the age of 5 years), antibiotic therapy,
nutritional and digestive management,
implementation of optimum prevention of lung infections in accordance with the immunisation schedule,
patient therapeutic education.

Role of the medicinal product in the care pathway

As in children aged 6 months and over, KALYDECO (ivacaftor) is a long-term treatment for cystic fibrosis, which should be prescribed from the outset in cystic fibrosis patients aged at least 4 months to less than 6 months and weighing 5 kg to less than 25 kg, with one of the gating (class III) mutations in the CFTR gene mentioned in the MA.

The optimal treatment duration for KALYDECO (ivacaftor) is not known, but it is probably a life-long treatment.

Clinical Benefit

Substantial

The Committee deems that the clinical benefit of KALYDECO (ivacaftor) is substantial in the MA indication in infants aged at least 4 months to less than 6 months.

Clinical Added Value

important

Considering:

an efficacy and safety of ivacaftor in infants aged at least 4 months to less than 6 months similar to those observed in infants aged at least 6 months to less than 1 year, based on the results of a non-comparative phase 3 study,
the safety profile of ivacaftor which appears to be acceptable,
the substantial medical need in the management of cystic fibrosis in the absence of any other treatment targeting the causes of the disease, reported by patient associations,

and despite:

the follow-up limited to 24 weeks of treatment not enabling evaluation of the long-term efficacy and safety of ivacaftor,
the low number of patients in the study cohorts and the absence of comparative data from a historic cohort, for example,

the Committee considers that, as in children aged over 6 months, KALYDECO (ivacaftor) provides an important clinical added value (CAV II) in the treatment of cystic fibrosis in infants aged at least 4 months to less than 6 months and weighing 5 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.